Trial Outcomes & Findings for A Study of BAX 930 in Children, Teenagers, and Adults Born With Thrombotic Thrombocytopenic Purpura (TTP) (NCT NCT03393975)
NCT ID: NCT03393975
Last Updated: 2026-02-09
Results Overview
As per planned analysis, data for this outcome measure were collected and reported only for the prophylaxis cohorts, in a combined manner for Periods 1 and 2 for TAK-755 and SoC treatments respectively, and separately for Period 3 in which all participants received TAK-755.
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
52 participants
Primary outcome timeframe
Up to 74.5 months
Results posted on
2026-02-09
Participant Flow
Participants took part in the study at various investigative sites globally from 13 October 2017 to 30 May 2024.
Participants with a diagnosis of severe congenital thrombotic thrombocytopenic purpura (cTTP) were enrolled in either prophylaxis or on demand cohorts. All participants received intravenous infusion of TAK-755 or standard treatment in Prophylaxis Periods 1 and 2, and TAK-755 in Prophylaxis Period 3 (hereafter Prophylaxis Periods 1, 2, and 3 are referred to simply as Periods 1, 2, and 3). 52 participants enrolled into the study but one participant withdrew before being randomized to treatment.
Participant milestones
| Measure |
Prophylaxis Cohort I: TAK-755 Then SoC
Participants received a single intravenous (IV) infusion of 40 international units per kilogram (IU/kg) rADAMTS13 manufactured in Orth, Austria (TAK-755 ORT), every 2 weeks (Q2W) for 6 months in Period 1 followed by standard of care (SoC) for 6 months in Period 2. Thereafter participants received rADAMTS13 manufactured in Singapore (TAK-755 SIN), dose IV infusion of 40 IU/kg Q2W for 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
Prophylaxis Cohort II: SoC Then TAK-755
Participants received SoC for 6 months in Period 1 followed by IV infusions of 40 IU/kg dose of TAK-755 ORT Q2W in Period 2 for the next 6 months. Thereafter participants received TAK-755 SIN dose IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
On Demand Cohort I: TAK-755
Participants experiencing an acute TTP event who met all other inclusion criteria and entered the study through the TAK-755 cohort of the Urgent Treatment Period received initial dose of IV infusion 40 IU/kg \[+/- 4 IU/kg\] TAK-755 ORT or TAK-755 SIN on Day 1 followed by a subsequent dose IV infusions of 20 IU/kg \[+/- 2 IU/kg\] TAK-755 ORT or TAK-755 SIN on Day 2 and an additional daily dose IV infusions of 15 IU/kg \[+/- 1.5 IU/kg\] TAK-755 on Day 3 until 2 days after the acute event was resolved. Upon resolution of the acute TTP event, participants had the option to either move to the prophylaxis cohort of the study or discontinue entirely.
|
On Demand Cohort II: SoC
Participants experiencing an acute TTP event who met all other inclusion criteria and entered the study through the SoC cohort of the Urgent Treatment Period received the investigator-recommended SoC and dosing regimen until the acute event was resolved. Upon resolution of the acute TTP event, participants had the option to either move to the prophylaxis cohort of the study or discontinue entirely.
|
|---|---|---|---|---|
|
Urgent Treatment Period
STARTED
|
0
|
0
|
2
|
4
|
|
Urgent Treatment Period
Safety Analysis Set
|
0
|
0
|
2
|
4
|
|
Urgent Treatment Period
Full Analysis Set
|
0
|
0
|
2
|
4
|
|
Urgent Treatment Period
Modified Full Analysis Set
|
0
|
0
|
2
|
4
|
|
Prophylaxis Period 2
STARTED
|
23
|
24
|
0
|
0
|
|
Prophylaxis Period 2
COMPLETED
|
23
|
23
|
0
|
0
|
|
Urgent Treatment Period
COMPLETED
|
0
|
0
|
2
|
3
|
|
Urgent Treatment Period
NOT COMPLETED
|
0
|
0
|
0
|
1
|
|
Prophylaxis Period 1
STARTED
|
23
|
25
|
0
|
0
|
|
Prophylaxis Period 1
Safety Analysis Set
|
23
|
25
|
0
|
0
|
|
Prophylaxis Period 1
Full Analysis Set
|
23
|
24
|
0
|
0
|
|
Prophylaxis Period 1
Modified Full Analysis Set
|
21
|
24
|
0
|
0
|
|
Prophylaxis Period 1
COMPLETED
|
23
|
24
|
0
|
0
|
|
Prophylaxis Period 1
NOT COMPLETED
|
0
|
1
|
0
|
0
|
|
Prophylaxis Period 2
NOT COMPLETED
|
0
|
1
|
0
|
0
|
|
Prophylaxis Period 3
STARTED
|
23
|
23
|
0
|
0
|
|
Prophylaxis Period 3
COMPLETED
|
23
|
23
|
0
|
0
|
|
Prophylaxis Period 3
NOT COMPLETED
|
0
|
0
|
0
|
0
|
Reasons for withdrawal
| Measure |
Prophylaxis Cohort I: TAK-755 Then SoC
Participants received a single intravenous (IV) infusion of 40 international units per kilogram (IU/kg) rADAMTS13 manufactured in Orth, Austria (TAK-755 ORT), every 2 weeks (Q2W) for 6 months in Period 1 followed by standard of care (SoC) for 6 months in Period 2. Thereafter participants received rADAMTS13 manufactured in Singapore (TAK-755 SIN), dose IV infusion of 40 IU/kg Q2W for 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
Prophylaxis Cohort II: SoC Then TAK-755
Participants received SoC for 6 months in Period 1 followed by IV infusions of 40 IU/kg dose of TAK-755 ORT Q2W in Period 2 for the next 6 months. Thereafter participants received TAK-755 SIN dose IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
On Demand Cohort I: TAK-755
Participants experiencing an acute TTP event who met all other inclusion criteria and entered the study through the TAK-755 cohort of the Urgent Treatment Period received initial dose of IV infusion 40 IU/kg \[+/- 4 IU/kg\] TAK-755 ORT or TAK-755 SIN on Day 1 followed by a subsequent dose IV infusions of 20 IU/kg \[+/- 2 IU/kg\] TAK-755 ORT or TAK-755 SIN on Day 2 and an additional daily dose IV infusions of 15 IU/kg \[+/- 1.5 IU/kg\] TAK-755 on Day 3 until 2 days after the acute event was resolved. Upon resolution of the acute TTP event, participants had the option to either move to the prophylaxis cohort of the study or discontinue entirely.
|
On Demand Cohort II: SoC
Participants experiencing an acute TTP event who met all other inclusion criteria and entered the study through the SoC cohort of the Urgent Treatment Period received the investigator-recommended SoC and dosing regimen until the acute event was resolved. Upon resolution of the acute TTP event, participants had the option to either move to the prophylaxis cohort of the study or discontinue entirely.
|
|---|---|---|---|---|
|
Urgent Treatment Period
Physician Decision
|
0
|
0
|
0
|
1
|
|
Prophylaxis Period 1
Reason not Specified
|
0
|
1
|
0
|
0
|
|
Prophylaxis Period 2
Reason not Specified
|
0
|
1
|
0
|
0
|
Baseline Characteristics
A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
Baseline characteristics by cohort
| Measure |
Prophylaxis Cohort I: TAK-755 Then SoC
n=21 Participants
Participants received a single IV infusion of 40 IU/kg TAK-755 ORT Q2W for 6 months in Period 1 followed by SoC for 6 months in Period 2. Thereafter participants received TAK-755 SIN dose IV infusion of 40 IU/kg Q2W for 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
Prophylaxis Cohort II: SoC Then TAK-755
n=27 Participants
Participants received SoC for 6 months in Period 1 followed by IV infusions of 40 IU/kg dose of TAK-755 ORT Q2W in Period 2 for the next 6 months. Thereafter participants received TAK-755 SIN dose IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
On Demand Cohort I: TAK-755
n=2 Participants
Participants experiencing an acute TTP event who met all other inclusion criteria and entered the study through the TAK-755 cohort of the Urgent Treatment Period received initial dose of IV infusions 40 IU/kg \[+/- 4 IU/kg\] TAK-755 ORT or TAK-755 SIN on Day 1 followed by a subsequent dose IV infusion of 20 IU/kg \[+/- 2 IU/kg\] TAK-755 ORT or TAK-755 SIN on Day 2 and an additional daily dose IV infusions of 15 IU/kg \[+/- 1.5 IU/kg\] TAK-755 on Day 3 until 2 days after the acute event was resolved. Upon resolution of the acute TTP event, participants had the option to either move to the prophylaxis cohort of the study or discontinue entirely.
|
On Demand Cohort II: SoC
n=4 Participants
Participants experiencing an acute TTP event who met all other inclusion criteria and entered the study through the SoC cohort of the Urgent Treatment Period received the investigator-recommended SoC and dosing regimen until the acute event was resolved. Upon resolution of the acute TTP event, participants had the option to either move to the prophylaxis cohort of the study or discontinue entirely.
|
Total
n=54 Participants
Total of all reporting groups
|
|---|---|---|---|---|---|
|
Age, Customized
Prophylaxis Cohort · ≥18 years
|
16 Participants
n=21 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
20 Participants
n=27 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
0 Participants
A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
0 Participants
A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
36 Participants
n=48 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
|
Age, Customized
Prophylaxis Cohort · 12 to <18 years
|
1 Participants
n=21 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
3 Participants
n=27 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
0 Participants
A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
0 Participants
A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
4 Participants
n=48 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
|
Age, Customized
Prophylaxis Cohort · 6 to <12 years
|
1 Participants
n=21 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
3 Participants
n=27 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
0 Participants
A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
0 Participants
A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
4 Participants
n=48 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
|
Age, Customized
Prophylaxis Cohort · <6 years
|
3 Participants
n=21 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
1 Participants
n=27 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
0 Participants
A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
0 Participants
A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
4 Participants
n=48 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
|
Age, Customized
On Demand Cohort · ≥18 years
|
0 Participants
A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
0 Participants
A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
2 Participants
n=2 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
3 Participants
n=4 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
5 Participants
n=6 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
|
Age, Customized
On Demand Cohort · 12 to <18 years
|
0 Participants
A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
0 Participants
A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
0 Participants
n=2 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
0 Participants
n=4 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
0 Participants
n=6 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
|
Age, Customized
On Demand Cohort · 6 to <12 years
|
0 Participants
A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
0 Participants
A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
0 Participants
n=2 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
0 Participants
n=4 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
0 Participants
n=6 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
|
Age, Customized
On Demand Cohort · <6 years
|
0 Participants
A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
0 Participants
A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
0 Participants
n=2 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
1 Participants
n=4 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
1 Participants
n=6 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
|
Sex: Female, Male
Prophylaxis Cohort · Female
|
12 Participants
n=21 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
16 Participants
n=27 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
0 Participants
A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
0 Participants
A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
28 Participants
n=48 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
|
Sex: Female, Male
Prophylaxis Cohort · Male
|
9 Participants
n=21 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
11 Participants
n=27 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
0 Participants
A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
0 Participants
A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
20 Participants
n=48 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
|
Sex: Female, Male
On Demand Cohort · Female
|
0 Participants
A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
0 Participants
A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
1 Participants
n=2 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
1 Participants
n=4 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
2 Participants
n=6 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
|
Sex: Female, Male
On Demand Cohort · Male
|
0 Participants
A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
0 Participants
A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
1 Participants
n=2 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
3 Participants
n=4 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
4 Participants
n=6 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
|
Ethnicity (NIH/OMB)
Prophylaxis Cohort · Hispanic or Latino
|
1 Participants
n=21 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
0 Participants
n=27 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
0 Participants
A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
0 Participants
A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
1 Participants
n=48 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
|
Ethnicity (NIH/OMB)
Prophylaxis Cohort · Not Hispanic or Latino
|
16 Participants
n=21 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
23 Participants
n=27 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
0 Participants
A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
0 Participants
A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
39 Participants
n=48 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
|
Ethnicity (NIH/OMB)
Prophylaxis Cohort · Unknown or Not Reported
|
4 Participants
n=21 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
4 Participants
n=27 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
0 Participants
A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
0 Participants
A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
8 Participants
n=48 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
|
Ethnicity (NIH/OMB)
On Demand Cohort · Hispanic or Latino
|
0 Participants
A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
0 Participants
A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
0 Participants
n=2 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
0 Participants
n=4 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
0 Participants
n=6 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
|
Ethnicity (NIH/OMB)
On Demand Cohort · Not Hispanic or Latino
|
0 Participants
A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
0 Participants
A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
2 Participants
n=2 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
4 Participants
n=4 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
6 Participants
n=6 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
|
Ethnicity (NIH/OMB)
On Demand Cohort · Unknown or Not Reported
|
0 Participants
A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
0 Participants
A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
0 Participants
n=2 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
0 Participants
n=4 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
0 Participants
n=6 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
|
Race (NIH/OMB)
Prophylaxis Cohort · American Indian or Alaska Native
|
0 Participants
n=21 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
0 Participants
n=27 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
0 Participants
A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
0 Participants
A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
0 Participants
n=48 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
|
Race (NIH/OMB)
Prophylaxis Cohort · Asian
|
2 Participants
n=21 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
3 Participants
n=27 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
0 Participants
A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
0 Participants
A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
5 Participants
n=48 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
|
Race (NIH/OMB)
Prophylaxis Cohort · Native Hawaiian or Other Pacific Islander
|
0 Participants
n=21 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
0 Participants
n=27 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
0 Participants
A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
0 Participants
A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
0 Participants
n=48 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
|
Race (NIH/OMB)
Prophylaxis Cohort · Black or African American
|
0 Participants
n=21 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
1 Participants
n=27 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
0 Participants
A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
0 Participants
A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
1 Participants
n=48 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
|
Race (NIH/OMB)
Prophylaxis Cohort · White
|
15 Participants
n=21 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
17 Participants
n=27 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
0 Participants
A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
0 Participants
A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
32 Participants
n=48 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
|
Race (NIH/OMB)
Prophylaxis Cohort · More than one race
|
0 Participants
n=21 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
1 Participants
n=27 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
0 Participants
A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
0 Participants
A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
1 Participants
n=48 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
|
Race (NIH/OMB)
Prophylaxis Cohort · Unknown or Not Reported
|
4 Participants
n=21 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
5 Participants
n=27 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
0 Participants
A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
0 Participants
A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
9 Participants
n=48 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
|
Race (NIH/OMB)
On Demand Cohort · American Indian or Alaska Native
|
0 Participants
A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
0 Participants
A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
0 Participants
n=2 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
0 Participants
n=4 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
0 Participants
n=6 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
|
Race (NIH/OMB)
On Demand Cohort · Asian
|
0 Participants
A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
0 Participants
A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
1 Participants
n=2 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
0 Participants
n=4 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
1 Participants
n=6 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
|
Race (NIH/OMB)
On Demand Cohort · Native Hawaiian or Other Pacific Islander
|
0 Participants
A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
0 Participants
A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
0 Participants
n=2 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
0 Participants
n=4 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
0 Participants
n=6 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
|
Race (NIH/OMB)
On Demand Cohort · Black or African American
|
0 Participants
A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
0 Participants
A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
0 Participants
n=2 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
1 Participants
n=4 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
1 Participants
n=6 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
|
Race (NIH/OMB)
On Demand Cohort · White
|
0 Participants
A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
0 Participants
A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
1 Participants
n=2 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
2 Participants
n=4 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
3 Participants
n=6 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
|
Race (NIH/OMB)
On Demand Cohort · More than one race
|
0 Participants
A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
0 Participants
A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
0 Participants
n=2 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
1 Participants
n=4 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
1 Participants
n=6 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
|
Race (NIH/OMB)
On Demand Cohort · Unknown or Not Reported
|
0 Participants
A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
0 Participants
A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
0 Participants
n=2 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
0 Participants
n=4 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
0 Participants
n=6 Participants • A cohort-wise data representation is used and number analyzed denotes the number of participants in each cohort presented row-wise.
|
PRIMARY outcome
Timeframe: Up to 74.5 monthsPopulation: Modified Full Analysis Set(mFAS): all FAS participants who were treated according to their randomized treatment sequence with exclusion of those enrolled prior to Nov. 2017 who were treated with SoC instead of randomized treatment of TAK-755 in period 1 because TAK-755 was unavailable.For participants enrolled prior to Nov 2017,only first 6 months of SoC treatment in period 1 was included in analysis.Overall number of participants analyzed:number of participants with data available for analyses.
As per planned analysis, data for this outcome measure were collected and reported only for the prophylaxis cohorts, in a combined manner for Periods 1 and 2 for TAK-755 and SoC treatments respectively, and separately for Period 3 in which all participants received TAK-755.
Outcome measures
| Measure |
Prophylaxis Cohort: TAK-755 (Period 3)
n=44 Participants
Participants received TAK-755 SIN dose of IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
Prophylaxis Cohort: SoC (Periods 1 and 2)
n=45 Participants
Participants received SoC for 6 months in either Period 1 or Period 2.
|
Prophylaxis Cohort: TAK-755
n=44 Participants
Participants received a single IV infusion of 40 IU/kg TAK-755 ORT Q2W for 6 months in either Period 1 or Period 2. Thereafter participants received TAK-755 SIN dose of IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
On Demand Cohort II: SoC
Participants experiencing an acute TTP event who met all other inclusion criteria and entered the study through the SoC cohort of the Urgent Treatment Period received the investigator-recommended SoC and dosing regimen until the acute event was resolved. Upon resolution of the acute TTP event, participants had the option to either move to the prophylaxis cohort of the study or discontinue entirely.
|
|---|---|---|---|---|
|
Number of Participants With Acute Thrombotic Thrombocytopenic Purpura (TTP) Events During Prophylactic Treatment
|
0 Participants
|
1 Participants
|
0 Participants
|
—
|
SECONDARY outcome
Timeframe: Up to 79.6 monthsPopulation: mFAS:all FAS participants who were treated according to their randomized treatment sequence with exclusion of those enrolled prior to Nov.2017 who were treated with SoC instead of randomized treatment of TAK-755 in period 1 as TAK-755 was unavailable. For participants enrolled prior to Nov 2017,only first 6 months of SoC treatment in period 1 was included in analysis.Overall number of participants: participants with acute TTP events that were confirmed by central lab data treated with TAK-755.
Percentage of acute TTP events responding to TAK-755, was defined as not requiring the use of another human disintegrin and metalloprotease with a thrombospondin type 1 motif, member 13 (ADAMTS13)-containing agent. As per planned analysis, data for this outcome measure were collected and reported only for the TAK-755 treatment arm of both the prophylaxis (irrespective of the prophylaxis periods) and on demand cohorts.
Outcome measures
| Measure |
Prophylaxis Cohort: TAK-755 (Period 3)
n=1 Participants
Participants received TAK-755 SIN dose of IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
Prophylaxis Cohort: SoC (Periods 1 and 2)
Participants received SoC for 6 months in either Period 1 or Period 2.
|
Prophylaxis Cohort: TAK-755
Participants received a single IV infusion of 40 IU/kg TAK-755 ORT Q2W for 6 months in either Period 1 or Period 2. Thereafter participants received TAK-755 SIN dose of IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
On Demand Cohort II: SoC
Participants experiencing an acute TTP event who met all other inclusion criteria and entered the study through the SoC cohort of the Urgent Treatment Period received the investigator-recommended SoC and dosing regimen until the acute event was resolved. Upon resolution of the acute TTP event, participants had the option to either move to the prophylaxis cohort of the study or discontinue entirely.
|
|---|---|---|---|---|
|
Percentage of Acute Thrombotic Thrombocytopenic Purpura (TTP) Events Responding to TAK-755
|
100 percentage of events
|
—
|
—
|
—
|
SECONDARY outcome
Timeframe: Up to 79.6 monthsPopulation: mFAS: all FAS participants who were treated according to their randomized treatment sequence with exclusion of those enrolled prior to Nov. 2017 who were treated with SoC instead of randomized treatment of TAK-755 in period 1 as TAK-755 was unavailable. For participants enrolled prior to Nov. 2017,only first 6 months of SoC treatment in period 1 was included in analysis.Overall number of participants analyzed:number of participants with acute TTP events that were confirmed by central lab data.
Time to resolution of acute TTP events following initiation of treatment with TAK-755 or SoC agent was assessed. Acute TPP events were considered resolved when: (a) Platelet count was \>150,000 per microliter (μL) or drop of platelet count was within 25 percent (%) of baseline, whichever occurred first, and (b) Elevation of lactate dehydrogenase (LDH) \<1.5 x baseline or \<1.5 x upper limit of normal (ULN). As per planned analysis, data for this outcome measure were collected and reported in a combined manner irrespective of the prophylaxis treatment Periods, partitioned per treatment received (TAK-755 and SoC) for the on demand and prophylactic cohorts.
Outcome measures
| Measure |
Prophylaxis Cohort: TAK-755 (Period 3)
n=1 Participants
Participants received TAK-755 SIN dose of IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
Prophylaxis Cohort: SoC (Periods 1 and 2)
n=1 Participants
Participants received SoC for 6 months in either Period 1 or Period 2.
|
Prophylaxis Cohort: TAK-755
Participants received a single IV infusion of 40 IU/kg TAK-755 ORT Q2W for 6 months in either Period 1 or Period 2. Thereafter participants received TAK-755 SIN dose of IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
On Demand Cohort II: SoC
n=1 Participants
Participants experiencing an acute TTP event who met all other inclusion criteria and entered the study through the SoC cohort of the Urgent Treatment Period received the investigator-recommended SoC and dosing regimen until the acute event was resolved. Upon resolution of the acute TTP event, participants had the option to either move to the prophylaxis cohort of the study or discontinue entirely.
|
|---|---|---|---|---|
|
Time to Resolution of Acute TTP Events
|
14.8 days
95% confidence interval was not estimable due to censoring.
|
3.0 days
95% confidence interval was not estimable due to censoring.
|
—
|
1.5 days
95% confidence interval was not estimable due to censoring.
|
SECONDARY outcome
Timeframe: Up to 79.6 monthsPopulation: mFAS included all FAS participants who were treated according to their randomized treatment sequence with exclusion of those enrolled prior to Nov. 2017 who were treated with SoC instead of randomized treatment of TAK-755 in period 1 as TAK-755 was unavailable. For participants enrolled prior to Nov. 2017, only first 6 months of SoC treatment in period 1 was included in analysis. Overall number of participants analyzed indicates the number of participants with data available for analyses.
Thrombocytopenia was defined as a decrease in platelet count ≥25 % of baseline or a platelet count \<150,000/μL, reported by treatment arm for the prophylactic cohort. As per planned analysis, data for this outcome measure were collected and reported only for the prophylaxis cohorts, in a combined manner for Periods 1 and 2 for TAK-755 and SoC treatments respectively, and separately for Period 3 in which all participants received TAK-755.
Outcome measures
| Measure |
Prophylaxis Cohort: TAK-755 (Period 3)
n=44 Participants
Participants received TAK-755 SIN dose of IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
Prophylaxis Cohort: SoC (Periods 1 and 2)
n=45 Participants
Participants received SoC for 6 months in either Period 1 or Period 2.
|
Prophylaxis Cohort: TAK-755
n=44 Participants
Participants received a single IV infusion of 40 IU/kg TAK-755 ORT Q2W for 6 months in either Period 1 or Period 2. Thereafter participants received TAK-755 SIN dose of IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
On Demand Cohort II: SoC
Participants experiencing an acute TTP event who met all other inclusion criteria and entered the study through the SoC cohort of the Urgent Treatment Period received the investigator-recommended SoC and dosing regimen until the acute event was resolved. Upon resolution of the acute TTP event, participants had the option to either move to the prophylaxis cohort of the study or discontinue entirely.
|
|---|---|---|---|---|
|
Number of Participants With Thrombocytopenia During Prophylactic Treatment
|
11 Participants
|
21 Participants
|
13 Participants
|
—
|
SECONDARY outcome
Timeframe: Up to 79.6 monthsPopulation: mFAS included all FAS participants who were treated according to their randomized treatment sequence with exclusion of those enrolled prior to Nov. 2017 who were treated with SoC instead of randomized treatment of TAK-755 in period 1 as TAK-755 was unavailable. For participants enrolled prior to Nov. 2017, only first 6 months of SoC treatment in period 1 was included in analysis. Overall number of participants analyzed indicates the number of participants with data available for analyses.
Microangiopathic hemolytic anemia was defined as an elevation of LDH \>1.5\* of baseline or \>1.5\*ULN (with a possible evidence of schistocytes on blood smear) and was reported by treatment arm for the prophylactic cohort. As per planned analysis, data for this outcome measure were collected and reported only for the prophylaxis cohorts, in a combined manner for Periods 1 and 2 for TAK-755 and SoC treatments respectively, and separately for Period 3 in which all participants received TAK-755.
Outcome measures
| Measure |
Prophylaxis Cohort: TAK-755 (Period 3)
n=44 Participants
Participants received TAK-755 SIN dose of IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
Prophylaxis Cohort: SoC (Periods 1 and 2)
n=45 Participants
Participants received SoC for 6 months in either Period 1 or Period 2.
|
Prophylaxis Cohort: TAK-755
n=44 Participants
Participants received a single IV infusion of 40 IU/kg TAK-755 ORT Q2W for 6 months in either Period 1 or Period 2. Thereafter participants received TAK-755 SIN dose of IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
On Demand Cohort II: SoC
Participants experiencing an acute TTP event who met all other inclusion criteria and entered the study through the SoC cohort of the Urgent Treatment Period received the investigator-recommended SoC and dosing regimen until the acute event was resolved. Upon resolution of the acute TTP event, participants had the option to either move to the prophylaxis cohort of the study or discontinue entirely.
|
|---|---|---|---|---|
|
Number of Participants With Microangiopathic Hemolytic Anemia During Prophylactic Treatment
|
13 Participants
|
12 Participants
|
8 Participants
|
—
|
SECONDARY outcome
Timeframe: Up to 79.6 monthsPopulation: mFAS included all FAS participants who were treated according to their randomized treatment sequence with exclusion of those enrolled prior to Nov. 2017 who were treated with SoC instead of randomized treatment of TAK-755 in period 1 as TAK-755 was unavailable. For participants enrolled prior to Nov. 2017, only first 6 months of SoC treatment in period 1 was included in analysis. Overall number of participants analyzed indicates the number of participants with data available for analyses.
Neurological symptoms (TTP related) (e.g., confusion, dysphonia, dysarthria, focal or general motor symptoms including seizures), were reported by treatment arm for the prophylactic cohort. As per planned analysis, data for this outcome measure were collected and reported only for the prophylaxis cohorts, in a combined manner for Periods 1 and 2 for TAK-755 and SoC treatments respectively, and separately for Period 3 in which all participants received TAK-755.
Outcome measures
| Measure |
Prophylaxis Cohort: TAK-755 (Period 3)
n=44 Participants
Participants received TAK-755 SIN dose of IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
Prophylaxis Cohort: SoC (Periods 1 and 2)
n=45 Participants
Participants received SoC for 6 months in either Period 1 or Period 2.
|
Prophylaxis Cohort: TAK-755
n=44 Participants
Participants received a single IV infusion of 40 IU/kg TAK-755 ORT Q2W for 6 months in either Period 1 or Period 2. Thereafter participants received TAK-755 SIN dose of IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
On Demand Cohort II: SoC
Participants experiencing an acute TTP event who met all other inclusion criteria and entered the study through the SoC cohort of the Urgent Treatment Period received the investigator-recommended SoC and dosing regimen until the acute event was resolved. Upon resolution of the acute TTP event, participants had the option to either move to the prophylaxis cohort of the study or discontinue entirely.
|
|---|---|---|---|---|
|
Number of Participants With Neurological Symptoms During Prophylactic Treatment
|
9 Participants
|
7 Participants
|
4 Participants
|
—
|
SECONDARY outcome
Timeframe: Up to 79.6 monthsPopulation: mFAS included all FAS participants who were treated according to their randomized treatment sequence with exclusion of those enrolled prior to Nov. 2017 who were treated with SoC instead of randomized treatment of TAK-755 in period 1 as TAK-755 was unavailable. For participants enrolled prior to Nov. 2017, only first 6 months of SoC treatment in period 1 was included in analysis. Overall number of participants analyzed indicates the number of participants with data available for analyses.
Renal dysfunction was defined as an increase in serum creatinine \>1.5\*baseline. Number of participants with renal dysfunction were reported by treatment arm for the prophylactic cohort. As per planned analysis, data for this outcome measure were collected and reported only for the prophylaxis cohorts, in a combined manner for Periods 1 and 2 for TAK-755 and SoC treatments respectively, and separately for Period 3 in which all participants received TAK-755.
Outcome measures
| Measure |
Prophylaxis Cohort: TAK-755 (Period 3)
n=44 Participants
Participants received TAK-755 SIN dose of IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
Prophylaxis Cohort: SoC (Periods 1 and 2)
n=45 Participants
Participants received SoC for 6 months in either Period 1 or Period 2.
|
Prophylaxis Cohort: TAK-755
n=44 Participants
Participants received a single IV infusion of 40 IU/kg TAK-755 ORT Q2W for 6 months in either Period 1 or Period 2. Thereafter participants received TAK-755 SIN dose of IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
On Demand Cohort II: SoC
Participants experiencing an acute TTP event who met all other inclusion criteria and entered the study through the SoC cohort of the Urgent Treatment Period received the investigator-recommended SoC and dosing regimen until the acute event was resolved. Upon resolution of the acute TTP event, participants had the option to either move to the prophylaxis cohort of the study or discontinue entirely.
|
|---|---|---|---|---|
|
Number of Participants With Renal Dysfunction During Prophylactic Treatment
|
4 Participants
|
2 Participants
|
5 Participants
|
—
|
SECONDARY outcome
Timeframe: Up to 79.6 monthsPopulation: mFAS included all FAS participants who were treated according to their randomized treatment sequence with exclusion of those enrolled prior to Nov. 2017 who were treated with SoC instead of randomized treatment of TAK-755 in period 1 as TAK-755 was unavailable. For participants enrolled prior to Nov. 2017, only first 6 months of SoC treatment in period 1 was included in analysis. Overall number of participants analyzed indicates the number of participants with data available for analyses.
Number of participants with abdominal pain (TTP related) were reported by treatment arm for the prophylaxis cohort. As per planned analysis, data for this outcome measure were collected and reported only for the prophylaxis cohorts, in a combined manner for Periods 1 and 2 for TAK-755 and SoC treatments respectively, and separately for Period 3 in which all participants received TAK-755.
Outcome measures
| Measure |
Prophylaxis Cohort: TAK-755 (Period 3)
n=44 Participants
Participants received TAK-755 SIN dose of IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
Prophylaxis Cohort: SoC (Periods 1 and 2)
n=45 Participants
Participants received SoC for 6 months in either Period 1 or Period 2.
|
Prophylaxis Cohort: TAK-755
n=44 Participants
Participants received a single IV infusion of 40 IU/kg TAK-755 ORT Q2W for 6 months in either Period 1 or Period 2. Thereafter participants received TAK-755 SIN dose of IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
On Demand Cohort II: SoC
Participants experiencing an acute TTP event who met all other inclusion criteria and entered the study through the SoC cohort of the Urgent Treatment Period received the investigator-recommended SoC and dosing regimen until the acute event was resolved. Upon resolution of the acute TTP event, participants had the option to either move to the prophylaxis cohort of the study or discontinue entirely.
|
|---|---|---|---|---|
|
Number of Participants With Abdominal Pain During Prophylactic Treatment
|
2 Participants
|
6 Participants
|
2 Participants
|
—
|
SECONDARY outcome
Timeframe: Up to 79.6 monthsPopulation: mFAS included all FAS participants who were treated according to their randomized treatment sequence with exclusion of those enrolled prior to Nov. 2017 who were treated with SoC instead of randomized treatment of TAK-755 in period 1 as TAK-755 was unavailable. For participants enrolled prior to Nov. 2017, only first 6 months of SoC treatment in period 1 was included in analysis. Overall number of participants analyzed indicates the number of participants with data available for analyses.
Number of supplemental doses prompted by subacute TTP events were reported by treatment for the prophylactic cohort. As per planned analysis, data for this outcome measure were collected and reported only for the prophylaxis cohorts, in a combined manner for Periods 1 and 2 for TAK-755 and SoC treatments respectively, and separately for Period 3 in which all participants received TAK-755.
Outcome measures
| Measure |
Prophylaxis Cohort: TAK-755 (Period 3)
n=44 Participants
Participants received TAK-755 SIN dose of IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
Prophylaxis Cohort: SoC (Periods 1 and 2)
n=45 Participants
Participants received SoC for 6 months in either Period 1 or Period 2.
|
Prophylaxis Cohort: TAK-755
n=44 Participants
Participants received a single IV infusion of 40 IU/kg TAK-755 ORT Q2W for 6 months in either Period 1 or Period 2. Thereafter participants received TAK-755 SIN dose of IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
On Demand Cohort II: SoC
Participants experiencing an acute TTP event who met all other inclusion criteria and entered the study through the SoC cohort of the Urgent Treatment Period received the investigator-recommended SoC and dosing regimen until the acute event was resolved. Upon resolution of the acute TTP event, participants had the option to either move to the prophylaxis cohort of the study or discontinue entirely.
|
|---|---|---|---|---|
|
Number of Supplemental Doses Prompted by Subacute TTP Event During Prophylactic Treatment
|
5 supplemental doses
|
9 supplemental doses
|
0 supplemental doses
|
—
|
SECONDARY outcome
Timeframe: Up to 79.6 monthsPopulation: mFAS included all FAS participants who were treated according to their randomized treatment sequence with exclusion of those enrolled prior to Nov. 2017 who were treated with SoC instead of randomized treatment of TAK-755 in period 1 as TAK-755 was unavailable. For participants enrolled prior to Nov. 2017, only first 6 months of SoC treatment in period 1 was included in analysis. Overall number of participants analyzed indicates the number of participants with data available for analyses.
Number of participants with dose modification not prompted by an acute TTP event were reported by treatment for the prophylactic cohort. As per planned analysis, data for this outcome measure were collected and reported only for the prophylaxis cohorts, in a combined manner for Periods 1 and 2 for TAK-755 and SoC treatments respectively, and separately for Period 3 in which all participants received TAK-755.
Outcome measures
| Measure |
Prophylaxis Cohort: TAK-755 (Period 3)
n=44 Participants
Participants received TAK-755 SIN dose of IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
Prophylaxis Cohort: SoC (Periods 1 and 2)
n=45 Participants
Participants received SoC for 6 months in either Period 1 or Period 2.
|
Prophylaxis Cohort: TAK-755
n=44 Participants
Participants received a single IV infusion of 40 IU/kg TAK-755 ORT Q2W for 6 months in either Period 1 or Period 2. Thereafter participants received TAK-755 SIN dose of IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
On Demand Cohort II: SoC
Participants experiencing an acute TTP event who met all other inclusion criteria and entered the study through the SoC cohort of the Urgent Treatment Period received the investigator-recommended SoC and dosing regimen until the acute event was resolved. Upon resolution of the acute TTP event, participants had the option to either move to the prophylaxis cohort of the study or discontinue entirely.
|
|---|---|---|---|---|
|
Number of Participants With Dose Modification Not Prompted by an Acute TTP Event During Prophylactic Treatment
|
1 Participants
|
3 Participants
|
0 Participants
|
—
|
SECONDARY outcome
Timeframe: Up to 79.6 monthsPopulation: mFAS included all FAS participants who were treated according to their randomized treatment sequence with exclusion of those enrolled prior to Nov. 2017 who were treated with SoC instead of randomized treatment of TAK-755 in period 1 as TAK-755 was unavailable. For participants enrolled prior to Nov. 2017, only first 6 months of SoC treatment in period 1 was included in analysis. Overall number of participants analyzed indicates the number of participants with data available for analyses.
Number of participants with acute TTP events on their final dose and dosing regimen for the prophylactic cohort were reported. As per planned analysis, data for this outcome measure were collected and reported only for the prophylaxis cohorts, in a combined manner for Periods 1 and 2 for TAK-755 and SoC treatments respectively, and separately for Period 3 in which all participants received TAK-755.
Outcome measures
| Measure |
Prophylaxis Cohort: TAK-755 (Period 3)
n=44 Participants
Participants received TAK-755 SIN dose of IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
Prophylaxis Cohort: SoC (Periods 1 and 2)
n=45 Participants
Participants received SoC for 6 months in either Period 1 or Period 2.
|
Prophylaxis Cohort: TAK-755
n=44 Participants
Participants received a single IV infusion of 40 IU/kg TAK-755 ORT Q2W for 6 months in either Period 1 or Period 2. Thereafter participants received TAK-755 SIN dose of IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
On Demand Cohort II: SoC
Participants experiencing an acute TTP event who met all other inclusion criteria and entered the study through the SoC cohort of the Urgent Treatment Period received the investigator-recommended SoC and dosing regimen until the acute event was resolved. Upon resolution of the acute TTP event, participants had the option to either move to the prophylaxis cohort of the study or discontinue entirely.
|
|---|---|---|---|---|
|
Number of Participants With Acute TTP Events on Their Final Dose
|
0 Participants
|
1 Participants
|
0 Participants
|
—
|
SECONDARY outcome
Timeframe: Up to 79.6 monthsPopulation: The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for this outcome measure were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
AE: Any untoward medical occurrence in participants administered IP that does not necessarily have a causal relationship with treatment. TEAE: AE that has start date-time on/after start date-time of first dose of treatment participant is taking on that assessment/period or if it has start date-time before start date-time of first dose but increases in severity on/after start date-time of the first dose of treatment. SAE: An untoward medical occurrence that at any dose meets 1 or more of following criteria: death; initial/prolonged in-patient hospitalization; life threatening experience; persistent/significant disability/incapacity; congenital anomaly, medically important event (may not be immediately life threatening or result in death or require hospitalization but may require medical or surgical intervention to prevent 1 of the other outcomes). Vital signs, clinical chemistry, hematology as assessed by the investigator were reported as AE.
Outcome measures
| Measure |
Prophylaxis Cohort: TAK-755 (Period 3)
n=48 Participants
Participants received TAK-755 SIN dose of IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
Prophylaxis Cohort: SoC (Periods 1 and 2)
n=2 Participants
Participants received SoC for 6 months in either Period 1 or Period 2.
|
Prophylaxis Cohort: TAK-755
n=47 Participants
Participants received a single IV infusion of 40 IU/kg TAK-755 ORT Q2W for 6 months in either Period 1 or Period 2. Thereafter participants received TAK-755 SIN dose of IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
On Demand Cohort II: SoC
n=4 Participants
Participants experiencing an acute TTP event who met all other inclusion criteria and entered the study through the SoC cohort of the Urgent Treatment Period received the investigator-recommended SoC and dosing regimen until the acute event was resolved. Upon resolution of the acute TTP event, participants had the option to either move to the prophylaxis cohort of the study or discontinue entirely.
|
|---|---|---|---|---|
|
Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Treatment Emergent Adverse Events (Serious TEAEs)
TEAES
|
44 Participants
|
0 Participants
|
42 Participants
|
3 Participants
|
|
Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Treatment Emergent Adverse Events (Serious TEAEs)
Serious TEAEs
|
8 Participants
|
0 Participants
|
6 Participants
|
1 Participants
|
SECONDARY outcome
Timeframe: Up to 79.6 monthsPopulation: The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. Overall number of participants analyzed is the number of participants with at least one assessment of the targeted parameter in the specified treatment.
Number of participants with inhibitory antibodies to ADAMTS13 were reported. As per planned analysis, data for this outcome measure were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the prophylaxis cohorts only.
Outcome measures
| Measure |
Prophylaxis Cohort: TAK-755 (Period 3)
n=4 Participants
Participants received TAK-755 SIN dose of IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
Prophylaxis Cohort: SoC (Periods 1 and 2)
Participants received SoC for 6 months in either Period 1 or Period 2.
|
Prophylaxis Cohort: TAK-755
n=6 Participants
Participants received a single IV infusion of 40 IU/kg TAK-755 ORT Q2W for 6 months in either Period 1 or Period 2. Thereafter participants received TAK-755 SIN dose of IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
On Demand Cohort II: SoC
Participants experiencing an acute TTP event who met all other inclusion criteria and entered the study through the SoC cohort of the Urgent Treatment Period received the investigator-recommended SoC and dosing regimen until the acute event was resolved. Upon resolution of the acute TTP event, participants had the option to either move to the prophylaxis cohort of the study or discontinue entirely.
|
|---|---|---|---|---|
|
Number of Participants With Inhibitory Antibodies to ADAMTS13
|
0 Participants
|
—
|
1 Participants
|
—
|
SECONDARY outcome
Timeframe: Up to 79.6 monthsPopulation: The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. Overall number of participants analyzed is the number of participants with at least one assessment of the targeted parameter in the specified treatment.
Total quantity of ADAMTS13 administered during the treatment of acute TTP events (all acute TTP events irrespective of central lab confirmation were included) was assessed. Acute TTP events typically require 3 to 4 days of intensified treatment. As per planned analysis, data for this outcome measure were collected and reported only for the TAK-755 treatment arm of both the prophylaxis (irrespective of the prophylaxis periods) and on demand cohorts.
Outcome measures
| Measure |
Prophylaxis Cohort: TAK-755 (Period 3)
n=2 Participants
Participants received TAK-755 SIN dose of IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
Prophylaxis Cohort: SoC (Periods 1 and 2)
Participants received SoC for 6 months in either Period 1 or Period 2.
|
Prophylaxis Cohort: TAK-755
Participants received a single IV infusion of 40 IU/kg TAK-755 ORT Q2W for 6 months in either Period 1 or Period 2. Thereafter participants received TAK-755 SIN dose of IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
On Demand Cohort II: SoC
Participants experiencing an acute TTP event who met all other inclusion criteria and entered the study through the SoC cohort of the Urgent Treatment Period received the investigator-recommended SoC and dosing regimen until the acute event was resolved. Upon resolution of the acute TTP event, participants had the option to either move to the prophylaxis cohort of the study or discontinue entirely.
|
|---|---|---|---|---|
|
Total Quantity of ADAMTS13 Administered During the Treatment of Acute TTP Events in Participants in TAK-755 Treatment Arm
|
5720.25 IU
Standard Deviation 189.858
|
—
|
—
|
—
|
SECONDARY outcome
Timeframe: PK-I (Month 1:Day 1 up to 12), PK-II (Month 12:Day 1 up to 12), and PK-III (Month 19:Day 1 up to 12): Pre-infusion and at multiple timepoints post-infusion up to 288 hoursPopulation: The PK Analysis Set included all FAS participants who had adequate post-dose PK measurements for at least one of the PK analytes without major protocol deviations or events that may affect the integrity of the PK data. Overall number of participants analyzed indicates the number of participants with data available for analyses. Number analyzed indicates the number of participants with data available for analyses in the specified category.
ADAMTS13 activity was measured by the fluorescent resonance energy transfer (FRETS) assay. IR was defined as body weight normalized maximum increase in plasma ADAMTS13 activity level. IR of ADAMTS13 activity for SoC agent and TAK-755 in plasma was assessed. (IU/mL)/(IU/kg) stands for (International units per milliliter)/(International units per kilogram). PK-I, PK-II, and PK-III denote the crossover PK evaluation of a maximum of 14 days at the start of Prophylaxis Treatment Period 1, end of Prophylaxis Treatment Periods 2 and 3 respectively. As per planned analysis, data for this outcome measure were collected and reported as per the treatment (intervention) received (rADAMTS13 manufactured in Orth, Austria \[TAK-755 ORT\], rADAMTS13 manufactured in Singapore \[TAK-755 SIN\], or SoC) during the course of the study, only for the prophylaxis cohorts. No participants received SoC in PK-II and PK-III thus there is no data for the same.
Outcome measures
| Measure |
Prophylaxis Cohort: TAK-755 (Period 3)
n=22 Participants
Participants received TAK-755 SIN dose of IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
Prophylaxis Cohort: SoC (Periods 1 and 2)
n=22 Participants
Participants received SoC for 6 months in either Period 1 or Period 2.
|
Prophylaxis Cohort: TAK-755
n=36 Participants
Participants received a single IV infusion of 40 IU/kg TAK-755 ORT Q2W for 6 months in either Period 1 or Period 2. Thereafter participants received TAK-755 SIN dose of IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
On Demand Cohort II: SoC
Participants experiencing an acute TTP event who met all other inclusion criteria and entered the study through the SoC cohort of the Urgent Treatment Period received the investigator-recommended SoC and dosing regimen until the acute event was resolved. Upon resolution of the acute TTP event, participants had the option to either move to the prophylaxis cohort of the study or discontinue entirely.
|
|---|---|---|---|---|
|
Incremental Recovery (IR) of ADAMTS13 Activity for SoC Agent and TAK-755 in Plasma During Prophylactic Treatment
PK-I: ADAMTS13 Activity
|
NA (IU/mL)/(IU/kg)
Standard Deviation NA
Mean and standard deviation (SD) were not estimable as the values were below the lower limit of quantification.
|
0.0212 (IU/mL)/(IU/kg)
Standard Deviation 0.0267
|
0.025 (IU/mL)/(IU/kg)
Standard Deviation 0.00592
|
—
|
|
Incremental Recovery (IR) of ADAMTS13 Activity for SoC Agent and TAK-755 in Plasma During Prophylactic Treatment
PK-II: ADAMTS13 Activity
|
0.0292 (IU/mL)/(IU/kg)
Standard Deviation 0.00630
|
—
|
0.0283 (IU/mL)/(IU/kg)
Standard Deviation 0.00644
|
—
|
|
Incremental Recovery (IR) of ADAMTS13 Activity for SoC Agent and TAK-755 in Plasma During Prophylactic Treatment
PK-III: ADAMTS13 Activity
|
0.0260 (IU/mL)/(IU/kg)
Standard Deviation 0.00617
|
—
|
—
|
—
|
SECONDARY outcome
Timeframe: PK-I (Month 1:Day 1 up to 12), PK-II (Month 12:Day 1 up to 12), and PK-III (Month 19:Day 1 up to 12): Pre-infusion and at multiple timepoints post-infusion up to 288 hoursPopulation: The PK Analysis Set included all FAS participants who had adequate post-dose PK measurements for at least one of the PK analytes without major protocol deviations or events that may affect the integrity of the PK data. Overall number of participants analyzed indicates the number of participants with data available for analyses. Number analyzed indicates the number of participants with data available for analyses in the specified category.
ADAMTS13 antigen was measured using a commercial ADAMTS13 enzyme-linked immunosorbent assay (ELISA) employing ADAMTS13 antigen. IR was defined as body weight normalized maximum increase in plasma ADAMTS13 antigen. IR of ADAMTS13 antigen for SoC agent and TAK-755 in plasma was assessed. (µg/mL)/ (µg/kg) stands for (microgram per milliliter)/(microgram per kilogram). PK-I, PK-II, and PK-III denote the crossover PK evaluation of a maximum of 14 days at the start of Prophylaxis Treatment Period 1, end of Prophylaxis Treatment Periods 2 and 3 respectively. As per planned analysis, data for this outcome measure were collected and reported as per the treatment (intervention) received (rADAMTS13 manufactured in Orth, Austria \[TAK-755 ORT\], rADAMTS13 manufactured in Singapore \[TAK-755 SIN\], or SoC) during the course of the study, only for the prophylaxis cohorts. No participants received SoC in PK-II and PK-III thus there is no data for the same.
Outcome measures
| Measure |
Prophylaxis Cohort: TAK-755 (Period 3)
n=24 Participants
Participants received TAK-755 SIN dose of IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
Prophylaxis Cohort: SoC (Periods 1 and 2)
n=23 Participants
Participants received SoC for 6 months in either Period 1 or Period 2.
|
Prophylaxis Cohort: TAK-755
n=37 Participants
Participants received a single IV infusion of 40 IU/kg TAK-755 ORT Q2W for 6 months in either Period 1 or Period 2. Thereafter participants received TAK-755 SIN dose of IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
On Demand Cohort II: SoC
Participants experiencing an acute TTP event who met all other inclusion criteria and entered the study through the SoC cohort of the Urgent Treatment Period received the investigator-recommended SoC and dosing regimen until the acute event was resolved. Upon resolution of the acute TTP event, participants had the option to either move to the prophylaxis cohort of the study or discontinue entirely.
|
|---|---|---|---|---|
|
IR of ADAMTS13 Antigen for SoC Agent and TAK-755 in Plasma During Prophylactic Treatment
PK-I: ADAMTS13 Antigen
|
NA (µg/mL)/ (µg/kg)
Standard Deviation NA
Mean and standard deviation (SD) were not estimable as the values were below the lower limit of quantification.
|
0.0186 (µg/mL)/ (µg/kg)
Standard Deviation 0.00605
|
0.0299 (µg/mL)/ (µg/kg)
Standard Deviation 0.00638
|
—
|
|
IR of ADAMTS13 Antigen for SoC Agent and TAK-755 in Plasma During Prophylactic Treatment
PK-II: ADAMTS13 Antigen
|
0.0324 (µg/mL)/ (µg/kg)
Standard Deviation 0.00668
|
—
|
0.0339 (µg/mL)/ (µg/kg)
Standard Deviation 0.0080
|
—
|
|
IR of ADAMTS13 Antigen for SoC Agent and TAK-755 in Plasma During Prophylactic Treatment
PK-III: ADAMTS13 Antigen
|
0.0264 (µg/mL)/ (µg/kg)
Standard Deviation 0.0102
|
—
|
—
|
—
|
SECONDARY outcome
Timeframe: PK-I (Month 1:Day 1 up to 12), PK-II (Month 12:Day 1 up to 12), and PK-III (Month 19:Day 1 up to 12): Pre-infusion and at multiple timepoints post-infusion up to 288 hoursPopulation: The PK Analysis Set included all FAS participants who had adequate post-dose PK measurements for at least one of the PK analytes without major protocol deviations or events that may affect the integrity of the PK data. Overall number of participants analyzed indicates the number of participants with data available for analyses. Number analyzed indicates the number of participants with data available for analyses in the specified category.
h\*IU/mL denotes for hours\*international units per milliliters. PK-I, PK-II, and PK-III denote the crossover PK evaluation of a maximum of 14 days at the start of Prophylaxis Treatment Period 1 and end of Prophylaxis Treatment Periods 2 and 3 respectively. As per planned analysis, data for this outcome measure were collected and reported as per the treatment (intervention) received (rADAMTS13 manufactured in Orth, Austria \[TAK-755 ORT\], rADAMTS13 manufactured in Singapore \[TAK-755 SIN\], or SoC) during the course of the study, only for the prophylaxis cohorts. No participants received SoC in PK-II and PK-III thus there is no data for the same.
Outcome measures
| Measure |
Prophylaxis Cohort: TAK-755 (Period 3)
n=22 Participants
Participants received TAK-755 SIN dose of IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
Prophylaxis Cohort: SoC (Periods 1 and 2)
n=31 Participants
Participants received SoC for 6 months in either Period 1 or Period 2.
|
Prophylaxis Cohort: TAK-755
n=33 Participants
Participants received a single IV infusion of 40 IU/kg TAK-755 ORT Q2W for 6 months in either Period 1 or Period 2. Thereafter participants received TAK-755 SIN dose of IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
On Demand Cohort II: SoC
Participants experiencing an acute TTP event who met all other inclusion criteria and entered the study through the SoC cohort of the Urgent Treatment Period received the investigator-recommended SoC and dosing regimen until the acute event was resolved. Upon resolution of the acute TTP event, participants had the option to either move to the prophylaxis cohort of the study or discontinue entirely.
|
|---|---|---|---|---|
|
Area Under the Plasma Curve [AUC]All of ADAMTS13 Activity for SoC Agent and TAK-755 in Plasma During Prophylactic Treatment
PK-II: ADAMTS13 Activity
|
52.98 h*IU/mL
Standard Deviation 13.358
|
—
|
52.83 h*IU/mL
Standard Deviation 11.940
|
—
|
|
Area Under the Plasma Curve [AUC]All of ADAMTS13 Activity for SoC Agent and TAK-755 in Plasma During Prophylactic Treatment
PK-I: ADAMTS13 Activity
|
—
|
10.56 h*IU/mL
Standard Deviation 8.263
|
44.15 h*IU/mL
Standard Deviation 11.197
|
—
|
|
Area Under the Plasma Curve [AUC]All of ADAMTS13 Activity for SoC Agent and TAK-755 in Plasma During Prophylactic Treatment
PK-III: ADAMTS13 Activity
|
65.65 h*IU/mL
Standard Deviation 23.487
|
—
|
—
|
—
|
SECONDARY outcome
Timeframe: PK-I (Month 1:Day 1 up to 12), PK-II (Month 12:Day 1 up to 12), and PK-III (Month 19:Day 1 up to 12): Pre-infusion and at multiple timepoints post-infusion up to 288 hoursPopulation: The PK Analysis Set included all FAS participants who had adequate post-dose PK measurements for at least one of the PK analytes without major protocol deviations or events that may affect the integrity of the PK data. Overall number of participants analyzed indicates the number of participants with data available for analyses. Number analyzed indicates the number of participants with data available for analyses in the specified category.
h\*µg/mL denotes for hours\*microgram per milliliters. PK-I, PK-II, and PK-III denote the crossover PK evaluation of a maximum of 14 days at the start of Prophylaxis Treatment Period 1 and end of Prophylaxis Treatment Periods 2 and 3 respectively. As per planned analysis, data for this outcome measure were collected and reported as per the treatment (intervention) received (rADAMTS13 manufactured in Orth, Austria \[TAK-755 ORT\], rADAMTS13 manufactured in Singapore \[TAK-755 SIN\], or SoC) during the course of the study, only for the prophylaxis cohorts. No participants received SoC in PK-II and PK-III thus there is no data for the same.
Outcome measures
| Measure |
Prophylaxis Cohort: TAK-755 (Period 3)
n=24 Participants
Participants received TAK-755 SIN dose of IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
Prophylaxis Cohort: SoC (Periods 1 and 2)
n=31 Participants
Participants received SoC for 6 months in either Period 1 or Period 2.
|
Prophylaxis Cohort: TAK-755
n=34 Participants
Participants received a single IV infusion of 40 IU/kg TAK-755 ORT Q2W for 6 months in either Period 1 or Period 2. Thereafter participants received TAK-755 SIN dose of IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
On Demand Cohort II: SoC
Participants experiencing an acute TTP event who met all other inclusion criteria and entered the study through the SoC cohort of the Urgent Treatment Period received the investigator-recommended SoC and dosing regimen until the acute event was resolved. Upon resolution of the acute TTP event, participants had the option to either move to the prophylaxis cohort of the study or discontinue entirely.
|
|---|---|---|---|---|
|
AUCall of ADAMTS13 Antigen for SoC Agent and TAK-755 in Plasma During Prophylactic Treatment
PK-II: ADAMTS13 Antigen
|
38.95 h*µg/mL
Standard Deviation 11.137
|
—
|
38.79 h*µg/mL
Standard Deviation 8.835
|
—
|
|
AUCall of ADAMTS13 Antigen for SoC Agent and TAK-755 in Plasma During Prophylactic Treatment
PK-I: ADAMTS13 Antigen
|
—
|
7.590 h*µg/mL
Standard Deviation 6.1344
|
34.12 h*µg/mL
Standard Deviation 9.646
|
—
|
|
AUCall of ADAMTS13 Antigen for SoC Agent and TAK-755 in Plasma During Prophylactic Treatment
PK-III: ADAMTS13 Antigen
|
49.74 h*µg/mL
Standard Deviation 19.147
|
—
|
—
|
—
|
SECONDARY outcome
Timeframe: PK-I (Month 1:Day 1 up to 12), PK-II (Month 12:Day 1 up to 12), and PK-III (Month 19:Day 1 up to 12): Pre-infusion and at multiple timepoints post-infusion up to 288 hoursPopulation: The PK Analysis Set included all FAS participants who had adequate post-dose PK measurements for at least one of the PK analytes without major protocol deviations or events that may affect the integrity of the PK data. Overall number of participants analyzed indicates the number of participants with data available for analyses. Number analyzed indicates the number of participants with data available for analyses in the specified category.
PK-I, PK-II, and PK-III denote the crossover PK evaluation of a maximum of 14 days at the start of Prophylaxis Treatment Period 1 and end of Prophylaxis Treatment Periods 2 and 3 respectively. As per planned analysis, data for this outcome measure were collected and reported as per the treatment (intervention) received (rADAMTS13 manufactured in Orth, Austria \[TAK-755 ORT\], rADAMTS13 manufactured in Singapore \[TAK-755 SIN\], or SoC) during the course of the study, only for the prophylaxis cohorts. No participants received SoC in PK-II and PK-III thus there is no data for the same.
Outcome measures
| Measure |
Prophylaxis Cohort: TAK-755 (Period 3)
n=24 Participants
Participants received TAK-755 SIN dose of IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
Prophylaxis Cohort: SoC (Periods 1 and 2)
n=22 Participants
Participants received SoC for 6 months in either Period 1 or Period 2.
|
Prophylaxis Cohort: TAK-755
n=34 Participants
Participants received a single IV infusion of 40 IU/kg TAK-755 ORT Q2W for 6 months in either Period 1 or Period 2. Thereafter participants received TAK-755 SIN dose of IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
On Demand Cohort II: SoC
Participants experiencing an acute TTP event who met all other inclusion criteria and entered the study through the SoC cohort of the Urgent Treatment Period received the investigator-recommended SoC and dosing regimen until the acute event was resolved. Upon resolution of the acute TTP event, participants had the option to either move to the prophylaxis cohort of the study or discontinue entirely.
|
|---|---|---|---|---|
|
Terminal Half-Life (t1/2) of ADAMTS13 Activity and ADAMTS13 Antigen for SoC Agent and TAK-755 in Plasma During Prophylactic Treatment
PK-I: ADAMTS13 Activity
|
—
|
62.88 hours
Standard Deviation 28.927
|
47.14 hours
Standard Deviation 11.573
|
—
|
|
Terminal Half-Life (t1/2) of ADAMTS13 Activity and ADAMTS13 Antigen for SoC Agent and TAK-755 in Plasma During Prophylactic Treatment
PK-I: ADAMTS13 Antigen
|
—
|
58.70 hours
Standard Deviation 23.575
|
53.59 hours
Standard Deviation 13.354
|
—
|
|
Terminal Half-Life (t1/2) of ADAMTS13 Activity and ADAMTS13 Antigen for SoC Agent and TAK-755 in Plasma During Prophylactic Treatment
PK-II: ADAMTS13 Activity
|
45.77 hours
Standard Deviation 10.231
|
—
|
52.51 hours
Standard Deviation 15.579
|
—
|
|
Terminal Half-Life (t1/2) of ADAMTS13 Activity and ADAMTS13 Antigen for SoC Agent and TAK-755 in Plasma During Prophylactic Treatment
PK-II: ADAMTS13 Antigen
|
49.72 hours
Standard Deviation 15.942
|
—
|
54.15 hours
Standard Deviation 16.553
|
—
|
|
Terminal Half-Life (t1/2) of ADAMTS13 Activity and ADAMTS13 Antigen for SoC Agent and TAK-755 in Plasma During Prophylactic Treatment
PK-III: ADAMTS13 Activity
|
35.38 hours
Standard Deviation 5.286
|
—
|
—
|
—
|
|
Terminal Half-Life (t1/2) of ADAMTS13 Activity and ADAMTS13 Antigen for SoC Agent and TAK-755 in Plasma During Prophylactic Treatment
PK-III: ADAMTS13 Antigen
|
39.85 hours
Standard Deviation 3.243
|
—
|
—
|
—
|
SECONDARY outcome
Timeframe: PK-I (Month 1:Day 1 up to 12), PK-II (Month 12:Day 1 up to 12), and PK-III (Month 19:Day 1 up to 12): Pre-infusion and at multiple timepoints post-infusion up to 288 hoursPopulation: The PK Analysis Set included all FAS participants who had adequate post-dose PK measurements for at least one of the PK analytes without major protocol deviations or events that may affect the integrity of the PK data. Overall number of participants analyzed indicates the number of participants with data available for analyses. Number analyzed indicates the number of participants with data available for analyses in the specified category.
PK-I, PK-II, and PK-III denote the crossover PK evaluation of a maximum of 14 days at the start of Prophylaxis Treatment Period 1 and end of Prophylaxis Treatment Periods 2 and 3 respectively. As per planned analysis, data for this outcome measure were collected and reported as per the treatment (intervention) received (rADAMTS13 manufactured in Orth, Austria \[TAK-755 ORT\], rADAMTS13 manufactured in Singapore \[TAK-755 SIN\], or SoC) during the course of the study, only for the prophylaxis cohorts. No participants received SoC in PK-II and PK-III thus there is no data for the same.
Outcome measures
| Measure |
Prophylaxis Cohort: TAK-755 (Period 3)
n=23 Participants
Participants received TAK-755 SIN dose of IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
Prophylaxis Cohort: SoC (Periods 1 and 2)
n=2 Participants
Participants received SoC for 6 months in either Period 1 or Period 2.
|
Prophylaxis Cohort: TAK-755
n=31 Participants
Participants received a single IV infusion of 40 IU/kg TAK-755 ORT Q2W for 6 months in either Period 1 or Period 2. Thereafter participants received TAK-755 SIN dose of IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
On Demand Cohort II: SoC
Participants experiencing an acute TTP event who met all other inclusion criteria and entered the study through the SoC cohort of the Urgent Treatment Period received the investigator-recommended SoC and dosing regimen until the acute event was resolved. Upon resolution of the acute TTP event, participants had the option to either move to the prophylaxis cohort of the study or discontinue entirely.
|
|---|---|---|---|---|
|
Mean Residence Time Extrapolated to Infinity (MRT0-inf) of ADAMTS13 Activity and ADAMTS13 Antigen for SoC Agent and TAK-755 in Plasma During Prophylactic Treatment
PK-I: ADAMTS13 Antigen
|
—
|
NA hours
Standard Deviation NA
The mean and SD were not estimable as the values were below the lower limit of quantification.
|
71.20 hours
Standard Deviation 16.538
|
—
|
|
Mean Residence Time Extrapolated to Infinity (MRT0-inf) of ADAMTS13 Activity and ADAMTS13 Antigen for SoC Agent and TAK-755 in Plasma During Prophylactic Treatment
PK-I: ADAMTS13 Activity
|
—
|
NA hours
Standard Deviation NA
The mean and SD were not estimable as the values were below the lower limit of quantification.
|
64.35 hours
Standard Deviation 17.498
|
—
|
|
Mean Residence Time Extrapolated to Infinity (MRT0-inf) of ADAMTS13 Activity and ADAMTS13 Antigen for SoC Agent and TAK-755 in Plasma During Prophylactic Treatment
PK-II: ADAMTS13 Activity
|
61.56 hours
Standard Deviation 11.762
|
—
|
65.89 hours
Standard Deviation 13.472
|
—
|
|
Mean Residence Time Extrapolated to Infinity (MRT0-inf) of ADAMTS13 Activity and ADAMTS13 Antigen for SoC Agent and TAK-755 in Plasma During Prophylactic Treatment
PK-II: ADAMTS13 Antigen
|
66.30 hours
Standard Deviation 18.738
|
—
|
72.36 hours
Standard Deviation 19.224
|
—
|
|
Mean Residence Time Extrapolated to Infinity (MRT0-inf) of ADAMTS13 Activity and ADAMTS13 Antigen for SoC Agent and TAK-755 in Plasma During Prophylactic Treatment
PK-III: ADAMTS13 Activity
|
41.67 hours
Standard Deviation 6.171
|
—
|
—
|
—
|
|
Mean Residence Time Extrapolated to Infinity (MRT0-inf) of ADAMTS13 Activity and ADAMTS13 Antigen for SoC Agent and TAK-755 in Plasma During Prophylactic Treatment
PK-III: ADAMTS13 Antigen
|
46.30 hours
Standard Deviation 4.266
|
—
|
—
|
—
|
SECONDARY outcome
Timeframe: PK-I (Month 1:Day 1 up to 12), PK-II (Month 12:Day 1 up to 12), and PK-III (Month 19:Day 1 up to 12): Pre-infusion and at multiple timepoints post-infusion up to 288 hoursPopulation: The PK Analysis Set included all FAS participants who had adequate post-dose PK measurements for at least one of the PK analytes without major protocol deviations or events that may affect the integrity of the PK data. Overall number of participants analyzed indicates the number of participants with data available for analyses. Number analyzed indicates the number of participants with data available for analyses in the specified category.
PK-I, PK-II, and PK-III denote the crossover PK evaluation of a maximum of 14 days at the start of Prophylaxis Treatment Period 1 and end of Prophylaxis Treatment Periods 2 and 3 respectively. As per planned analysis, data for this outcome measure were collected and reported as per the treatment (intervention) received (rADAMTS13 manufactured in Orth, Austria \[TAK-755 ORT\], rADAMTS13 manufactured in Singapore \[TAK-755 SIN\], or SoC) during the course of the study, only for the prophylaxis cohorts. No participants received SoC in PK-II and PK-III thus there is no data for the same.
Outcome measures
| Measure |
Prophylaxis Cohort: TAK-755 (Period 3)
n=23 Participants
Participants received TAK-755 SIN dose of IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
Prophylaxis Cohort: SoC (Periods 1 and 2)
n=2 Participants
Participants received SoC for 6 months in either Period 1 or Period 2.
|
Prophylaxis Cohort: TAK-755
n=31 Participants
Participants received a single IV infusion of 40 IU/kg TAK-755 ORT Q2W for 6 months in either Period 1 or Period 2. Thereafter participants received TAK-755 SIN dose of IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
On Demand Cohort II: SoC
Participants experiencing an acute TTP event who met all other inclusion criteria and entered the study through the SoC cohort of the Urgent Treatment Period received the investigator-recommended SoC and dosing regimen until the acute event was resolved. Upon resolution of the acute TTP event, participants had the option to either move to the prophylaxis cohort of the study or discontinue entirely.
|
|---|---|---|---|---|
|
Clearance (CL) of ADAMTS13 Activity and ADAMTS13 Antigen for SoC Agent and TAK-755 in Plasma During Prophylactic Treatment
PK-I: ADAMTS13 Activity
|
—
|
NA liters per hour (L/h)
Standard Deviation NA
Mean and standard deviation (SD) were not estimable as the values were below the lower limit of quantification.
|
0.0618 liters per hour (L/h)
Standard Deviation 0.0144
|
—
|
|
Clearance (CL) of ADAMTS13 Activity and ADAMTS13 Antigen for SoC Agent and TAK-755 in Plasma During Prophylactic Treatment
PK-I: ADAMTS13 Antigen
|
—
|
NA liters per hour (L/h)
Standard Deviation NA
Mean and standard deviation (SD) were not estimable as the values were below the lower limit of quantification.
|
0.0456 liters per hour (L/h)
Standard Deviation 0.0117
|
—
|
|
Clearance (CL) of ADAMTS13 Activity and ADAMTS13 Antigen for SoC Agent and TAK-755 in Plasma During Prophylactic Treatment
PK-II: ADAMTS13 Activity
|
0.0549 liters per hour (L/h)
Standard Deviation 0.0117
|
—
|
0.0530 liters per hour (L/h)
Standard Deviation 0.0134
|
—
|
|
Clearance (CL) of ADAMTS13 Activity and ADAMTS13 Antigen for SoC Agent and TAK-755 in Plasma During Prophylactic Treatment
PK-II: ADAMTS13 Antigen
|
0.0480 liters per hour (L/h)
Standard Deviation 0.0133
|
—
|
0.0409 liters per hour (L/h)
Standard Deviation 0.0109
|
—
|
|
Clearance (CL) of ADAMTS13 Activity and ADAMTS13 Antigen for SoC Agent and TAK-755 in Plasma During Prophylactic Treatment
PK-III: ADAMTS13 Activity
|
0.0553 liters per hour (L/h)
Standard Deviation 0.0177
|
—
|
—
|
—
|
|
Clearance (CL) of ADAMTS13 Activity and ADAMTS13 Antigen for SoC Agent and TAK-755 in Plasma During Prophylactic Treatment
PK-III: ADAMTS13 Antigen
|
0.0462 liters per hour (L/h)
Standard Deviation 0.0110
|
—
|
—
|
—
|
SECONDARY outcome
Timeframe: PK-I (Month 1:Day 1 up to 12), PK-II (Month 12:Day 1 up to 12), and PK-III (Month 19:Day 1 up to 12): Pre-infusion and at multiple timepoints post-infusion up to 288 hoursPopulation: The PK Analysis Set included all FAS participants who had adequate post-dose PK measurements for at least one of the PK analytes without major protocol deviations or events that may affect the integrity of the PK data. Overall number of participants analyzed indicates the number of participants with data available for analyses. Number analyzed indicates the number of participants with data available for analyses in the specified category.
PK-I, PK-II, and PK-III denote the crossover PK evaluation of a maximum of 14 days at the start of Prophylaxis Treatment Period 1 and end of Prophylaxis Treatment Periods 2 and 3 respectively. As per planned analysis, data for this outcome measure were collected and reported as per the treatment (intervention) received (rADAMTS13 manufactured in Orth, Austria \[TAK-755 ORT\], rADAMTS13 manufactured in Singapore \[TAK-755 SIN\], or SoC) during the course of the study, only for the prophylaxis cohorts. No participants received SoC in PK-II and PK-III thus there is no data for the same.
Outcome measures
| Measure |
Prophylaxis Cohort: TAK-755 (Period 3)
n=23 Participants
Participants received TAK-755 SIN dose of IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
Prophylaxis Cohort: SoC (Periods 1 and 2)
n=2 Participants
Participants received SoC for 6 months in either Period 1 or Period 2.
|
Prophylaxis Cohort: TAK-755
n=31 Participants
Participants received a single IV infusion of 40 IU/kg TAK-755 ORT Q2W for 6 months in either Period 1 or Period 2. Thereafter participants received TAK-755 SIN dose of IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
On Demand Cohort II: SoC
Participants experiencing an acute TTP event who met all other inclusion criteria and entered the study through the SoC cohort of the Urgent Treatment Period received the investigator-recommended SoC and dosing regimen until the acute event was resolved. Upon resolution of the acute TTP event, participants had the option to either move to the prophylaxis cohort of the study or discontinue entirely.
|
|---|---|---|---|---|
|
Volume at Steady State (Vss) of ADAMTS13 Activity and ADAMTS13 Antigen for SoC Agent and TAK-755 in Plasma During Prophylactic Treatment
PK-II: ADAMTS13 Activity
|
3.304 liters
Standard Deviation 0.635
|
—
|
3.401 liters
Standard Deviation 0.715
|
—
|
|
Volume at Steady State (Vss) of ADAMTS13 Activity and ADAMTS13 Antigen for SoC Agent and TAK-755 in Plasma During Prophylactic Treatment
PK-II: ADAMTS13 Antigen
|
3.007 liters
Standard Deviation 0.613
|
—
|
2.812 liters
Standard Deviation 0.479
|
—
|
|
Volume at Steady State (Vss) of ADAMTS13 Activity and ADAMTS13 Antigen for SoC Agent and TAK-755 in Plasma During Prophylactic Treatment
PK-I: ADAMTS13 Activity
|
—
|
NA liters
Standard Deviation NA
The mean and SD were not estimable as the values were below the lower limit of quantification.
|
3.852 liters
Standard Deviation 0.916
|
—
|
|
Volume at Steady State (Vss) of ADAMTS13 Activity and ADAMTS13 Antigen for SoC Agent and TAK-755 in Plasma During Prophylactic Treatment
PK-I: ADAMTS13 Antigen
|
—
|
NA liters
Standard Deviation NA
The mean and SD were not estimable as the values were below the lower limit of quantification.
|
3.124 liters
Standard Deviation 0.650
|
—
|
|
Volume at Steady State (Vss) of ADAMTS13 Activity and ADAMTS13 Antigen for SoC Agent and TAK-755 in Plasma During Prophylactic Treatment
PK-III: ADAMTS13 Activity
|
2.265 liters
Standard Deviation 0.669
|
—
|
—
|
—
|
|
Volume at Steady State (Vss) of ADAMTS13 Activity and ADAMTS13 Antigen for SoC Agent and TAK-755 in Plasma During Prophylactic Treatment
PK-III: ADAMTS13 Antigen
|
2.172 liters
Standard Deviation 0.698
|
—
|
—
|
—
|
SECONDARY outcome
Timeframe: PK-I (Month 1:Day 1 up to 12), PK-II (Month 12:Day 1 up to 12), and PK-III (Month 19:Day 1 up to 12): Pre-infusion and at multiple timepoints post-infusion up to 288 hoursPopulation: The PK Analysis Set included all FAS participants who had adequate post-dose PK measurements for at least one of the PK analytes without major protocol deviations or events that may affect the integrity of the PK data. Overall number of participants analyzed indicates the number of participants with data available for analyses. Number analyzed indicates the number of participants with data available for analyses in the specified category.
IU/mL stands for International units per milliliter. PK-I, PK-II, and PK-III denote the crossover PK evaluation of a maximum of 14 days at the start of Prophylaxis Treatment Period 1 and end of Prophylaxis Treatment Periods 2 and 3 respectively. As per planned analysis, data for this outcome measure were collected and reported as per the treatment (intervention) received (rADAMTS13 manufactured in Orth,Austria \[TAK-755 ORT\], rADAMTS13 manufactured in Singapore \[TAK-755 SIN\], or SoC) during the course of the study, only for the prophylaxis cohorts. No participants received SoC in PK-II and PK-III thus there is no data for the same.
Outcome measures
| Measure |
Prophylaxis Cohort: TAK-755 (Period 3)
n=22 Participants
Participants received TAK-755 SIN dose of IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
Prophylaxis Cohort: SoC (Periods 1 and 2)
n=41 Participants
Participants received SoC for 6 months in either Period 1 or Period 2.
|
Prophylaxis Cohort: TAK-755
n=36 Participants
Participants received a single IV infusion of 40 IU/kg TAK-755 ORT Q2W for 6 months in either Period 1 or Period 2. Thereafter participants received TAK-755 SIN dose of IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
On Demand Cohort II: SoC
Participants experiencing an acute TTP event who met all other inclusion criteria and entered the study through the SoC cohort of the Urgent Treatment Period received the investigator-recommended SoC and dosing regimen until the acute event was resolved. Upon resolution of the acute TTP event, participants had the option to either move to the prophylaxis cohort of the study or discontinue entirely.
|
|---|---|---|---|---|
|
Maximum Concentration (Cmax) of ADAMTS13 Activity for SoC Agent and TAK-755 in Plasma During Prophylactic Treatment
PK-I: ADAMTS13 Activity
|
NA IU/mL
Standard Deviation NA
Mean and SD were not estimable as the values were below the lower limit of quantification.
|
0.192 IU/mL
Standard Deviation 0.102
|
1.003 IU/mL
Standard Deviation 0.235
|
—
|
|
Maximum Concentration (Cmax) of ADAMTS13 Activity for SoC Agent and TAK-755 in Plasma During Prophylactic Treatment
PK-II: ADAMTS13 Activity
|
1.162 IU/mL
Standard Deviation 0.250
|
—
|
1.130 IU/mL
Standard Deviation 0.253
|
—
|
|
Maximum Concentration (Cmax) of ADAMTS13 Activity for SoC Agent and TAK-755 in Plasma During Prophylactic Treatment
PK-III: ADAMTS13 Activity
|
1.036 IU/mL
Standard Deviation 0.253
|
—
|
—
|
—
|
SECONDARY outcome
Timeframe: PK-I (Month 1:Day 1 up to 12), PK-II (Month 12:Day 1 up to 12), and PK-III (Month 19:Day 1 up to 12): Pre-infusion and at multiple timepoints post-infusion up to 288 hoursPopulation: The PK Analysis Set included all FAS participants who had adequate post-dose PK measurements for at least one of the PK analytes without major protocol deviations or events that may affect the integrity of the PK data. Overall number of participants analyzed indicates the number of participants with data available for analyses. Number analyzed indicates the number of participants with data available for analyses in the specified category.
µg/mL stands for microgram per milliliter. PK-I, PK-II, and PK-III denote the crossover PK evaluation of a maximum of 14 days at the start of Prophylaxis Treatment Period 1 and end of Prophylaxis Treatment Periods 2 and 3 respectively. As per planned analysis, data for this outcome measure were collected and reported as per the treatment (intervention) received (rADAMTS13 manufactured in Orth, Austria \[TAK-755 ORT\], rADAMTS13 manufactured in Singapore \[TAK-755 SIN\], or SoC) during the course of the study, only for the prophylaxis cohorts. No participants received SoC in PK-II and PK-III thus there is no data for the same.
Outcome measures
| Measure |
Prophylaxis Cohort: TAK-755 (Period 3)
n=24 Participants
Participants received TAK-755 SIN dose of IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
Prophylaxis Cohort: SoC (Periods 1 and 2)
n=41 Participants
Participants received SoC for 6 months in either Period 1 or Period 2.
|
Prophylaxis Cohort: TAK-755
n=37 Participants
Participants received a single IV infusion of 40 IU/kg TAK-755 ORT Q2W for 6 months in either Period 1 or Period 2. Thereafter participants received TAK-755 SIN dose of IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
On Demand Cohort II: SoC
Participants experiencing an acute TTP event who met all other inclusion criteria and entered the study through the SoC cohort of the Urgent Treatment Period received the investigator-recommended SoC and dosing regimen until the acute event was resolved. Upon resolution of the acute TTP event, participants had the option to either move to the prophylaxis cohort of the study or discontinue entirely.
|
|---|---|---|---|---|
|
Cmax of ADAMTS13 Antigen for SoC Agent and TAK-755 in Plasma During Prophylactic Treatment
PK-I: ADAMTS13 Antigen
|
NA µg/mL
Standard Deviation NA
Mean and SD were not estimable as the values were below the lower limit of quantification.
|
0.141 µg/mL
Standard Deviation 0.0710
|
0.713 µg/mL
Standard Deviation 0.145
|
—
|
|
Cmax of ADAMTS13 Antigen for SoC Agent and TAK-755 in Plasma During Prophylactic Treatment
PK-II: ADAMTS13 Antigen
|
0.844 µg/mL
Standard Deviation 0.168
|
—
|
0.804 µg/mL
Standard Deviation 0.185
|
—
|
|
Cmax of ADAMTS13 Antigen for SoC Agent and TAK-755 in Plasma During Prophylactic Treatment
PK-III: ADAMTS13 Antigen
|
0.715 µg/mL
Standard Deviation 0.203
|
—
|
—
|
—
|
SECONDARY outcome
Timeframe: PK-I (Month 1:Day 12), PK-II (Month 12:Day 12), and PK-III (Month 19:Day 12): Post-infusion at 288 hoursPopulation: The Pharmacodynamic (PD) Analysis Set included all FAS participants who had at least one valid data point for at least one of the PD outcome measures and had no major protocol deviations or events that may affect the integrity of the PD data. Overall number of participants analyzed indicates the number of participants with data available for analyses. Number analyzed indicates the number of participants with data available for analyses in the specified category.
VWF:Ag is a measure of total VWF protein and was assessed using a sandwich ELISA employing polyclonal anti-human-VWF antibodies. Assessments of VWF:Ag at baseline and following infusion of the SoC agent and TAK-755 treatment during the initial PK assessment were reported. PK-I, PK-II, and PK-III denote the crossover PK evaluation of a maximum of 14 days at the start of Prophylaxis Treatment Period 1 and end of Prophylaxis Treatment Periods 2 and 3 respectively. As per planned analysis, data for this outcome measure were collected and reported as per the treatment (intervention) received (rADAMTS13 manufactured in Orth, Austria \[TAK-755 ORT\], rADAMTS13 manufactured in Singapore \[TAK-755 SIN\], or SoC) during the course of the study, only for the prophylaxis cohorts. No participants received SoC in PK-II and PK-III thus there is no data for the same.
Outcome measures
| Measure |
Prophylaxis Cohort: TAK-755 (Period 3)
n=21 Participants
Participants received TAK-755 SIN dose of IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
Prophylaxis Cohort: SoC (Periods 1 and 2)
n=19 Participants
Participants received SoC for 6 months in either Period 1 or Period 2.
|
Prophylaxis Cohort: TAK-755
n=23 Participants
Participants received a single IV infusion of 40 IU/kg TAK-755 ORT Q2W for 6 months in either Period 1 or Period 2. Thereafter participants received TAK-755 SIN dose of IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
On Demand Cohort II: SoC
Participants experiencing an acute TTP event who met all other inclusion criteria and entered the study through the SoC cohort of the Urgent Treatment Period received the investigator-recommended SoC and dosing regimen until the acute event was resolved. Upon resolution of the acute TTP event, participants had the option to either move to the prophylaxis cohort of the study or discontinue entirely.
|
|---|---|---|---|---|
|
Change From Baseline in Assessment of Von Willebrand Factor:Antigen (VWF:Ag) During Prophylactic Treatment
VWF:Ag : PK-I
|
—
|
3.67 percentage of VWF:Ag
Standard Deviation 19.705
|
-0.11 percentage of VWF:Ag
Standard Deviation 17.064
|
—
|
|
Change From Baseline in Assessment of Von Willebrand Factor:Antigen (VWF:Ag) During Prophylactic Treatment
VWF:Ag : PK-II
|
-1.31 percentage of VWF:Ag
Standard Deviation 22.259
|
—
|
0.48 percentage of VWF:Ag
Standard Deviation 34.798
|
—
|
SECONDARY outcome
Timeframe: PK-I (Month 1:Day 12), PK-II (Month 12:Day 12), and PK-III (Month 19:Day 12): Post-infusion at 288 hoursPopulation: The PD Analysis Set included all FAS participants who had at least one valid data point for at least one of the PD outcome measures and had no major protocol deviations or events that may affect the integrity of the PD data. Overall number of participants analyzed indicates the number of participants with data available for analyses. Number analyzed indicates the number of participants with data available for analyses in the specified category.
VWF:RCo provides a measure of the ability of VWF to bind platelet glycoprotein Ib. Stabilized platelets are agglutinated in the presence of VWF and the antibiotic Ristocetin. Assessments of VWF:RCo at baseline and following infusion of the SoC agent and TAK-755 treatment during the initial PK assessment was reported. PK-I, PK-II, and PK-III denote the crossover PK evaluation of a maximum of 14 days at the start of Prophylaxis Treatment Period 1 and end of Prophylaxis Treatment Periods 2 and 3 respectively. As per planned analysis, data for this outcome measure were collected and reported as per the treatment (intervention) received (rADAMTS13 manufactured in Orth, Austria \[TAK-755 ORT\], rADAMTS13 manufactured in Singapore \[TAK-755 SIN\], or SoC) during the course of the study, only for the prophylaxis cohorts. No participants received SoC in PK-II and PK-III thus there is no data for the same.
Outcome measures
| Measure |
Prophylaxis Cohort: TAK-755 (Period 3)
n=21 Participants
Participants received TAK-755 SIN dose of IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
Prophylaxis Cohort: SoC (Periods 1 and 2)
n=24 Participants
Participants received SoC for 6 months in either Period 1 or Period 2.
|
Prophylaxis Cohort: TAK-755
n=23 Participants
Participants received a single IV infusion of 40 IU/kg TAK-755 ORT Q2W for 6 months in either Period 1 or Period 2. Thereafter participants received TAK-755 SIN dose of IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
On Demand Cohort II: SoC
Participants experiencing an acute TTP event who met all other inclusion criteria and entered the study through the SoC cohort of the Urgent Treatment Period received the investigator-recommended SoC and dosing regimen until the acute event was resolved. Upon resolution of the acute TTP event, participants had the option to either move to the prophylaxis cohort of the study or discontinue entirely.
|
|---|---|---|---|---|
|
Change From Baseline in Assessment of Von Willebrand Factor:Ristocetin Cofactor Activity (VWF:RCo) During Prophylactic Treatment
VWF:RCo- PK-I
|
—
|
9.99 percentage of VWF:RCo
Standard Deviation 33.230
|
3.63 percentage of VWF:RCo
Standard Deviation 42.178
|
—
|
|
Change From Baseline in Assessment of Von Willebrand Factor:Ristocetin Cofactor Activity (VWF:RCo) During Prophylactic Treatment
VWF:RCo- PK-II
|
3.60 percentage of VWF:RCo
Standard Deviation 30.678
|
—
|
7.45 percentage of VWF:RCo
Standard Deviation 31.917
|
—
|
SECONDARY outcome
Timeframe: PK-I (Month 1:Day 1), PK-II (Month 12:Day 1), and PK-III (Month 19:Day 1): Pre-infusion (within 1 hour)Population: The PD analysis set included all FAS participants who had at least one valid data point for at least one of the PD outcome measures and had no major protocol deviations or events that may affect the integrity of the PD data. Overall number of participants analyzed indicates the number of participants with data available for analyses. Number analyzed indicates the number of participants with data available for analyses in the specified category.
PK-I, PK-II, and PK-III denote the crossover PK evaluation of a maximum of 14 days at the start of Prophylaxis Treatment Period 1 and end of Prophylaxis Treatment Periods 2 and 3 respectively. As per planned analysis, data for this outcome measure were collected and reported as per the treatment (intervention) received (rADAMTS13 manufactured in Orth, Austria \[TAK-755 ORT\], rADAMTS13 manufactured in Singapore \[TAK-755 SIN\], or SoC) during the course of the study, only for the prophylaxis cohorts. No participants received SoC in PK-II and PK-III thus there is no data for the same.
Outcome measures
| Measure |
Prophylaxis Cohort: TAK-755 (Period 3)
n=22 Participants
Participants received TAK-755 SIN dose of IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
Prophylaxis Cohort: SoC (Periods 1 and 2)
n=44 Participants
Participants received SoC for 6 months in either Period 1 or Period 2.
|
Prophylaxis Cohort: TAK-755
n=36 Participants
Participants received a single IV infusion of 40 IU/kg TAK-755 ORT Q2W for 6 months in either Period 1 or Period 2. Thereafter participants received TAK-755 SIN dose of IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
On Demand Cohort II: SoC
Participants experiencing an acute TTP event who met all other inclusion criteria and entered the study through the SoC cohort of the Urgent Treatment Period received the investigator-recommended SoC and dosing regimen until the acute event was resolved. Upon resolution of the acute TTP event, participants had the option to either move to the prophylaxis cohort of the study or discontinue entirely.
|
|---|---|---|---|---|
|
Assessment of ADAMTS13 Activity Expressed as Pre-Infusion ADAMTS13 Levels
ADAMTS13 Activity: PK-I
|
NA IU/mL
Standard Deviation NA
Mean and SD were not estimable as the values were below the lower limit of quantification.
|
NA IU/mL
Standard Deviation NA
Mean and SD were not estimable as the values were below the lower limit of quantification.
|
NA IU/mL
Standard Deviation NA
Mean and SD were not estimable as the values were below the lower limit of quantification.
|
—
|
|
Assessment of ADAMTS13 Activity Expressed as Pre-Infusion ADAMTS13 Levels
ADAMTS13 Activity: PK-II
|
NA IU/mL
Standard Deviation NA
Mean and SD were not estimable as the values were below the lower limit of quantification.
|
—
|
NA IU/mL
Standard Deviation NA
Mean and SD were not estimable as the values were below the lower limit of quantification.
|
—
|
|
Assessment of ADAMTS13 Activity Expressed as Pre-Infusion ADAMTS13 Levels
ADAMTS13 Activity: PK-III
|
NA IU/mL
Standard Deviation NA
Mean and SD were not estimable as the values were below the lower limit of quantification.
|
—
|
—
|
—
|
SECONDARY outcome
Timeframe: PK-I (Month 1:Day 1), PK-II (Month 12:Day 1), and PK-III (Month 19:Day 1): Pre-infusion (within 1 hour)Population: The PD analysis set included all FAS participants who had at least one valid data point for at least one of the PD outcome measures and had no major protocol deviations or events that may affect the integrity of the PD data. Overall number of participants analyzed indicates the number of participants with data available for analyses. Number analyzed indicates the number of participants with data available for analyses in the specified category.
PK-I, PK-II, and PK-III denote the crossover PK evaluation of a maximum of 14 days at the start of Prophylaxis Treatment Period 1 and end of Prophylaxis Treatment Periods 2 and 3 respectively. As per planned analysis, data for this outcome measure were collected and reported as per the treatment (intervention) received (rADAMTS13 manufactured in Orth, Austria \[TAK-755 ORT\], rADAMTS13 manufactured in Singapore \[TAK-755 SIN\], or SoC) during the course of the study, only for the prophylaxis cohorts. No participants received SoC in PK-II and PK-III thus there is no data for the same.
Outcome measures
| Measure |
Prophylaxis Cohort: TAK-755 (Period 3)
n=25 Participants
Participants received TAK-755 SIN dose of IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
Prophylaxis Cohort: SoC (Periods 1 and 2)
n=43 Participants
Participants received SoC for 6 months in either Period 1 or Period 2.
|
Prophylaxis Cohort: TAK-755
n=36 Participants
Participants received a single IV infusion of 40 IU/kg TAK-755 ORT Q2W for 6 months in either Period 1 or Period 2. Thereafter participants received TAK-755 SIN dose of IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
On Demand Cohort II: SoC
Participants experiencing an acute TTP event who met all other inclusion criteria and entered the study through the SoC cohort of the Urgent Treatment Period received the investigator-recommended SoC and dosing regimen until the acute event was resolved. Upon resolution of the acute TTP event, participants had the option to either move to the prophylaxis cohort of the study or discontinue entirely.
|
|---|---|---|---|---|
|
Assessment of Select VWF Parameters Expressed as Pre-Infusion Levels of VWF:RCo
PK-I: VWF:RCo
|
137.62 percentage of VWF:RCo
Standard Deviation 52.978
|
148.46 percentage of VWF:RCo
Standard Deviation 51.415
|
145.54 percentage of VWF:RCo
Standard Deviation 54.698
|
—
|
|
Assessment of Select VWF Parameters Expressed as Pre-Infusion Levels of VWF:RCo
PK-II: VWF:RCo
|
154.98 percentage of VWF:RCo
Standard Deviation 74.444
|
—
|
155.76 percentage of VWF:RCo
Standard Deviation 63.032
|
—
|
SECONDARY outcome
Timeframe: PK-I (Month 1:Day 1), PK-II (Month 12:Day 1), and PK-III (Month 19:Day 1): Pre-infusion (within 1 hour)Population: The PD analysis set included all FAS participants who had at least one valid data point for at least one of the PD outcome measures and had no major protocol deviations or events that may affect the integrity of the PD data. Overall number of participants analyzed indicates the number of participants with data available for analyses. Number analyzed indicates the number of participants with data available for analyses in the specified category.
PK-I, PK-II, and PK-III denote the crossover PK evaluation of a maximum of 14 days at the start of Prophylaxis Treatment Period 1 and end of Prophylaxis Treatment Periods 2 and 3 respectively. As per planned analysis, data for this outcome measure were collected and reported as per the treatment (intervention) received (rADAMTS13 manufactured in Orth, Austria \[TAK-755 ORT\], rADAMTS13 manufactured in Singapore \[TAK-755 SIN\], or SoC) during the course of the study, only for the prophylaxis cohorts. No participants received SoC in PK-II and PK-III thus there is no data for the same.
Outcome measures
| Measure |
Prophylaxis Cohort: TAK-755 (Period 3)
n=25 Participants
Participants received TAK-755 SIN dose of IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
Prophylaxis Cohort: SoC (Periods 1 and 2)
n=35 Participants
Participants received SoC for 6 months in either Period 1 or Period 2.
|
Prophylaxis Cohort: TAK-755
n=37 Participants
Participants received a single IV infusion of 40 IU/kg TAK-755 ORT Q2W for 6 months in either Period 1 or Period 2. Thereafter participants received TAK-755 SIN dose of IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
On Demand Cohort II: SoC
Participants experiencing an acute TTP event who met all other inclusion criteria and entered the study through the SoC cohort of the Urgent Treatment Period received the investigator-recommended SoC and dosing regimen until the acute event was resolved. Upon resolution of the acute TTP event, participants had the option to either move to the prophylaxis cohort of the study or discontinue entirely.
|
|---|---|---|---|---|
|
Assessment of Select VWF Parameters Expressed as Pre-Infusion Levels of VWF:Ag
PK-I: VWF:Ag
|
NA percentage of VWF:Ag
Standard Deviation NA
Mean and SD were not estimable as the values were below the lower limit of quantification.
|
105.07 percentage of VWF:Ag
Standard Deviation 40.539
|
110.38 percentage of VWF:Ag
Standard Deviation 45.205
|
—
|
|
Assessment of Select VWF Parameters Expressed as Pre-Infusion Levels of VWF:Ag
PK-II: VWF:Ag
|
116.66 percentage of VWF:Ag
Standard Deviation 60.338
|
—
|
120.48 percentage of VWF:Ag
Standard Deviation 49.224
|
—
|
SECONDARY outcome
Timeframe: PK-I (Month 1:Day 1), PK-II (Month 12:Day 1), and PK-III (Month 19:Day 1): Pre-infusion (within 1 hour)Population: The PD analysis set included all FAS participants who had at least one valid data point for at least one of the PD outcome measures and had no major protocol deviations or events that may affect the integrity of the PD data. Overall number of participants analyzed indicates the number of participants with data available for analyses. Number analyzed indicates the number of participants with data available for analyses in the specified category.
PK-I, PK-II, and PK-III denote the crossover PK evaluation of a maximum of 14 days at the start of Prophylaxis Treatment Period 1 and end of Prophylaxis Treatment Periods 2 and 3 respectively. As per planned analysis, data for this outcome measure were collected and reported as per the treatment (intervention) received (rADAMTS13 manufactured in Orth, Austria \[TAK-755 ORT\], rADAMTS13 manufactured in Singapore \[TAK-755 SIN\], or SoC) during the course of the study, only for the prophylaxis cohorts. No participants received SoC in PK-II and PK-III thus there is no data for the same.
Outcome measures
| Measure |
Prophylaxis Cohort: TAK-755 (Period 3)
n=25 Participants
Participants received TAK-755 SIN dose of IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
Prophylaxis Cohort: SoC (Periods 1 and 2)
n=39 Participants
Participants received SoC for 6 months in either Period 1 or Period 2.
|
Prophylaxis Cohort: TAK-755
n=38 Participants
Participants received a single IV infusion of 40 IU/kg TAK-755 ORT Q2W for 6 months in either Period 1 or Period 2. Thereafter participants received TAK-755 SIN dose of IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
On Demand Cohort II: SoC
Participants experiencing an acute TTP event who met all other inclusion criteria and entered the study through the SoC cohort of the Urgent Treatment Period received the investigator-recommended SoC and dosing regimen until the acute event was resolved. Upon resolution of the acute TTP event, participants had the option to either move to the prophylaxis cohort of the study or discontinue entirely.
|
|---|---|---|---|---|
|
Assessment of Select VWF Parameters Expressed as Pre-Infusion Levels of VWF:mm Low Resolution (Res.) Intermediate
PK-I: VWF:mm Low Res. Intermediate
|
NA % of VWF:mm Low Res.Intermediate
Standard Deviation NA
Mean and SD were not estimable as the values were below the lower limit of quantification.
|
31.22 % of VWF:mm Low Res.Intermediate
Standard Deviation 2.955
|
32.14 % of VWF:mm Low Res.Intermediate
Standard Deviation 3.375
|
—
|
|
Assessment of Select VWF Parameters Expressed as Pre-Infusion Levels of VWF:mm Low Resolution (Res.) Intermediate
PK-II: VWF:mm Low Res. Intermediate
|
31.10 % of VWF:mm Low Res.Intermediate
Standard Deviation 3.616
|
—
|
30.87 % of VWF:mm Low Res.Intermediate
Standard Deviation 3.754
|
—
|
SECONDARY outcome
Timeframe: PK-I (Month 1:Day 1), PK-II (Month 12:Day 1), and PK-III (Month 19:Day 1): Pre-infusion (within 1 hour)Population: The PD analysis set included all FAS participants who had at least one valid data point for at least one of the PD outcome measures and had no major protocol deviations or events that may affect the integrity of the PD data. Overall number of participants analyzed indicates the number of participants with data available for analyses. Number analyzed indicates the number of participants with data available for analyses in the specified category.
PK-I, PK-II, and PK-III denote the crossover PK evaluation of a maximum of 14 days at the start of Prophylaxis Treatment Period 1 and end of Prophylaxis Treatment Periods 2 and 3 respectively. As per planned analysis, data for this outcome measure were collected and reported as per the treatment (intervention) received (rADAMTS13 manufactured in Orth, Austria \[TAK-755 ORT\], rADAMTS13 manufactured in Singapore \[TAK-755 SIN\], or SoC) during the course of the study, only for the prophylaxis cohorts. No participants received SoC in PK-II and PK-III thus there is no data for the same.
Outcome measures
| Measure |
Prophylaxis Cohort: TAK-755 (Period 3)
n=25 Participants
Participants received TAK-755 SIN dose of IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
Prophylaxis Cohort: SoC (Periods 1 and 2)
n=39 Participants
Participants received SoC for 6 months in either Period 1 or Period 2.
|
Prophylaxis Cohort: TAK-755
n=38 Participants
Participants received a single IV infusion of 40 IU/kg TAK-755 ORT Q2W for 6 months in either Period 1 or Period 2. Thereafter participants received TAK-755 SIN dose of IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
On Demand Cohort II: SoC
Participants experiencing an acute TTP event who met all other inclusion criteria and entered the study through the SoC cohort of the Urgent Treatment Period received the investigator-recommended SoC and dosing regimen until the acute event was resolved. Upon resolution of the acute TTP event, participants had the option to either move to the prophylaxis cohort of the study or discontinue entirely.
|
|---|---|---|---|---|
|
Assessment of Select VWF Parameters Expressed as Pre-Infusion Levels of VWF:mm Low Res. Large
PK-II: VWF:mm Low Res. Large
|
44.93 % of VWF:mm Low Res. Large
Standard Deviation 7.319
|
—
|
46.17 % of VWF:mm Low Res. Large
Standard Deviation 5.670
|
—
|
|
Assessment of Select VWF Parameters Expressed as Pre-Infusion Levels of VWF:mm Low Res. Large
PK-I: VWF:mm Low Res. Large
|
NA % of VWF:mm Low Res. Large
Standard Deviation NA
Mean and SD were not estimable as the values were below the lower limit of quantification.
|
46.03 % of VWF:mm Low Res. Large
Standard Deviation 5.043
|
45.98 % of VWF:mm Low Res. Large
Standard Deviation 5.920
|
—
|
SECONDARY outcome
Timeframe: PK-I (Month 1:Day 1), PK-II (Month 12:Day 1), and PK-III (Month 19:Day 1): Pre-infusion (within 1 hour)Population: The PD analysis set included all FAS participants who had at least one valid data point for at least one of the PD outcome measures and had no major protocol deviations or events that may affect the integrity of the PD data. Overall number of participants analyzed indicates the number of participants with data available for analyses. Number analyzed indicates the number of participants with data available for analyses in the specified category.
PK-I, PK-II, and PK-III denote the crossover PK evaluation of a maximum of 14 days at the start of Prophylaxis Treatment Period 1 and end of Prophylaxis Treatment Periods 2 and 3 respectively. As per planned analysis, data for this outcome measure were collected and reported as per the treatment (intervention) received (rADAMTS13 manufactured in Orth, Austria \[TAK-755 ORT\], rADAMTS13 manufactured in Singapore \[TAK-755 SIN\], or SoC) during the course of the study, only for the prophylaxis cohorts. No participants received SoC in PK-II and PK-III thus there is no data for the same.
Outcome measures
| Measure |
Prophylaxis Cohort: TAK-755 (Period 3)
n=25 Participants
Participants received TAK-755 SIN dose of IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
Prophylaxis Cohort: SoC (Periods 1 and 2)
n=39 Participants
Participants received SoC for 6 months in either Period 1 or Period 2.
|
Prophylaxis Cohort: TAK-755
n=38 Participants
Participants received a single IV infusion of 40 IU/kg TAK-755 ORT Q2W for 6 months in either Period 1 or Period 2. Thereafter participants received TAK-755 SIN dose of IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
On Demand Cohort II: SoC
Participants experiencing an acute TTP event who met all other inclusion criteria and entered the study through the SoC cohort of the Urgent Treatment Period received the investigator-recommended SoC and dosing regimen until the acute event was resolved. Upon resolution of the acute TTP event, participants had the option to either move to the prophylaxis cohort of the study or discontinue entirely.
|
|---|---|---|---|---|
|
Assessment of Select VWF Parameters Expressed as Pre-Infusion Levels of VWF:mm Low Res. Small
PK-I: VWF:mm Low Res. Small
|
NA % of VWF:mm Low Res. Small
Standard Deviation NA
Mean and SD were not estimable as the values were below the lower limit of quantification.
|
22.77 % of VWF:mm Low Res. Small
Standard Deviation 5.161
|
21.66 % of VWF:mm Low Res. Small
Standard Deviation 4.230
|
—
|
|
Assessment of Select VWF Parameters Expressed as Pre-Infusion Levels of VWF:mm Low Res. Small
PK-II: VWF:mm Low Res. Small
|
23.96 % of VWF:mm Low Res. Small
Standard Deviation 5.320
|
—
|
22.96 % of VWF:mm Low Res. Small
Standard Deviation 3.047
|
—
|
SECONDARY outcome
Timeframe: Up to 79.6 monthsPopulation: The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. 2 participants randomized to Prophylaxis Cohort I received actual treatment as per Prophylaxis Cohort II and are presented per actual treatment arm.
Total binding antibodies to ADAMTS13 were measured by an ELISA-based assay, detecting total immunoglobulins (IgG, IgA, and IgM). As per planned analysis, data for this outcome measure were collected and reported per sequence (Prophylaxis Cohort I: TAK-755 Then SoC and Prophylaxis Cohort II: SoC Then TAK-755) for the prophylaxis cohorts only.
Outcome measures
| Measure |
Prophylaxis Cohort: TAK-755 (Period 3)
n=27 Participants
Participants received TAK-755 SIN dose of IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
Prophylaxis Cohort: SoC (Periods 1 and 2)
Participants received SoC for 6 months in either Period 1 or Period 2.
|
Prophylaxis Cohort: TAK-755
n=21 Participants
Participants received a single IV infusion of 40 IU/kg TAK-755 ORT Q2W for 6 months in either Period 1 or Period 2. Thereafter participants received TAK-755 SIN dose of IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
On Demand Cohort II: SoC
Participants experiencing an acute TTP event who met all other inclusion criteria and entered the study through the SoC cohort of the Urgent Treatment Period received the investigator-recommended SoC and dosing regimen until the acute event was resolved. Upon resolution of the acute TTP event, participants had the option to either move to the prophylaxis cohort of the study or discontinue entirely.
|
|---|---|---|---|---|
|
Number of Participants With Total Binding Antibodies to ADAMTS13 During Prophylactic Treatment
|
2 Participants
|
—
|
0 Participants
|
—
|
SECONDARY outcome
Timeframe: Up to 79.6 monthsPopulation: The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. 2 participants randomized to Prophylaxis Cohort I received actual treatment as per Prophylaxis Cohort II and are presented per actual treatment arm.
Neutralizing antibodies were measured by a Bethesda method with Nijmegen modification using the ADAMTS13 FRETS-VWF73 activity assay. As per planned analysis, data for this outcome measure were collected and reported per sequence (Prophylaxis Cohort I: TAK-755 Then SoC and Prophylaxis Cohort II: SoC Then TAK-755) for the prophylaxis cohorts only.
Outcome measures
| Measure |
Prophylaxis Cohort: TAK-755 (Period 3)
n=27 Participants
Participants received TAK-755 SIN dose of IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
Prophylaxis Cohort: SoC (Periods 1 and 2)
Participants received SoC for 6 months in either Period 1 or Period 2.
|
Prophylaxis Cohort: TAK-755
n=21 Participants
Participants received a single IV infusion of 40 IU/kg TAK-755 ORT Q2W for 6 months in either Period 1 or Period 2. Thereafter participants received TAK-755 SIN dose of IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
On Demand Cohort II: SoC
Participants experiencing an acute TTP event who met all other inclusion criteria and entered the study through the SoC cohort of the Urgent Treatment Period received the investigator-recommended SoC and dosing regimen until the acute event was resolved. Upon resolution of the acute TTP event, participants had the option to either move to the prophylaxis cohort of the study or discontinue entirely.
|
|---|---|---|---|---|
|
Number of Participants With Neutralizing Antibodies to ADAMTS13 During Prophylactic Treatment
|
1 Participants
|
—
|
0 Participants
|
—
|
SECONDARY outcome
Timeframe: Up to 79.6 monthsPopulation: The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. 2 participants randomized to Prophylaxis Cohort I received actual treatment as per Prophylaxis Cohort II and are presented per actual treatment arm.
Total immunoglobulin antibodies (Immunoglobulin G \[IgG\], A \[IgA\], and M \[IgM\]) against CHO protein were analyzed using ELISA assay. As per planned analysis, data for this outcome measure were collected and reported per sequence (Prophylaxis Cohort I: TAK-755 Then SoC and Prophylaxis Cohort II: SoC Then TAK-755) for the prophylaxis cohorts only.
Outcome measures
| Measure |
Prophylaxis Cohort: TAK-755 (Period 3)
n=27 Participants
Participants received TAK-755 SIN dose of IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
Prophylaxis Cohort: SoC (Periods 1 and 2)
Participants received SoC for 6 months in either Period 1 or Period 2.
|
Prophylaxis Cohort: TAK-755
n=21 Participants
Participants received a single IV infusion of 40 IU/kg TAK-755 ORT Q2W for 6 months in either Period 1 or Period 2. Thereafter participants received TAK-755 SIN dose of IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
On Demand Cohort II: SoC
Participants experiencing an acute TTP event who met all other inclusion criteria and entered the study through the SoC cohort of the Urgent Treatment Period received the investigator-recommended SoC and dosing regimen until the acute event was resolved. Upon resolution of the acute TTP event, participants had the option to either move to the prophylaxis cohort of the study or discontinue entirely.
|
|---|---|---|---|---|
|
Number of Participants With Anti-Chinese Hamster Ovary (Anti-CHO) Protein Antibodies During Prophylactic Treatment
|
2 Participants
|
—
|
0 Participants
|
—
|
SECONDARY outcome
Timeframe: Baseline, Urgent Treatment Period: Day 7, End of Period 1 (Month 6), End of Period 2 (Month 12), and End of Period 3 (Month 19)Population: Modified FAS. Overall number of participants analyzed are the number of participants with data available for analyses. Number analyzed is the number of participants with data available for analyses for specified category.
The cTTP-PEQ consists of 26 questions designed to assess the participant's experience of fatigue, joint, muscle, abdominal \&chest pain in the previous 24 hours, neurologic manifestations, bruising, feelings of depression and mood alterations, and activity limitation in the past 7 days, and participant's attitudes, experienced side effects, work/school absences and travel impact associated with treatment received for TTP during the previous 2 weeks. The cTTP PEQ is focused on measuring the symptoms and impacts of disease. The total scores range from 0 to 162. A higher score indicates greater burden and poor quality of life. As per planned analysis,for the prophylaxis cohorts the data for this outcome measure were collected and reported by categorizing as per Prophylaxis Periods and per age groups,≥12 years,12 to 18 years,≥18 years for both on demand(OD) and prophylaxis cohorts. No participants in the OD Cohorts had cTTP-PEQ data available for analysis at scheduled post-baseline visits.
Outcome measures
| Measure |
Prophylaxis Cohort: TAK-755 (Period 3)
n=12 Participants
Participants received TAK-755 SIN dose of IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
Prophylaxis Cohort: SoC (Periods 1 and 2)
Participants received SoC for 6 months in either Period 1 or Period 2.
|
Prophylaxis Cohort: TAK-755
n=22 Participants
Participants received a single IV infusion of 40 IU/kg TAK-755 ORT Q2W for 6 months in either Period 1 or Period 2. Thereafter participants received TAK-755 SIN dose of IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
On Demand Cohort II: SoC
Participants experiencing an acute TTP event who met all other inclusion criteria and entered the study through the SoC cohort of the Urgent Treatment Period received the investigator-recommended SoC and dosing regimen until the acute event was resolved. Upon resolution of the acute TTP event, participants had the option to either move to the prophylaxis cohort of the study or discontinue entirely.
|
|---|---|---|---|---|
|
Health Related Quality of Life (HRQoL) Assessed as Change From Baseline in cTTP-Patient Experience Questionnaire (cTTP-PEQ) Total Score
≥12 years: Total Score, End of Period 1
|
-2.2 score on a scale
Standard Deviation 12.47
|
—
|
-2.6 score on a scale
Standard Deviation 24.17
|
—
|
|
Health Related Quality of Life (HRQoL) Assessed as Change From Baseline in cTTP-Patient Experience Questionnaire (cTTP-PEQ) Total Score
≥12 years: Total Score, End of Period 2
|
-1.4 score on a scale
Standard Deviation 18.38
|
—
|
-10.4 score on a scale
Standard Deviation 18.13
|
—
|
|
Health Related Quality of Life (HRQoL) Assessed as Change From Baseline in cTTP-Patient Experience Questionnaire (cTTP-PEQ) Total Score
≥12 years: Total Score, End of Period 3
|
—
|
—
|
-10.6 score on a scale
Standard Deviation 13.13
|
—
|
|
Health Related Quality of Life (HRQoL) Assessed as Change From Baseline in cTTP-Patient Experience Questionnaire (cTTP-PEQ) Total Score
12 to < 18 years: Total Score, End of Period 1
|
12.0 score on a scale
Standard Deviation NA
SD was not estimable for a single participant.
|
—
|
—
|
—
|
|
Health Related Quality of Life (HRQoL) Assessed as Change From Baseline in cTTP-Patient Experience Questionnaire (cTTP-PEQ) Total Score
12 to < 18 years: Total Score, End of Period 2
|
—
|
—
|
13.0 score on a scale
Standard Deviation NA
SD was not estimable for a single participant.
|
—
|
|
Health Related Quality of Life (HRQoL) Assessed as Change From Baseline in cTTP-Patient Experience Questionnaire (cTTP-PEQ) Total Score
12 to < 18 years: Total Score, End of Period 3
|
—
|
—
|
-6.0 score on a scale
Standard Deviation NA
SD was not estimable for a single participant.
|
—
|
|
Health Related Quality of Life (HRQoL) Assessed as Change From Baseline in cTTP-Patient Experience Questionnaire (cTTP-PEQ) Total Score
≥18 years: Total Score, End of Period 1
|
-3.5 score on a scale
Standard Deviation 12.22
|
—
|
-2.6 score on a scale
Standard Deviation 24.17
|
—
|
|
Health Related Quality of Life (HRQoL) Assessed as Change From Baseline in cTTP-Patient Experience Questionnaire (cTTP-PEQ) Total Score
≥18 years: Total Score, End of Period 2
|
-1.4 score on a scale
Standard Deviation 18.38
|
—
|
-12.5 score on a scale
Standard Deviation 17.37
|
—
|
|
Health Related Quality of Life (HRQoL) Assessed as Change From Baseline in cTTP-Patient Experience Questionnaire (cTTP-PEQ) Total Score
≥18 years: Total Score, End of Period 3
|
—
|
—
|
-10.9 score on a scale
Standard Deviation 13.41
|
—
|
SECONDARY outcome
Timeframe: Baseline, Urgent Treatment Period: Day 7, End of Period 1 (Month 6), End of Period 2 (Month 12), and End of Period 3 (Month 19)Population: Modified FAS. Overall number of participants analyzed are the number of participants with data available for analyses. Number analyzed is the number of participants with data available for analyses for specified category. No participants in the OD Cohorts had SF-36v2 data available for analysis at scheduled post-baseline visits.
SF-36v2 is questionnaire that evaluated participant's health related quality of life. It included 36 questions related to 8 health dimensions: physical functioning, role-physical(role limitations due to physical health problems), bodily pain, general health, vitality(energy/fatigue),social functioning, role-emotional(role limitations due to emotional problems),\& mental health. Based on these 4 scales(physical functioning, role-physical, bodily pain, general health), physical component score was generated which ranges between 0 \&100, with higher scores indicating a better quality of life. Based on these 4 scales(vitality, social functioning, role-emotional,\&mental health), mental component score was generated ranging between 0\&100, with higher scores=better quality of life. As per planned analysis, for the prophylaxis cohorts data for this outcome measure were collected\&reported by categorizing as per Prophylaxis Periods and per component scores for both on demand\&prophylaxis cohorts.
Outcome measures
| Measure |
Prophylaxis Cohort: TAK-755 (Period 3)
n=11 Participants
Participants received TAK-755 SIN dose of IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
Prophylaxis Cohort: SoC (Periods 1 and 2)
Participants received SoC for 6 months in either Period 1 or Period 2.
|
Prophylaxis Cohort: TAK-755
n=21 Participants
Participants received a single IV infusion of 40 IU/kg TAK-755 ORT Q2W for 6 months in either Period 1 or Period 2. Thereafter participants received TAK-755 SIN dose of IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
On Demand Cohort II: SoC
Participants experiencing an acute TTP event who met all other inclusion criteria and entered the study through the SoC cohort of the Urgent Treatment Period received the investigator-recommended SoC and dosing regimen until the acute event was resolved. Upon resolution of the acute TTP event, participants had the option to either move to the prophylaxis cohort of the study or discontinue entirely.
|
|---|---|---|---|---|
|
Health Related Quality of Life (HRQoL) Assessed as Change From Baseline in Physical and Mental Component Scores of the 36-Item Short Form Health Survey Version 2 (SF-36v2)
Physical Component Score: End of Period 1
|
-0.532 score on a scale
Standard Deviation 4.8297
|
—
|
3.934 score on a scale
Standard Deviation 10.1098
|
—
|
|
Health Related Quality of Life (HRQoL) Assessed as Change From Baseline in Physical and Mental Component Scores of the 36-Item Short Form Health Survey Version 2 (SF-36v2)
Physical Component Score: End of Period 2
|
2.625 score on a scale
Standard Deviation 6.4219
|
—
|
3.121 score on a scale
Standard Deviation 6.3196
|
—
|
|
Health Related Quality of Life (HRQoL) Assessed as Change From Baseline in Physical and Mental Component Scores of the 36-Item Short Form Health Survey Version 2 (SF-36v2)
Physical Component Score: End of Period 3
|
—
|
—
|
1.019 score on a scale
Standard Deviation 4.8970
|
—
|
|
Health Related Quality of Life (HRQoL) Assessed as Change From Baseline in Physical and Mental Component Scores of the 36-Item Short Form Health Survey Version 2 (SF-36v2)
Mental Component Score: End of Period 1
|
5.418 score on a scale
Standard Deviation 5.0244
|
—
|
-7.263 score on a scale
Standard Deviation 7.4244
|
—
|
|
Health Related Quality of Life (HRQoL) Assessed as Change From Baseline in Physical and Mental Component Scores of the 36-Item Short Form Health Survey Version 2 (SF-36v2)
Mental Component Score: End of Period 2
|
-4.853 score on a scale
Standard Deviation 9.9302
|
—
|
2.880 score on a scale
Standard Deviation 5.2646
|
—
|
|
Health Related Quality of Life (HRQoL) Assessed as Change From Baseline in Physical and Mental Component Scores of the 36-Item Short Form Health Survey Version 2 (SF-36v2)
Mental Component Score: End of Period 3
|
—
|
—
|
3.852 score on a scale
Standard Deviation 7.4066
|
—
|
SECONDARY outcome
Timeframe: Baseline, Urgent Treatment Period: Day 7, End of Period 1 (Month 6), End of Period 2 (Month 12), and End of Period 3 (Month 19)Population: Modified FAS. Overall number of participants analyzed are the number of participants with data available for analyses. Number analyzed is the number of participants with data available for analyses for specified category. No participants in the OD Cohorts had TSQM-9 data available for analysis at scheduled post-baseline visits.
TSQM is a treatment satisfaction measure used to assess the overall level of participant's satisfaction or dissatisfaction with their medications. TSQM-9 is a 9-item, validated, self-administered instrument used to assess participant's satisfaction with medication. The three domains assessed are treatment effectiveness, convenience, and global satisfaction. The score of each of the 3 domains is based on an algorithm to create a score of 0 to 100. Higher score indicates greater satisfaction in that domain. As per planned analysis, for the prophylaxis cohorts data for this outcome measure were collected and reported by categorizing as per Prophylaxis Periods and per domain scores for both on demand and prophylaxis cohorts.
Outcome measures
| Measure |
Prophylaxis Cohort: TAK-755 (Period 3)
n=10 Participants
Participants received TAK-755 SIN dose of IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
Prophylaxis Cohort: SoC (Periods 1 and 2)
Participants received SoC for 6 months in either Period 1 or Period 2.
|
Prophylaxis Cohort: TAK-755
n=19 Participants
Participants received a single IV infusion of 40 IU/kg TAK-755 ORT Q2W for 6 months in either Period 1 or Period 2. Thereafter participants received TAK-755 SIN dose of IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
On Demand Cohort II: SoC
Participants experiencing an acute TTP event who met all other inclusion criteria and entered the study through the SoC cohort of the Urgent Treatment Period received the investigator-recommended SoC and dosing regimen until the acute event was resolved. Upon resolution of the acute TTP event, participants had the option to either move to the prophylaxis cohort of the study or discontinue entirely.
|
|---|---|---|---|---|
|
Health Related Quality of Life (HRQoL) Assessed as Change From Baseline in Abbreviated 9-item Treatment Satisfaction Questionnaire for Medication (TSQM-9) Domain Scores
Treatment Effectiveness Score: End of Period 1
|
4.4444 score on a scale
Standard Deviation 13.55778
|
—
|
26.8519 score on a scale
Standard Deviation 36.07548
|
—
|
|
Health Related Quality of Life (HRQoL) Assessed as Change From Baseline in Abbreviated 9-item Treatment Satisfaction Questionnaire for Medication (TSQM-9) Domain Scores
Treatment Effectiveness Score: End of Period 2
|
12.9630 score on a scale
Standard Deviation 17.09330
|
—
|
25.0000 score on a scale
Standard Deviation 17.42119
|
—
|
|
Health Related Quality of Life (HRQoL) Assessed as Change From Baseline in Abbreviated 9-item Treatment Satisfaction Questionnaire for Medication (TSQM-9) Domain Scores
Treatment Effectiveness Score: End of Period 3
|
—
|
—
|
22.2222 score on a scale
Standard Deviation 20.11626
|
—
|
|
Health Related Quality of Life (HRQoL) Assessed as Change From Baseline in Abbreviated 9-item Treatment Satisfaction Questionnaire for Medication (TSQM-9) Domain Scores
Convenience Score: End of Period 1
|
1.6667 score on a scale
Standard Deviation 11.43059
|
—
|
36.1111 score on a scale
Standard Deviation 16.75900
|
—
|
|
Health Related Quality of Life (HRQoL) Assessed as Change From Baseline in Abbreviated 9-item Treatment Satisfaction Questionnaire for Medication (TSQM-9) Domain Scores
Convenience Score: End of Period 2
|
14.8148 score on a scale
Standard Deviation 27.81479
|
—
|
27.7778 score on a scale
Standard Deviation 15.27076
|
—
|
|
Health Related Quality of Life (HRQoL) Assessed as Change From Baseline in Abbreviated 9-item Treatment Satisfaction Questionnaire for Medication (TSQM-9) Domain Scores
Convenience Score: End of Period 3
|
—
|
—
|
21.0526 score on a scale
Standard Deviation 29.48919
|
—
|
|
Health Related Quality of Life (HRQoL) Assessed as Change From Baseline in Abbreviated 9-item Treatment Satisfaction Questionnaire for Medication (TSQM-9) Domain Scores
Global Satisfaction Score: End of Period 1
|
5.0000 score on a scale
Standard Deviation 16.85270
|
—
|
28.5714 score on a scale
Standard Deviation 9.03508
|
—
|
|
Health Related Quality of Life (HRQoL) Assessed as Change From Baseline in Abbreviated 9-item Treatment Satisfaction Questionnaire for Medication (TSQM-9) Domain Scores
Global Satisfaction Score: End of Period 2
|
14.2857 score on a scale
Standard Deviation 21.66536
|
—
|
23.5714 score on a scale
Standard Deviation 16.85270
|
—
|
|
Health Related Quality of Life (HRQoL) Assessed as Change From Baseline in Abbreviated 9-item Treatment Satisfaction Questionnaire for Medication (TSQM-9) Domain Scores
Global Satisfaction Score: End of Period 3
|
—
|
—
|
22.9323 score on a scale
Standard Deviation 16.07433
|
—
|
SECONDARY outcome
Timeframe: Baseline, Urgent Treatment Period: Day 7, End of Period 1 (Month 6), End of Period 2 (Month 12), and End of Period 3 (Month 19)Population: Modified FAS. Overall number of participants analyzed are the number of participants with data available for analyses. Number analyzed is the number of participants with data available for analyses for specified category. No participants in the OD Cohorts had EQ-5D-3L data available for analysis at scheduled post-baseline visits.
EQ-5D-3L health questionnaire is a participant-answered questionnaire scoring 5 dimensions(domains) - mobility, self-care, usual activities, pain/discomfort, and anxiety/depression. Each dimension is scored on an ordinal scale with 3 available levels of response and scores ranging from 1 to 3, "no problems," "some problems," and "extreme problems," respectively. Lower scores for the domains in the EQ-5D-3L indicate improvement. As per planned analysis, for the prophylaxis cohorts data for this outcome measure were collected and reported by categorizing as per Prophylaxis Periods and per domain scores for both on demand and prophylaxis cohorts.
Outcome measures
| Measure |
Prophylaxis Cohort: TAK-755 (Period 3)
n=10 Participants
Participants received TAK-755 SIN dose of IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
Prophylaxis Cohort: SoC (Periods 1 and 2)
Participants received SoC for 6 months in either Period 1 or Period 2.
|
Prophylaxis Cohort: TAK-755
n=19 Participants
Participants received a single IV infusion of 40 IU/kg TAK-755 ORT Q2W for 6 months in either Period 1 or Period 2. Thereafter participants received TAK-755 SIN dose of IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
On Demand Cohort II: SoC
Participants experiencing an acute TTP event who met all other inclusion criteria and entered the study through the SoC cohort of the Urgent Treatment Period received the investigator-recommended SoC and dosing regimen until the acute event was resolved. Upon resolution of the acute TTP event, participants had the option to either move to the prophylaxis cohort of the study or discontinue entirely.
|
|---|---|---|---|---|
|
Health Related Quality of Life (HRQoL) Assessed as Change From Baseline in EuroQoL 5 Dimensions Questionnaire 3-Level (EQ-5D-3L) Domain Scores
Mobility: End of Period 1
|
-0.1 score on a scale
Standard Deviation 0.32
|
—
|
0.3 score on a scale
Standard Deviation 0.46
|
—
|
|
Health Related Quality of Life (HRQoL) Assessed as Change From Baseline in EuroQoL 5 Dimensions Questionnaire 3-Level (EQ-5D-3L) Domain Scores
Mobility: End of Period 2
|
0.0 score on a scale
Standard Deviation 0.00
|
—
|
-0.1 score on a scale
Standard Deviation 0.30
|
—
|
|
Health Related Quality of Life (HRQoL) Assessed as Change From Baseline in EuroQoL 5 Dimensions Questionnaire 3-Level (EQ-5D-3L) Domain Scores
Mobility: End of Period 3
|
—
|
—
|
-0.1 score on a scale
Standard Deviation 0.23
|
—
|
|
Health Related Quality of Life (HRQoL) Assessed as Change From Baseline in EuroQoL 5 Dimensions Questionnaire 3-Level (EQ-5D-3L) Domain Scores
Self-Care: End of Period 1
|
0.0 score on a scale
Standard Deviation 0.00
|
—
|
0.1 score on a scale
Standard Deviation 0.35
|
—
|
|
Health Related Quality of Life (HRQoL) Assessed as Change From Baseline in EuroQoL 5 Dimensions Questionnaire 3-Level (EQ-5D-3L) Domain Scores
Self-Care: End of Period 2
|
0.0 score on a scale
Standard Deviation 0.00
|
—
|
0.0 score on a scale
Standard Deviation 0.00
|
—
|
|
Health Related Quality of Life (HRQoL) Assessed as Change From Baseline in EuroQoL 5 Dimensions Questionnaire 3-Level (EQ-5D-3L) Domain Scores
Self-Care: End of Period 3
|
—
|
—
|
-0.1 score on a scale
Standard Deviation 0.23
|
—
|
|
Health Related Quality of Life (HRQoL) Assessed as Change From Baseline in EuroQoL 5 Dimensions Questionnaire 3-Level (EQ-5D-3L) Domain Scores
Usual Activities: End of Period 1
|
-0.2 score on a scale
Standard Deviation 0.42
|
—
|
0.0 score on a scale
Standard Deviation 0.53
|
—
|
|
Health Related Quality of Life (HRQoL) Assessed as Change From Baseline in EuroQoL 5 Dimensions Questionnaire 3-Level (EQ-5D-3L) Domain Scores
Usual Activities: End of Period 2
|
0.0 score on a scale
Standard Deviation 0.63
|
—
|
0.0 score on a scale
Standard Deviation 0.45
|
—
|
|
Health Related Quality of Life (HRQoL) Assessed as Change From Baseline in EuroQoL 5 Dimensions Questionnaire 3-Level (EQ-5D-3L) Domain Scores
Usual Activities: End of Period 3
|
—
|
—
|
-0.1 score on a scale
Standard Deviation 0.40
|
—
|
|
Health Related Quality of Life (HRQoL) Assessed as Change From Baseline in EuroQoL 5 Dimensions Questionnaire 3-Level (EQ-5D-3L) Domain Scores
Pain/Discomfort: End of Period 1
|
-0.2 score on a scale
Standard Deviation 0.63
|
—
|
0.3 score on a scale
Standard Deviation 0.46
|
—
|
|
Health Related Quality of Life (HRQoL) Assessed as Change From Baseline in EuroQoL 5 Dimensions Questionnaire 3-Level (EQ-5D-3L) Domain Scores
Pain/Discomfort: End of Period 2
|
0.2 score on a scale
Standard Deviation 0.41
|
—
|
-0.3 score on a scale
Standard Deviation 0.65
|
—
|
|
Health Related Quality of Life (HRQoL) Assessed as Change From Baseline in EuroQoL 5 Dimensions Questionnaire 3-Level (EQ-5D-3L) Domain Scores
Pain/Discomfort: End of Period 3
|
—
|
—
|
-0.2 score on a scale
Standard Deviation 0.54
|
—
|
|
Health Related Quality of Life (HRQoL) Assessed as Change From Baseline in EuroQoL 5 Dimensions Questionnaire 3-Level (EQ-5D-3L) Domain Scores
Anxiety/Depression: End of Period 1
|
0.0 score on a scale
Standard Deviation 0.00
|
—
|
0.1 score on a scale
Standard Deviation 0.64
|
—
|
|
Health Related Quality of Life (HRQoL) Assessed as Change From Baseline in EuroQoL 5 Dimensions Questionnaire 3-Level (EQ-5D-3L) Domain Scores
Anxiety/Depression: End of Period 2
|
0.0 score on a scale
Standard Deviation 0.00
|
—
|
-0.1 score on a scale
Standard Deviation 0.30
|
—
|
|
Health Related Quality of Life (HRQoL) Assessed as Change From Baseline in EuroQoL 5 Dimensions Questionnaire 3-Level (EQ-5D-3L) Domain Scores
Anxiety/Depression: End of Period 3
|
—
|
—
|
-0.1 score on a scale
Standard Deviation 0.46
|
—
|
SECONDARY outcome
Timeframe: Baseline, Urgent Treatment Period: Day 7, End of Period 1 (Month 6), End of Period 2 (Month 12), and End of Period 3 (Month 19)Population: Modified FAS. Overall number of participants analyzed are the number of participants with data available for analyses. Number analyzed is the number of participants with data available for analyses for specified category. No participants in the OD Cohorts had EQ-5D-Y data available for analysis at scheduled post-baseline visits.
EQ-5D-Y health questionnaire is a participant answered questionnaire scoring 5 dimensions (domains) - mobility, self-care, usual activities, pain/discomfort and anxiety/depression assessed in participants aged from 8 to 16 years. The EQ-5D-Y descriptive system includes 5 descriptive items: Mobility, self-care, doing usual activities, having pain or discomfort, and feeling anxiety or depressed. Each dimension is scored at 3 levels: 1=No problems, 2=some problems, and 3=a lot of problems. Lower scores for the domains in the EQ-5D-Y indicate improvement. As per planned analysis, for the prophylaxis cohorts data for this outcome measure were collected and reported by categorizing as per Prophylaxis Periods and per domain scores for both on demand and prophylaxis cohorts.
Outcome measures
| Measure |
Prophylaxis Cohort: TAK-755 (Period 3)
n=3 Participants
Participants received TAK-755 SIN dose of IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
Prophylaxis Cohort: SoC (Periods 1 and 2)
Participants received SoC for 6 months in either Period 1 or Period 2.
|
Prophylaxis Cohort: TAK-755
n=4 Participants
Participants received a single IV infusion of 40 IU/kg TAK-755 ORT Q2W for 6 months in either Period 1 or Period 2. Thereafter participants received TAK-755 SIN dose of IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
On Demand Cohort II: SoC
Participants experiencing an acute TTP event who met all other inclusion criteria and entered the study through the SoC cohort of the Urgent Treatment Period received the investigator-recommended SoC and dosing regimen until the acute event was resolved. Upon resolution of the acute TTP event, participants had the option to either move to the prophylaxis cohort of the study or discontinue entirely.
|
|---|---|---|---|---|
|
Health Related Quality of Life (HRQoL) Assessed as Change From Baseline in EQ-5D-youth (EQ-5D-Y) Domain Scores
Usual Activities: End of Period 2
|
0.0 score on a scale
Standard Deviation NA
SD was not estimable for a single participant.
|
—
|
0.0 score on a scale
Standard Deviation 0.00
|
—
|
|
Health Related Quality of Life (HRQoL) Assessed as Change From Baseline in EQ-5D-youth (EQ-5D-Y) Domain Scores
Mobility: End of Period 1
|
0.0 score on a scale
Standard Deviation 0.00
|
—
|
0.0 score on a scale
Standard Deviation NA
SD was not estimable for a single participant.
|
—
|
|
Health Related Quality of Life (HRQoL) Assessed as Change From Baseline in EQ-5D-youth (EQ-5D-Y) Domain Scores
Mobility: End of Period 2
|
0.0 score on a scale
Standard Deviation NA
SD was not estimable for a single participant.
|
—
|
0.0 score on a scale
Standard Deviation 0.00
|
—
|
|
Health Related Quality of Life (HRQoL) Assessed as Change From Baseline in EQ-5D-youth (EQ-5D-Y) Domain Scores
Mobility: End of Period 3
|
—
|
—
|
0.0 score on a scale
Standard Deviation 0.00
|
—
|
|
Health Related Quality of Life (HRQoL) Assessed as Change From Baseline in EQ-5D-youth (EQ-5D-Y) Domain Scores
Self-Care: End of Period 1
|
0.0 score on a scale
Standard Deviation 0.00
|
—
|
0.0 score on a scale
Standard Deviation NA
SD was not estimable for a single participant.
|
—
|
|
Health Related Quality of Life (HRQoL) Assessed as Change From Baseline in EQ-5D-youth (EQ-5D-Y) Domain Scores
Self-Care: End of Period 2
|
0.0 score on a scale
Standard Deviation NA
SD was not estimable for a single participant.
|
—
|
0.0 score on a scale
Standard Deviation 0.00
|
—
|
|
Health Related Quality of Life (HRQoL) Assessed as Change From Baseline in EQ-5D-youth (EQ-5D-Y) Domain Scores
Self-Care: End of Period 3
|
—
|
—
|
0.0 score on a scale
Standard Deviation 0.00
|
—
|
|
Health Related Quality of Life (HRQoL) Assessed as Change From Baseline in EQ-5D-youth (EQ-5D-Y) Domain Scores
Usual Activities: End of Period 1
|
0.0 score on a scale
Standard Deviation 0.00
|
—
|
0.0 score on a scale
Standard Deviation NA
SD was not estimable for a single participant.
|
—
|
|
Health Related Quality of Life (HRQoL) Assessed as Change From Baseline in EQ-5D-youth (EQ-5D-Y) Domain Scores
Usual Activities: End of Period 3
|
—
|
—
|
0.0 score on a scale
Standard Deviation 0.00
|
—
|
|
Health Related Quality of Life (HRQoL) Assessed as Change From Baseline in EQ-5D-youth (EQ-5D-Y) Domain Scores
Pain/Discomfort: End of Period 1
|
-0.7 score on a scale
Standard Deviation 0.58
|
—
|
1.0 score on a scale
Standard Deviation NA
SD was not estimable for a single participant.
|
—
|
|
Health Related Quality of Life (HRQoL) Assessed as Change From Baseline in EQ-5D-youth (EQ-5D-Y) Domain Scores
Pain/Discomfort: End of Period 2
|
0.0 score on a scale
Standard Deviation NA
SD was not estimable for a single participant.
|
—
|
-0.7 score on a scale
Standard Deviation 0.58
|
—
|
|
Health Related Quality of Life (HRQoL) Assessed as Change From Baseline in EQ-5D-youth (EQ-5D-Y) Domain Scores
Pain/Discomfort: End of Period 3
|
—
|
—
|
-0.3 score on a scale
Standard Deviation 0.96
|
—
|
|
Health Related Quality of Life (HRQoL) Assessed as Change From Baseline in EQ-5D-youth (EQ-5D-Y) Domain Scores
Anxiety/Depression: End of Period 1
|
0.0 score on a scale
Standard Deviation 0.00
|
—
|
0.0 score on a scale
Standard Deviation NA
SD was not estimable for a single participant.
|
—
|
|
Health Related Quality of Life (HRQoL) Assessed as Change From Baseline in EQ-5D-youth (EQ-5D-Y) Domain Scores
Anxiety/Depression: End of Period 2
|
0.0 score on a scale
Standard Deviation NA
SD was not estimable for a single participant.
|
—
|
0.3 score on a scale
Standard Deviation 0.58
|
—
|
|
Health Related Quality of Life (HRQoL) Assessed as Change From Baseline in EQ-5D-youth (EQ-5D-Y) Domain Scores
Anxiety/Depression: End of Period 3
|
—
|
—
|
0.0 score on a scale
Standard Deviation 0.00
|
—
|
SECONDARY outcome
Timeframe: Baseline, Urgent Treatment Period: Day 7, End of Period 1 (Month 6), End of Period 2 (Month 12), and End of Period 3 (Month 19)Population: Modified FAS. Overall number of participants analyzed are the number of participants with data available for analyses. Number analyzed is the number of participants with data available for analyses for specified category. No participants in the OD Cohorts had Peds QL data available for analysis at scheduled post-baseline visits.
The PedsQL is a generic health related quality of life instrument designed specifically for a pediatric population and captures following domains: physical functioning, emotional functioning, social functioning, school functioning, psychosocial summary, physical health and total score. The Peds-QL total score consists of all 23 items of all domains. This modular instrument uses a 5-point scale: from 0 (never) to 4 (almost always). Items are reversed scored and linearly transformed to a 0-100 scale as follows: 0=100, 1=75, 2=50, 3=25, 4=0. Higher scores indicate better quality of life. As per planned analysis, for the prophylaxis cohorts data for this outcome measure were collected and reported by categorizing as per Prophylaxis Periods and per age groups, 2 to \< 5 years, 5 to \< 8 years, 8 to \< 13 years, and 13 to \< 18 years, for both on demand and prophylaxis cohorts.
Outcome measures
| Measure |
Prophylaxis Cohort: TAK-755 (Period 3)
n=5 Participants
Participants received TAK-755 SIN dose of IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
Prophylaxis Cohort: SoC (Periods 1 and 2)
Participants received SoC for 6 months in either Period 1 or Period 2.
|
Prophylaxis Cohort: TAK-755
n=7 Participants
Participants received a single IV infusion of 40 IU/kg TAK-755 ORT Q2W for 6 months in either Period 1 or Period 2. Thereafter participants received TAK-755 SIN dose of IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
On Demand Cohort II: SoC
Participants experiencing an acute TTP event who met all other inclusion criteria and entered the study through the SoC cohort of the Urgent Treatment Period received the investigator-recommended SoC and dosing regimen until the acute event was resolved. Upon resolution of the acute TTP event, participants had the option to either move to the prophylaxis cohort of the study or discontinue entirely.
|
|---|---|---|---|---|
|
Health Related Quality of Life (HRQoL) Assessed as Change From Baseline in Pediatric Quality of Life Inventory (Peds QL) Scale Total Scores
5 to < 8 years, End of Period 1
|
-16.3043 score on a scale
Standard Deviation NA
SD was not estimable for a single participant.
|
—
|
29.3478 score on a scale
Standard Deviation NA
SD was not estimable for a single participant.
|
—
|
|
Health Related Quality of Life (HRQoL) Assessed as Change From Baseline in Pediatric Quality of Life Inventory (Peds QL) Scale Total Scores
5 to < 8 years, End of Period 2
|
32.6087 score on a scale
Standard Deviation NA
SD was not estimable for a single participant.
|
—
|
-6.5217 score on a scale
Standard Deviation NA
SD was not estimable for a single participant.
|
—
|
|
Health Related Quality of Life (HRQoL) Assessed as Change From Baseline in Pediatric Quality of Life Inventory (Peds QL) Scale Total Scores
5 to < 8 years, End of Period 3
|
—
|
—
|
17.9348 score on a scale
Standard Deviation 19.21486
|
—
|
|
Health Related Quality of Life (HRQoL) Assessed as Change From Baseline in Pediatric Quality of Life Inventory (Peds QL) Scale Total Scores
8 to < 13 years, End of Period 1
|
15.2174 score on a scale
Standard Deviation 13.83470
|
—
|
-2.1739 score on a scale
Standard Deviation NA
SD was not estimable for a single participant.
|
—
|
|
Health Related Quality of Life (HRQoL) Assessed as Change From Baseline in Pediatric Quality of Life Inventory (Peds QL) Scale Total Scores
8 to < 13 years, End of Period 2
|
1.0870 score on a scale
Standard Deviation NA
SD was not estimable for a single participant.
|
—
|
8.6957 score on a scale
Standard Deviation 23.05783
|
—
|
|
Health Related Quality of Life (HRQoL) Assessed as Change From Baseline in Pediatric Quality of Life Inventory (Peds QL) Scale Total Scores
8 to < 13 years, End of Period 3
|
—
|
—
|
5.7971 score on a scale
Standard Deviation 7.39876
|
—
|
|
Health Related Quality of Life (HRQoL) Assessed as Change From Baseline in Pediatric Quality of Life Inventory (Peds QL) Scale Total Scores
2 to < 5 years, End of Period 1
|
—
|
—
|
25.0000 score on a scale
Standard Deviation 47.14045
|
—
|
|
Health Related Quality of Life (HRQoL) Assessed as Change From Baseline in Pediatric Quality of Life Inventory (Peds QL) Scale Total Scores
2 to < 5 years, End of Period 2
|
27.3810 score on a scale
Standard Deviation 47.14045
|
—
|
—
|
—
|
|
Health Related Quality of Life (HRQoL) Assessed as Change From Baseline in Pediatric Quality of Life Inventory (Peds QL) Scale Total Scores
2 to < 5 years, End of Period 3
|
—
|
—
|
24.4048 score on a scale
Standard Deviation 49.66583
|
—
|
SECONDARY outcome
Timeframe: Up to 79.6 monthsPopulation: Modified FAS included all FAS participants who were treated according to their randomized treatment sequence with the exclusion of those enrolled prior to November 2017 who were treated with SoC instead of the randomized treatment of TAK-755 in period 1 because TAK-755 was not available. For participants enrolled prior to November 2017, only the first 6 months of SoC treatment in period 1 was included in the analysis.
The annualized number of days participants stayed in hospital for acute TTP events were assessed. As per planned analysis, data for this outcome measure were collected and reported only for the prophylaxis cohorts in a combined manner for Periods 1 and 2 for SoC treatment and for Periods 1, 2, and 3 for TAK-755 treatment respectively.
Outcome measures
| Measure |
Prophylaxis Cohort: TAK-755 (Period 3)
n=45 Participants
Participants received TAK-755 SIN dose of IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
Prophylaxis Cohort: SoC (Periods 1 and 2)
Participants received SoC for 6 months in either Period 1 or Period 2.
|
Prophylaxis Cohort: TAK-755
n=44 Participants
Participants received a single IV infusion of 40 IU/kg TAK-755 ORT Q2W for 6 months in either Period 1 or Period 2. Thereafter participants received TAK-755 SIN dose of IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
On Demand Cohort II: SoC
Participants experiencing an acute TTP event who met all other inclusion criteria and entered the study through the SoC cohort of the Urgent Treatment Period received the investigator-recommended SoC and dosing regimen until the acute event was resolved. Upon resolution of the acute TTP event, participants had the option to either move to the prophylaxis cohort of the study or discontinue entirely.
|
|---|---|---|---|---|
|
Resource Utilization: Annualized Length of Hospital Stay for Acute TTP Events for Prophylaxis Cohorts
|
0.00 days/year
Interval 0.0 to 3.4
|
—
|
0.00 days/year
Interval 0.0 to 0.0
|
—
|
SECONDARY outcome
Timeframe: Up to 79.6 monthsPopulation: Modified FAS included all FAS participants who were treated according to their randomized treatment sequence with the exclusion of those enrolled prior to November 2017 who were treated with SoC instead of the randomized treatment of TAK-755 in period 1 because TAK-755 was not available. For participants enrolled prior to November 2017, only the first 6 months of SoC treatment in period 1 was included in the analysis.
Annualized number of acute care visits was calculated as the number of acute care visits × 365.25/(End date - treatment start date + 1). As per planned analysis, data for this outcome measure were collected and reported only for the prophylaxis cohorts in a combined manner for Periods 1 and 2 for SoC treatment and for Periods 1, 2, and 3 for TAK-755 treatment respectively.
Outcome measures
| Measure |
Prophylaxis Cohort: TAK-755 (Period 3)
n=45 Participants
Participants received TAK-755 SIN dose of IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
Prophylaxis Cohort: SoC (Periods 1 and 2)
Participants received SoC for 6 months in either Period 1 or Period 2.
|
Prophylaxis Cohort: TAK-755
n=44 Participants
Participants received a single IV infusion of 40 IU/kg TAK-755 ORT Q2W for 6 months in either Period 1 or Period 2. Thereafter participants received TAK-755 SIN dose of IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
On Demand Cohort II: SoC
Participants experiencing an acute TTP event who met all other inclusion criteria and entered the study through the SoC cohort of the Urgent Treatment Period received the investigator-recommended SoC and dosing regimen until the acute event was resolved. Upon resolution of the acute TTP event, participants had the option to either move to the prophylaxis cohort of the study or discontinue entirely.
|
|---|---|---|---|---|
|
Resource Utilization: Annualized Number of Acute Care Visits for Prophylaxis Cohorts
|
0.14 acute care visits per year
Standard Deviation 0.498
|
—
|
0.60 acute care visits per year
Standard Deviation 1.331
|
—
|
SECONDARY outcome
Timeframe: Up to 79.6 monthsPopulation: Modified FAS included all FAS participants who were treated according to their randomized treatment sequence with the exclusion of those enrolled prior to November 2017 who were treated with SoC instead of the randomized treatment of TAK-755 in period 1 because TAK-755 was not available. For participants enrolled prior to November 2017, only the first 6 months of SoC treatment in period 1 was included in the analysis.
Annualized number of days missed from school or work were assessed. As per planned analysis, data for this outcome measure were collected and reported only for the prophylaxis cohorts in a combined manner for Periods 1 and 2 for SoC treatment and for Periods 1, 2, and 3 for TAK-755 treatment respectively.
Outcome measures
| Measure |
Prophylaxis Cohort: TAK-755 (Period 3)
n=45 Participants
Participants received TAK-755 SIN dose of IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
Prophylaxis Cohort: SoC (Periods 1 and 2)
Participants received SoC for 6 months in either Period 1 or Period 2.
|
Prophylaxis Cohort: TAK-755
n=44 Participants
Participants received a single IV infusion of 40 IU/kg TAK-755 ORT Q2W for 6 months in either Period 1 or Period 2. Thereafter participants received TAK-755 SIN dose of IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
On Demand Cohort II: SoC
Participants experiencing an acute TTP event who met all other inclusion criteria and entered the study through the SoC cohort of the Urgent Treatment Period received the investigator-recommended SoC and dosing regimen until the acute event was resolved. Upon resolution of the acute TTP event, participants had the option to either move to the prophylaxis cohort of the study or discontinue entirely.
|
|---|---|---|---|---|
|
Resource Utilization: Annualized Number of Days Missed From School or Work for Prophylaxis Cohorts
|
0.00 days/year
Interval 0.0 to 294.5
|
—
|
0.00 days/year
Interval 0.0 to 180.3
|
—
|
Adverse Events
Prophylaxis Cohort: TAK-755
Serious events: 6 serious events
Other events: 39 other events
Deaths: 0 deaths
Prophylaxis Cohort: SoC
Serious events: 8 serious events
Other events: 37 other events
Deaths: 0 deaths
On Demand Cohort I: TAK-755
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
On Demand Cohort II: SoC
Serious events: 1 serious events
Other events: 3 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Prophylaxis Cohort: TAK-755
n=47 participants at risk
Participants received a single IV infusion of 40 IU/kg TAK-755 ORT Q2W for 6 months in either Period 1 or Period 2. Thereafter participants received TAK-755 SIN dose of IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
Prophylaxis Cohort: SoC
n=48 participants at risk
Participants received SoC for 6 months in either Period 1 or Period 2.
|
On Demand Cohort I: TAK-755
n=2 participants at risk
Participants experiencing an acute TTP event who met all other inclusion criteria and entered the study through the TAK-755 cohort of the Urgent Treatment Period received initial dose of IV infusions 40 IU/kg \[+/- 4 IU/kg\] TAK-755 ORT or TAK-755 SIN infusion on Day 1 followed by a subsequent dose IV infusions of 20 IU/kg \[+/- 2 IU/kg\] TAK-755 ORT or TAK-755 SIN on Day 2 and an additional daily dose IV infusions of 15 IU/kg \[+/- 1.5 IU/kg\] TAK-755 on Day 3 until 2 days after the acute event was resolved. Upon resolution of the acute TTP event, participants had the option to either move to the prophylaxis cohort of the study or discontinue entirely.
|
On Demand Cohort II: SoC
n=4 participants at risk
Participants experiencing an acute TTP event who met all other inclusion criteria and entered the study through the SoC cohort of the Urgent Treatment Period received the investigator-recommended SoC and dosing regimen until the acute event was resolved. Upon resolution of the acute TTP event, participants had the option to either move to the prophylaxis cohort of the study or discontinue entirely.
|
|---|---|---|---|---|
|
Gastrointestinal disorders
Abdominal pain
|
2.1%
1/47 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/48 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/2 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/4 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
|
Reproductive system and breast disorders
Adnexal torsion
|
2.1%
1/47 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/48 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/2 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/4 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
|
Infections and infestations
Gastroenteritis clostridial
|
2.1%
1/47 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/48 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/2 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/4 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
|
Nervous system disorders
Headache
|
0.00%
0/47 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
2.1%
1/48 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/2 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/4 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
|
Endocrine disorders
Hyperthyroidism
|
2.1%
1/47 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/48 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/2 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/4 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
|
Reproductive system and breast disorders
Ovarian cyst
|
2.1%
1/47 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/48 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/2 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/4 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
|
Investigations
Platelet count decreased
|
0.00%
0/47 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
2.1%
1/48 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/2 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/4 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
|
Infections and infestations
Pneumonia
|
2.1%
1/47 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/48 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/2 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/4 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
|
General disorders
Pyrexia
|
0.00%
0/47 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
2.1%
1/48 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/2 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/4 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
|
Injury, poisoning and procedural complications
Road traffic accident
|
0.00%
0/47 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
2.1%
1/48 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/2 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/4 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
|
Immune system disorders
Seasonal allergy
|
0.00%
0/47 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
2.1%
1/48 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/2 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/4 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
|
Injury, poisoning and procedural complications
Shoulder fracture
|
0.00%
0/47 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
2.1%
1/48 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/2 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/4 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
|
Respiratory, thoracic and mediastinal disorders
Sinus disorder
|
0.00%
0/47 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
2.1%
1/48 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/2 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/4 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
|
Cardiac disorders
Tachycardia
|
2.1%
1/47 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/48 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/2 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/4 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
|
Blood and lymphatic system disorders
Thrombocytopenia
|
0.00%
0/47 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
4.2%
2/48 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/2 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
25.0%
1/4 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
|
Blood and lymphatic system disorders
Thrombotic thrombocytopenic purpura
|
2.1%
1/47 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/48 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/2 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/4 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
Other adverse events
| Measure |
Prophylaxis Cohort: TAK-755
n=47 participants at risk
Participants received a single IV infusion of 40 IU/kg TAK-755 ORT Q2W for 6 months in either Period 1 or Period 2. Thereafter participants received TAK-755 SIN dose of IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
|
Prophylaxis Cohort: SoC
n=48 participants at risk
Participants received SoC for 6 months in either Period 1 or Period 2.
|
On Demand Cohort I: TAK-755
n=2 participants at risk
Participants experiencing an acute TTP event who met all other inclusion criteria and entered the study through the TAK-755 cohort of the Urgent Treatment Period received initial dose of IV infusions 40 IU/kg \[+/- 4 IU/kg\] TAK-755 ORT or TAK-755 SIN infusion on Day 1 followed by a subsequent dose IV infusions of 20 IU/kg \[+/- 2 IU/kg\] TAK-755 ORT or TAK-755 SIN on Day 2 and an additional daily dose IV infusions of 15 IU/kg \[+/- 1.5 IU/kg\] TAK-755 on Day 3 until 2 days after the acute event was resolved. Upon resolution of the acute TTP event, participants had the option to either move to the prophylaxis cohort of the study or discontinue entirely.
|
On Demand Cohort II: SoC
n=4 participants at risk
Participants experiencing an acute TTP event who met all other inclusion criteria and entered the study through the SoC cohort of the Urgent Treatment Period received the investigator-recommended SoC and dosing regimen until the acute event was resolved. Upon resolution of the acute TTP event, participants had the option to either move to the prophylaxis cohort of the study or discontinue entirely.
|
|---|---|---|---|---|
|
Gastrointestinal disorders
Abdominal pain
|
17.0%
8/47 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
12.5%
6/48 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/2 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/4 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
|
Injury, poisoning and procedural complications
Allergic transfusion reaction
|
0.00%
0/47 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
14.6%
7/48 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/2 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
25.0%
1/4 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
|
Blood and lymphatic system disorders
Anaemia
|
6.4%
3/47 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
4.2%
2/48 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/2 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/4 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
6.4%
3/47 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
6.2%
3/48 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/2 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/4 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
|
General disorders
Asthenia
|
6.4%
3/47 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
2.1%
1/48 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/2 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/4 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
|
Investigations
Blood lactate dehydrogenase increased
|
8.5%
4/47 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
2.1%
1/48 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/2 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
25.0%
1/4 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
|
Infections and infestations
COVID-19
|
17.0%
8/47 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
6.2%
3/48 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/2 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/4 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
17.0%
8/47 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
6.2%
3/48 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/2 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/4 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
|
Gastrointestinal disorders
Diarrhoea
|
19.1%
9/47 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
4.2%
2/48 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/2 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/4 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
|
Nervous system disorders
Dizziness
|
10.6%
5/47 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/48 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/2 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/4 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
|
Ear and labyrinth disorders
Ear pain
|
6.4%
3/47 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/48 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/2 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/4 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
6.4%
3/47 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
8.3%
4/48 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/2 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/4 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
|
General disorders
Fatigue
|
10.6%
5/47 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
14.6%
7/48 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/2 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/4 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
|
Vascular disorders
Haematoma
|
6.4%
3/47 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
4.2%
2/48 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/2 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/4 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
|
Nervous system disorders
Headache
|
31.9%
15/47 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
20.8%
10/48 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/2 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
25.0%
1/4 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
|
Vascular disorders
Hypertension
|
6.4%
3/47 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/48 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/2 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/4 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
|
Nervous system disorders
Hypoaesthesia
|
8.5%
4/47 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/48 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/2 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
25.0%
1/4 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
|
Injury, poisoning and procedural complications
Immunisation reaction
|
6.4%
3/47 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
2.1%
1/48 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/2 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/4 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
|
Infections and infestations
Influenza
|
6.4%
3/47 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/48 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/2 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/4 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
|
Metabolism and nutrition disorders
Iron deficiency
|
6.4%
3/47 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
4.2%
2/48 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/2 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/4 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
|
Nervous system disorders
Lethargy
|
10.6%
5/47 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
6.2%
3/48 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/2 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/4 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
|
General disorders
Malaise
|
6.4%
3/47 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
4.2%
2/48 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/2 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/4 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
|
Nervous system disorders
Migraine
|
14.9%
7/47 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
4.2%
2/48 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/2 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/4 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
4.3%
2/47 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
6.2%
3/48 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/2 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/4 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
|
Infections and infestations
Nasopharyngitis
|
17.0%
8/47 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
12.5%
6/48 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/2 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/4 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
|
Gastrointestinal disorders
Nausea
|
17.0%
8/47 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
4.2%
2/48 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/2 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
25.0%
1/4 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
|
Infections and infestations
Oral herpes
|
6.4%
3/47 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
2.1%
1/48 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/2 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/4 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
10.6%
5/47 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
8.3%
4/48 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/2 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/4 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
|
Nervous system disorders
Paraesthesia
|
2.1%
1/47 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
4.2%
2/48 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/2 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
25.0%
1/4 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
|
Skin and subcutaneous tissue disorders
Petechiae
|
0.00%
0/47 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/48 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/2 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
25.0%
1/4 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
|
Infections and infestations
Pharyngitis
|
6.4%
3/47 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/48 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/2 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/4 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
|
Investigations
Platelet count decreased
|
8.5%
4/47 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
10.4%
5/48 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/2 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/4 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
4.3%
2/47 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
6.2%
3/48 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/2 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
25.0%
1/4 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
|
General disorders
Pyrexia
|
12.8%
6/47 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
4.2%
2/48 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/2 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/4 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
|
Skin and subcutaneous tissue disorders
Rash
|
4.3%
2/47 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
8.3%
4/48 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/2 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/4 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
|
Infections and infestations
Rhinitis
|
14.9%
7/47 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
4.2%
2/48 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/2 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/4 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhoea
|
10.6%
5/47 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/48 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/2 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/4 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
|
Immune system disorders
Seasonal allergy
|
6.4%
3/47 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
2.1%
1/48 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/2 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/4 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
|
Nervous system disorders
Syncope
|
8.5%
4/47 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
2.1%
1/48 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/2 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/4 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
|
Cardiac disorders
Tachycardia
|
2.1%
1/47 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
6.2%
3/48 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/2 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/4 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
|
Blood and lymphatic system disorders
Thrombocytopenia
|
6.4%
3/47 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
6.2%
3/48 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/2 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
25.0%
1/4 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
|
Infections and infestations
Tooth abscess
|
2.1%
1/47 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/48 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/2 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
25.0%
1/4 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
|
Infections and infestations
Upper respiratory tract infection
|
12.8%
6/47 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
6.2%
3/48 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/2 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/4 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
|
Skin and subcutaneous tissue disorders
Urticaria
|
4.3%
2/47 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
14.6%
7/48 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/2 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/4 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
|
Infections and infestations
Viral infection
|
8.5%
4/47 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
4.2%
2/48 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/2 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/4 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
|
Gastrointestinal disorders
Vomiting
|
17.0%
8/47 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
12.5%
6/48 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/2 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
0.00%
0/4 • From first dose of study drug up to end of study (79.6 months)
The Safety Analysis Set included all participants treated with at least 1 dose of TAK-755 or SoC treatment after randomization. As per planned analysis, data for adverse events were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the on demand and prophylaxis cohorts.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place