Trial Outcomes & Findings for An Open-label Safety and Efficacy Study of Recombinant FVIII in Patients With Severe Hemophilia A (NCT NCT02172950)
NCT ID: NCT02172950
Last Updated: 2021-10-27
Results Overview
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
246 participants
Primary outcome timeframe
At the closest visit after 100 EDs (up to 5 years).
Results posted on
2021-10-27
Participant Flow
Participant milestones
| Measure |
Previously Treated Patients (PTPs)
The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule for the subject based upon the subject's pharmacokinetic (PK) profile, rVIII-SingleChain PK data, previous FVIII treatment regimen, and bleeding phenotype, if available.
rVIII-SingleChain: Recombinant single-chain coagulation factor VIII
|
Previously Untreated Patients (PUPs)
The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule at their discretion, taking into consideration the World Federation of Hemophilia (WFH) guidelines, the type of bleeding episode, location of the bleeding, subject's age, and other disease characteristics.
rVIII-SingleChain: Recombinant single-chain coagulation factor VIII
|
|---|---|---|
|
Overall Study
STARTED
|
222
|
24
|
|
Overall Study
COMPLETED
|
197
|
19
|
|
Overall Study
NOT COMPLETED
|
25
|
5
|
Reasons for withdrawal
| Measure |
Previously Treated Patients (PTPs)
The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule for the subject based upon the subject's pharmacokinetic (PK) profile, rVIII-SingleChain PK data, previous FVIII treatment regimen, and bleeding phenotype, if available.
rVIII-SingleChain: Recombinant single-chain coagulation factor VIII
|
Previously Untreated Patients (PUPs)
The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule at their discretion, taking into consideration the World Federation of Hemophilia (WFH) guidelines, the type of bleeding episode, location of the bleeding, subject's age, and other disease characteristics.
rVIII-SingleChain: Recombinant single-chain coagulation factor VIII
|
|---|---|---|
|
Overall Study
Physician Decision
|
3
|
3
|
|
Overall Study
Lack of Efficacy
|
1
|
0
|
|
Overall Study
Adverse Event
|
4
|
1
|
|
Overall Study
Withdrawal by Subject
|
10
|
0
|
|
Overall Study
Protocol Violation
|
1
|
0
|
|
Overall Study
Lost to Follow-up
|
1
|
0
|
|
Overall Study
Death
|
1
|
0
|
|
Overall Study
Patient traveling
|
1
|
0
|
|
Overall Study
Patient went to other country
|
1
|
0
|
|
Overall Study
Patient relocated overseas
|
1
|
1
|
|
Overall Study
Patient moving
|
1
|
0
|
Baseline Characteristics
Age was analyzed separately as PTPs and PUPs
Baseline characteristics by cohort
| Measure |
Previously Treated Patients (PTPs)
n=222 Participants
The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule for the subject based upon the subject's pharmacokinetic (PK) profile, rVIII-SingleChain PK data, previous FVIII treatment regimen, and bleeding phenotype, if available.
rVIII-SingleChain: Recombinant single-chain coagulation factor VIII
|
Previously Untreated Patients (PUPs)
n=24 Participants
The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule at their discretion, taking into consideration the World Federation of Hemophilia (WFH) guidelines, the type of bleeding episode, location of the bleeding, subject's age, and other disease characteristics.
rVIII-SingleChain: Recombinant single-chain coagulation factor VIII
|
Total
n=246 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
95 Participants
n=222 Participants
|
24 Participants
n=24 Participants
|
119 Participants
n=246 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
126 Participants
n=222 Participants
|
0 Participants
n=24 Participants
|
126 Participants
n=246 Participants
|
|
Age, Categorical
>=65 years
|
1 Participants
n=222 Participants
|
0 Participants
n=24 Participants
|
1 Participants
n=246 Participants
|
|
Age, Continuous
PTPs
|
22.5 years
STANDARD_DEVIATION 14.55 • n=222 Participants • Age was analyzed separately as PTPs and PUPs
|
—
|
22.5 years
STANDARD_DEVIATION 14.55 • n=222 Participants • Age was analyzed separately as PTPs and PUPs
|
|
Age, Continuous
PUPs
|
—
|
1.4 years
STANDARD_DEVIATION 1.18 • n=24 Participants • Age was analyzed separately as PTPs and PUPs
|
1.4 years
STANDARD_DEVIATION 1.18 • n=24 Participants • Age was analyzed separately as PTPs and PUPs
|
|
Sex: Female, Male
Female
|
0 Participants
n=222 Participants
|
0 Participants
n=24 Participants
|
0 Participants
n=246 Participants
|
|
Sex: Female, Male
Male
|
222 Participants
n=222 Participants
|
24 Participants
n=24 Participants
|
246 Participants
n=246 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
10 Participants
n=222 Participants
|
1 Participants
n=24 Participants
|
11 Participants
n=246 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
212 Participants
n=222 Participants
|
23 Participants
n=24 Participants
|
235 Participants
n=246 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=222 Participants
|
0 Participants
n=24 Participants
|
0 Participants
n=246 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=222 Participants
|
0 Participants
n=24 Participants
|
0 Participants
n=246 Participants
|
|
Race (NIH/OMB)
Asian
|
50 Participants
n=222 Participants
|
2 Participants
n=24 Participants
|
52 Participants
n=246 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=222 Participants
|
0 Participants
n=24 Participants
|
0 Participants
n=246 Participants
|
|
Race (NIH/OMB)
White
|
158 Participants
n=222 Participants
|
15 Participants
n=24 Participants
|
173 Participants
n=246 Participants
|
|
Race (NIH/OMB)
Black or African American
|
12 Participants
n=222 Participants
|
7 Participants
n=24 Participants
|
19 Participants
n=246 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=222 Participants
|
0 Participants
n=24 Participants
|
0 Participants
n=246 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
2 Participants
n=222 Participants
|
0 Participants
n=24 Participants
|
2 Participants
n=246 Participants
|
|
Region of Enrollment
Czechia
|
2 participants
n=222 Participants
|
0 participants
n=24 Participants
|
2 participants
n=246 Participants
|
|
Region of Enrollment
United States
|
21 participants
n=222 Participants
|
1 participants
n=24 Participants
|
22 participants
n=246 Participants
|
|
Region of Enrollment
Malaysia
|
13 participants
n=222 Participants
|
2 participants
n=24 Participants
|
15 participants
n=246 Participants
|
|
Region of Enrollment
Thailand
|
10 participants
n=222 Participants
|
0 participants
n=24 Participants
|
10 participants
n=246 Participants
|
|
Region of Enrollment
Portugal
|
2 participants
n=222 Participants
|
3 participants
n=24 Participants
|
5 participants
n=246 Participants
|
|
Region of Enrollment
Austria
|
6 participants
n=222 Participants
|
0 participants
n=24 Participants
|
6 participants
n=246 Participants
|
|
Region of Enrollment
Netherlands
|
9 participants
n=222 Participants
|
3 participants
n=24 Participants
|
12 participants
n=246 Participants
|
|
Region of Enrollment
Ireland
|
5 participants
n=222 Participants
|
0 participants
n=24 Participants
|
5 participants
n=246 Participants
|
|
Region of Enrollment
Poland
|
36 participants
n=222 Participants
|
0 participants
n=24 Participants
|
36 participants
n=246 Participants
|
|
Region of Enrollment
France
|
7 participants
n=222 Participants
|
0 participants
n=24 Participants
|
7 participants
n=246 Participants
|
|
Region of Enrollment
Romania
|
4 participants
n=222 Participants
|
0 participants
n=24 Participants
|
4 participants
n=246 Participants
|
|
Region of Enrollment
Hungary
|
2 participants
n=222 Participants
|
0 participants
n=24 Participants
|
2 participants
n=246 Participants
|
|
Region of Enrollment
Japan
|
7 participants
n=222 Participants
|
0 participants
n=24 Participants
|
7 participants
n=246 Participants
|
|
Region of Enrollment
Philippines
|
18 participants
n=222 Participants
|
0 participants
n=24 Participants
|
18 participants
n=246 Participants
|
|
Region of Enrollment
Ukraine
|
17 participants
n=222 Participants
|
0 participants
n=24 Participants
|
17 participants
n=246 Participants
|
|
Region of Enrollment
United Kingdom
|
1 participants
n=222 Participants
|
0 participants
n=24 Participants
|
1 participants
n=246 Participants
|
|
Region of Enrollment
Switzerland
|
1 participants
n=222 Participants
|
0 participants
n=24 Participants
|
1 participants
n=246 Participants
|
|
Region of Enrollment
Spain
|
6 participants
n=222 Participants
|
0 participants
n=24 Participants
|
6 participants
n=246 Participants
|
|
Region of Enrollment
Lebanon
|
9 participants
n=222 Participants
|
3 participants
n=24 Participants
|
12 participants
n=246 Participants
|
|
Region of Enrollment
Canada
|
1 participants
n=222 Participants
|
0 participants
n=24 Participants
|
1 participants
n=246 Participants
|
|
Region of Enrollment
Italy
|
5 participants
n=222 Participants
|
4 participants
n=24 Participants
|
9 participants
n=246 Participants
|
|
Region of Enrollment
South Africa
|
14 participants
n=222 Participants
|
8 participants
n=24 Participants
|
22 participants
n=246 Participants
|
|
Region of Enrollment
Georgia
|
5 participants
n=222 Participants
|
0 participants
n=24 Participants
|
5 participants
n=246 Participants
|
|
Region of Enrollment
Australia
|
11 participants
n=222 Participants
|
0 participants
n=24 Participants
|
11 participants
n=246 Participants
|
|
Region of Enrollment
Germany
|
10 participants
n=222 Participants
|
0 participants
n=24 Participants
|
10 participants
n=246 Participants
|
PRIMARY outcome
Timeframe: At the closest visit after 100 EDs (up to 5 years).Population: Safety population (SP) - comprised all the Enrolled Population who received at least 1 dose of rVIII-SingleChain (CSL627) during the study for any reason.
Outcome measures
| Measure |
CSL627: Previously Treated Patients (PTPs)
n=198 Participants
The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule for the subject based upon the subject's pharmacokinetic (PK) profile, rVIII-SingleChain PK data, previous FVIII treatment regimen, and bleeding phenotype, if available.
rVIII-SingleChain: Recombinant single-chain coagulation factor VIII
|
CSL627: Previously Untreated Patients (PUPs)
The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule at their discretion, taking into consideration the World Federation of Hemophilia (WFH) guidelines, the type of bleeding episode, location of the bleeding, subject's age, and other disease characteristics.
rVIII-SingleChain: Recombinant single-chain coagulation factor VIII
|
|---|---|---|
|
Incidence of Inhibitor Formation to FVIII in Previously Treated Patients (PTPs) With 100 Exposure Days (EDs) to CSL627
|
0 percentage of paticipants
|
—
|
PRIMARY outcome
Timeframe: At the closest visit after 50 EDs (up to 5 years).Population: SP
High-titer inhibitor is defined as an inhibitor titer of ≥ 5 Bethesda units/mL.
Outcome measures
| Measure |
CSL627: Previously Treated Patients (PTPs)
n=24 Participants
The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule for the subject based upon the subject's pharmacokinetic (PK) profile, rVIII-SingleChain PK data, previous FVIII treatment regimen, and bleeding phenotype, if available.
rVIII-SingleChain: Recombinant single-chain coagulation factor VIII
|
CSL627: Previously Untreated Patients (PUPs)
The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule at their discretion, taking into consideration the World Federation of Hemophilia (WFH) guidelines, the type of bleeding episode, location of the bleeding, subject's age, and other disease characteristics.
rVIII-SingleChain: Recombinant single-chain coagulation factor VIII
|
|---|---|---|
|
Number of Previously Untreated Patients (PUPs) With High-titer Inhibitor Formation to FVIII With at Least 50 EDs to CSL627
|
5 participants
|
—
|
PRIMARY outcome
Timeframe: Up to 5 yearsPopulation: Efficacy population (EP) - comprised all subjects in the Safety Population who received at least 1 dose of rVIII-SingleChain (CSL627) for either routine prophylaxis treatment or on-demand treatment during the study. This endpoint was analyzed for PUPs only.
Percentage of major bleeding episodes treated successfully where treatment success for a bleeding episode is defined as a rating of "excellent" or "good" on the investigator's clinical assessment of hemostatic efficacy 4-point scale "excellent, good, moderate or poor/no response". Major bleeding episodes are defined as bleeding episodes for which a subject is required to seek treatment at the hemophilia center or that threatens the subject's life or loss of limb.
Outcome measures
| Measure |
CSL627: Previously Treated Patients (PTPs)
n=1 Major bleeds
The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule for the subject based upon the subject's pharmacokinetic (PK) profile, rVIII-SingleChain PK data, previous FVIII treatment regimen, and bleeding phenotype, if available.
rVIII-SingleChain: Recombinant single-chain coagulation factor VIII
|
CSL627: Previously Untreated Patients (PUPs)
The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule at their discretion, taking into consideration the World Federation of Hemophilia (WFH) guidelines, the type of bleeding episode, location of the bleeding, subject's age, and other disease characteristics.
rVIII-SingleChain: Recombinant single-chain coagulation factor VIII
|
|---|---|---|
|
Percent Treatment Success for Major Bleeding Episodes in PUPs
|
100 percentage of treatment success
|
—
|
PRIMARY outcome
Timeframe: Up to 5 yearsPopulation: EP. This endpoint was analyzed for PUPs only.
The annualized spontaneous bleeding rate for PUPs taking prophylaxis and on-demand treatment regimens.
Outcome measures
| Measure |
CSL627: Previously Treated Patients (PTPs)
n=23 Participants
The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule for the subject based upon the subject's pharmacokinetic (PK) profile, rVIII-SingleChain PK data, previous FVIII treatment regimen, and bleeding phenotype, if available.
rVIII-SingleChain: Recombinant single-chain coagulation factor VIII
|
CSL627: Previously Untreated Patients (PUPs)
The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule at their discretion, taking into consideration the World Federation of Hemophilia (WFH) guidelines, the type of bleeding episode, location of the bleeding, subject's age, and other disease characteristics.
rVIII-SingleChain: Recombinant single-chain coagulation factor VIII
|
|---|---|---|
|
Annualized Spontaneous Bleeding Rate in PUPs
On-demand
|
1.9 Number of Spontaneous bleeds per year
Standard Deviation 2.252
|
—
|
|
Annualized Spontaneous Bleeding Rate in PUPs
Prophylaxis
|
4.04 Number of Spontaneous bleeds per year
Standard Deviation 6.374
|
—
|
SECONDARY outcome
Timeframe: Up to 5 yearsPopulation: EP
Percentage of bleeding episodes treated successfully where treatment success for a bleeding episode is defined as a rating of "excellent" or "good" on the investigator's clinical assessment of hemostatic efficacy 4-point scale "excellent, good, moderate or poor/no response".
Outcome measures
| Measure |
CSL627: Previously Treated Patients (PTPs)
n=2413 Number of treated bleeding events
The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule for the subject based upon the subject's pharmacokinetic (PK) profile, rVIII-SingleChain PK data, previous FVIII treatment regimen, and bleeding phenotype, if available.
rVIII-SingleChain: Recombinant single-chain coagulation factor VIII
|
CSL627: Previously Untreated Patients (PUPs)
The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule at their discretion, taking into consideration the World Federation of Hemophilia (WFH) guidelines, the type of bleeding episode, location of the bleeding, subject's age, and other disease characteristics.
rVIII-SingleChain: Recombinant single-chain coagulation factor VIII
|
|---|---|---|
|
Percentage of Bleeding Episodes Treated Successfully in PTPs
|
87.1 Percentage of treated bleeding events
Interval 75.3 to 93.7
|
—
|
SECONDARY outcome
Timeframe: Up to 5 yearsPopulation: EP
The annualized bleeding rate for PTPs and PUPs taking prophylaxis and on-demand treatment regimens
Outcome measures
| Measure |
CSL627: Previously Treated Patients (PTPs)
n=222 Participants
The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule for the subject based upon the subject's pharmacokinetic (PK) profile, rVIII-SingleChain PK data, previous FVIII treatment regimen, and bleeding phenotype, if available.
rVIII-SingleChain: Recombinant single-chain coagulation factor VIII
|
CSL627: Previously Untreated Patients (PUPs)
n=23 Participants
The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule at their discretion, taking into consideration the World Federation of Hemophilia (WFH) guidelines, the type of bleeding episode, location of the bleeding, subject's age, and other disease characteristics.
rVIII-SingleChain: Recombinant single-chain coagulation factor VIII
|
|---|---|---|
|
Annualized Bleeding Rate in PTPs and PUPs
On-demand
|
28.32 Number of bleeds per year
Standard Deviation 29.014
|
5.12 Number of bleeds per year
Standard Deviation 5.331
|
|
Annualized Bleeding Rate in PTPs and PUPs
Prophylaxis
|
2.84 Number of bleeds per year
Standard Deviation 4.981
|
5.94 Number of bleeds per year
Standard Deviation 7.705
|
SECONDARY outcome
Timeframe: Up to 5 yearsPopulation: EP
Outcome measures
| Measure |
CSL627: Previously Treated Patients (PTPs)
n=2413 Treated bleeds
The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule for the subject based upon the subject's pharmacokinetic (PK) profile, rVIII-SingleChain PK data, previous FVIII treatment regimen, and bleeding phenotype, if available.
rVIII-SingleChain: Recombinant single-chain coagulation factor VIII
|
CSL627: Previously Untreated Patients (PUPs)
n=315 Treated bleeds
The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule at their discretion, taking into consideration the World Federation of Hemophilia (WFH) guidelines, the type of bleeding episode, location of the bleeding, subject's age, and other disease characteristics.
rVIII-SingleChain: Recombinant single-chain coagulation factor VIII
|
|---|---|---|
|
Percentage of Bleeding Episodes Requiring 1, 2, 3, or > 3 Injections of CSL627 to Achieve Hemostasis in PTPs and PUPs
1 infusion
|
71.5 Percentage of treated bleeds
|
77.5 Percentage of treated bleeds
|
|
Percentage of Bleeding Episodes Requiring 1, 2, 3, or > 3 Injections of CSL627 to Achieve Hemostasis in PTPs and PUPs
2 infusions
|
14.8 Percentage of treated bleeds
|
11.4 Percentage of treated bleeds
|
|
Percentage of Bleeding Episodes Requiring 1, 2, 3, or > 3 Injections of CSL627 to Achieve Hemostasis in PTPs and PUPs
3 infusions
|
6.9 Percentage of treated bleeds
|
5.4 Percentage of treated bleeds
|
|
Percentage of Bleeding Episodes Requiring 1, 2, 3, or > 3 Injections of CSL627 to Achieve Hemostasis in PTPs and PUPs
>3 infusions
|
6.3 Percentage of treated bleeds
|
3.5 Percentage of treated bleeds
|
SECONDARY outcome
Timeframe: Up to 5 yearsPopulation: EP. This endpoint analyzes PTPs and PUPs that received on-demand treatment.
Outcome measures
| Measure |
CSL627: Previously Treated Patients (PTPs)
n=11 Participants
The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule for the subject based upon the subject's pharmacokinetic (PK) profile, rVIII-SingleChain PK data, previous FVIII treatment regimen, and bleeding phenotype, if available.
rVIII-SingleChain: Recombinant single-chain coagulation factor VIII
|
CSL627: Previously Untreated Patients (PUPs)
n=12 Participants
The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule at their discretion, taking into consideration the World Federation of Hemophilia (WFH) guidelines, the type of bleeding episode, location of the bleeding, subject's age, and other disease characteristics.
rVIII-SingleChain: Recombinant single-chain coagulation factor VIII
|
|---|---|---|
|
Mean Number of On-demand Infusions of CSL627
per participant per month
|
6.26 Number of infusions
Standard Deviation 4.778
|
1.23 Number of infusions
Standard Deviation 1.296
|
|
Mean Number of On-demand Infusions of CSL627
per participant per year
|
75.18 Number of infusions
Standard Deviation 57.335
|
14.75 Number of infusions
Standard Deviation 15.547
|
SECONDARY outcome
Timeframe: Up to 5 yearsPopulation: EP. This endpoint analyzes PTPs and PUPs that received on-demand treatment.
Outcome measures
| Measure |
CSL627: Previously Treated Patients (PTPs)
n=11 Participants
The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule for the subject based upon the subject's pharmacokinetic (PK) profile, rVIII-SingleChain PK data, previous FVIII treatment regimen, and bleeding phenotype, if available.
rVIII-SingleChain: Recombinant single-chain coagulation factor VIII
|
CSL627: Previously Untreated Patients (PUPs)
n=12 Participants
The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule at their discretion, taking into consideration the World Federation of Hemophilia (WFH) guidelines, the type of bleeding episode, location of the bleeding, subject's age, and other disease characteristics.
rVIII-SingleChain: Recombinant single-chain coagulation factor VIII
|
|---|---|---|
|
Mean On-demand Dose Administered of CSL627
per participant per month
|
210.39 IU/kg
Standard Deviation 188.106
|
41.93 IU/kg
Standard Deviation 44.643
|
|
Mean On-demand Dose Administered of CSL627
per participant per year
|
2524.69 IU/kg
Standard Deviation 2257.278
|
503.16 IU/kg
Standard Deviation 535.712
|
SECONDARY outcome
Timeframe: Up to 5 yearsPopulation: EP. This endpoint analyzes PTPs and PUPs that received prophylaxis treatment.
Outcome measures
| Measure |
CSL627: Previously Treated Patients (PTPs)
n=211 Participants
The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule for the subject based upon the subject's pharmacokinetic (PK) profile, rVIII-SingleChain PK data, previous FVIII treatment regimen, and bleeding phenotype, if available.
rVIII-SingleChain: Recombinant single-chain coagulation factor VIII
|
CSL627: Previously Untreated Patients (PUPs)
n=23 Participants
The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule at their discretion, taking into consideration the World Federation of Hemophilia (WFH) guidelines, the type of bleeding episode, location of the bleeding, subject's age, and other disease characteristics.
rVIII-SingleChain: Recombinant single-chain coagulation factor VIII
|
|---|---|---|
|
Mean Prophylaxis Dose Administered of CSL627
per participant per month
|
380.95 IU/kg
Standard Deviation 130.079
|
389.30 IU/kg
Standard Deviation 243.191
|
|
Mean Prophylaxis Dose Administered of CSL627
per participant per year
|
4571.35 IU/kg
Standard Deviation 1560.944
|
4671.54 IU/kg
Standard Deviation 2918.288
|
SECONDARY outcome
Timeframe: Day of surgery up to 336 hours post-surgeryPopulation: Surgery population
Outcome measures
| Measure |
CSL627: Previously Treated Patients (PTPs)
n=24 Participants
The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule for the subject based upon the subject's pharmacokinetic (PK) profile, rVIII-SingleChain PK data, previous FVIII treatment regimen, and bleeding phenotype, if available.
rVIII-SingleChain: Recombinant single-chain coagulation factor VIII
|
CSL627: Previously Untreated Patients (PUPs)
The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule at their discretion, taking into consideration the World Federation of Hemophilia (WFH) guidelines, the type of bleeding episode, location of the bleeding, subject's age, and other disease characteristics.
rVIII-SingleChain: Recombinant single-chain coagulation factor VIII
|
|---|---|---|
|
Mean Total Amount of CSL627 Administered During Surgery Period in PTPs
|
51663.0 IU
Standard Deviation 62033.25
|
—
|
SECONDARY outcome
Timeframe: Day of surgery up to 336 hours post-surgeryPopulation: Surgery population
Outcome measures
| Measure |
CSL627: Previously Treated Patients (PTPs)
n=3 Participants
The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule for the subject based upon the subject's pharmacokinetic (PK) profile, rVIII-SingleChain PK data, previous FVIII treatment regimen, and bleeding phenotype, if available.
rVIII-SingleChain: Recombinant single-chain coagulation factor VIII
|
CSL627: Previously Untreated Patients (PUPs)
The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule at their discretion, taking into consideration the World Federation of Hemophilia (WFH) guidelines, the type of bleeding episode, location of the bleeding, subject's age, and other disease characteristics.
rVIII-SingleChain: Recombinant single-chain coagulation factor VIII
|
|---|---|---|
|
Total Amount of CSL627 Administered During Surgery Period in PUPs
Participant 1
|
15693 IU
|
—
|
|
Total Amount of CSL627 Administered During Surgery Period in PUPs
Participant 2
|
5631 IU
|
—
|
|
Total Amount of CSL627 Administered During Surgery Period in PUPs
Participant 3
|
7330 IU
|
—
|
SECONDARY outcome
Timeframe: From the start of surgery through the post-operative recovery (generally up to 14 days after surgery)Population: Surgery population - comprised all subjects in the Safety Population who received at least 1 dose of rVIII-SingleChain for surgical prophylaxis
The investigator will rate the efficacy of the rVIII-SingleChain treatment during surgery based on a hemostatic efficacy four point rating scale of "excellent, good, moderate or poor/no response".
Outcome measures
| Measure |
CSL627: Previously Treated Patients (PTPs)
n=32 Surgeries
The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule for the subject based upon the subject's pharmacokinetic (PK) profile, rVIII-SingleChain PK data, previous FVIII treatment regimen, and bleeding phenotype, if available.
rVIII-SingleChain: Recombinant single-chain coagulation factor VIII
|
CSL627: Previously Untreated Patients (PUPs)
n=3 Surgeries
The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule at their discretion, taking into consideration the World Federation of Hemophilia (WFH) guidelines, the type of bleeding episode, location of the bleeding, subject's age, and other disease characteristics.
rVIII-SingleChain: Recombinant single-chain coagulation factor VIII
|
|---|---|---|
|
Hemostatic Efficacy of rVIII-SingleChain for PTPs and PUPs Who Undergo Surgery
Excellent
|
28 Number of surgeries
|
3 Number of surgeries
|
|
Hemostatic Efficacy of rVIII-SingleChain for PTPs and PUPs Who Undergo Surgery
Good
|
4 Number of surgeries
|
0 Number of surgeries
|
|
Hemostatic Efficacy of rVIII-SingleChain for PTPs and PUPs Who Undergo Surgery
Moderate
|
0 Number of surgeries
|
0 Number of surgeries
|
|
Hemostatic Efficacy of rVIII-SingleChain for PTPs and PUPs Who Undergo Surgery
Poor/No response
|
0 Number of surgeries
|
0 Number of surgeries
|
SECONDARY outcome
Timeframe: Up to 5 yearsPopulation: SP
Outcome measures
| Measure |
CSL627: Previously Treated Patients (PTPs)
n=198 Participants
The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule for the subject based upon the subject's pharmacokinetic (PK) profile, rVIII-SingleChain PK data, previous FVIII treatment regimen, and bleeding phenotype, if available.
rVIII-SingleChain: Recombinant single-chain coagulation factor VIII
|
CSL627: Previously Untreated Patients (PUPs)
The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule at their discretion, taking into consideration the World Federation of Hemophilia (WFH) guidelines, the type of bleeding episode, location of the bleeding, subject's age, and other disease characteristics.
rVIII-SingleChain: Recombinant single-chain coagulation factor VIII
|
|---|---|---|
|
Incidence of Inhibitor Formation to FVIII in PTPs After 10 EDs and After 50 EDs
after 50 EDs
|
0 percentage of participants
|
—
|
|
Incidence of Inhibitor Formation to FVIII in PTPs After 10 EDs and After 50 EDs
after 10 EDs
|
0 percentage of participants
|
—
|
SECONDARY outcome
Timeframe: PTPs: At the closest visit after 100 EDs (up to 5 years). PUPs: At the closest visit after 50 EDs (up to 5 years).Population: SP
Outcome measures
| Measure |
CSL627: Previously Treated Patients (PTPs)
n=222 Participants
The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule for the subject based upon the subject's pharmacokinetic (PK) profile, rVIII-SingleChain PK data, previous FVIII treatment regimen, and bleeding phenotype, if available.
rVIII-SingleChain: Recombinant single-chain coagulation factor VIII
|
CSL627: Previously Untreated Patients (PUPs)
n=24 Participants
The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule at their discretion, taking into consideration the World Federation of Hemophilia (WFH) guidelines, the type of bleeding episode, location of the bleeding, subject's age, and other disease characteristics.
rVIII-SingleChain: Recombinant single-chain coagulation factor VIII
|
|---|---|---|
|
Percentage of PTPs and PUPs Developing Antibodies Against CSL627
|
15.3 percentage of participants
|
70.8 percentage of participants
|
SECONDARY outcome
Timeframe: PTPs: At the closest visit after 100 EDs (up to 5 years). PUPs: At the closest visit after 50 EDs (up to 5 years).Population: SP
Outcome measures
| Measure |
CSL627: Previously Treated Patients (PTPs)
n=222 Participants
The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule for the subject based upon the subject's pharmacokinetic (PK) profile, rVIII-SingleChain PK data, previous FVIII treatment regimen, and bleeding phenotype, if available.
rVIII-SingleChain: Recombinant single-chain coagulation factor VIII
|
CSL627: Previously Untreated Patients (PUPs)
n=24 Participants
The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule at their discretion, taking into consideration the World Federation of Hemophilia (WFH) guidelines, the type of bleeding episode, location of the bleeding, subject's age, and other disease characteristics.
rVIII-SingleChain: Recombinant single-chain coagulation factor VIII
|
|---|---|---|
|
Percentage of PTPs and PUPs Developing Antibodies to Chinese Hamster Ovary (CHO) Proteins
|
0 percentage of participants
|
0 percentage of participants
|
SECONDARY outcome
Timeframe: At the closest visit after 10 EDs (up to 5 years)Population: SP
High-titer inhibitor is defined as an inhibitor titer of ≥ 5 Bethesda units/mL.
Outcome measures
| Measure |
CSL627: Previously Treated Patients (PTPs)
n=24 Participants
The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule for the subject based upon the subject's pharmacokinetic (PK) profile, rVIII-SingleChain PK data, previous FVIII treatment regimen, and bleeding phenotype, if available.
rVIII-SingleChain: Recombinant single-chain coagulation factor VIII
|
CSL627: Previously Untreated Patients (PUPs)
The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule at their discretion, taking into consideration the World Federation of Hemophilia (WFH) guidelines, the type of bleeding episode, location of the bleeding, subject's age, and other disease characteristics.
rVIII-SingleChain: Recombinant single-chain coagulation factor VIII
|
|---|---|---|
|
Number of PUPs With High-titer Inhibitor Formation to FVIII After 10 EDs With CSL627
|
4 participants
|
—
|
SECONDARY outcome
Timeframe: At the closest visit after 10 and after 50 EDs (up to 5 years)Population: SP
Low-titer inhibitor is defined as an inhibitor titer of less than 5 Bethesda units/mL.
Outcome measures
| Measure |
CSL627: Previously Treated Patients (PTPs)
n=24 Participants
The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule for the subject based upon the subject's pharmacokinetic (PK) profile, rVIII-SingleChain PK data, previous FVIII treatment regimen, and bleeding phenotype, if available.
rVIII-SingleChain: Recombinant single-chain coagulation factor VIII
|
CSL627: Previously Untreated Patients (PUPs)
The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule at their discretion, taking into consideration the World Federation of Hemophilia (WFH) guidelines, the type of bleeding episode, location of the bleeding, subject's age, and other disease characteristics.
rVIII-SingleChain: Recombinant single-chain coagulation factor VIII
|
|---|---|---|
|
Number of PUPs With Low-titer Inhibitor Formation to FVIII After 10 EDs and After 50 EDs With CSL627
after 10 EDs
|
4 participants
|
—
|
|
Number of PUPs With Low-titer Inhibitor Formation to FVIII After 10 EDs and After 50 EDs With CSL627
after 50 EDs
|
0 participants
|
—
|
SECONDARY outcome
Timeframe: Up to 5 yearsPopulation: SP
Outcome measures
| Measure |
CSL627: Previously Treated Patients (PTPs)
n=24 Participants
The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule for the subject based upon the subject's pharmacokinetic (PK) profile, rVIII-SingleChain PK data, previous FVIII treatment regimen, and bleeding phenotype, if available.
rVIII-SingleChain: Recombinant single-chain coagulation factor VIII
|
CSL627: Previously Untreated Patients (PUPs)
The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule at their discretion, taking into consideration the World Federation of Hemophilia (WFH) guidelines, the type of bleeding episode, location of the bleeding, subject's age, and other disease characteristics.
rVIII-SingleChain: Recombinant single-chain coagulation factor VIII
|
|---|---|---|
|
Incidence of Total Inhibitor Formation to FVIII in PUPs
|
50.0 percentage of participants
|
—
|
SECONDARY outcome
Timeframe: Up to 5 yearsPopulation: EP. This endpoint was analyzed for PUPs only.
Percentage of bleeding episodes treated successfully where treatment success for a bleeding episode is defined as a rating of "excellent" or "good" on the investigator's clinical assessment of hemostatic efficacy 4-point scale "excellent, good, moderate or poor/no response". Non-major bleeding episodes are those not requiring treatment at the hemophilia center or not threatening subject's life or loss of limb.
Outcome measures
| Measure |
CSL627: Previously Treated Patients (PTPs)
n=315 Treated bleeding events
The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule for the subject based upon the subject's pharmacokinetic (PK) profile, rVIII-SingleChain PK data, previous FVIII treatment regimen, and bleeding phenotype, if available.
rVIII-SingleChain: Recombinant single-chain coagulation factor VIII
|
CSL627: Previously Untreated Patients (PUPs)
The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule at their discretion, taking into consideration the World Federation of Hemophilia (WFH) guidelines, the type of bleeding episode, location of the bleeding, subject's age, and other disease characteristics.
rVIII-SingleChain: Recombinant single-chain coagulation factor VIII
|
|---|---|---|
|
Percent Treatment Success for Non-major Bleeding Episodes in PUPs
|
92.1 percentage of treated bleeding events
Interval 87.0 to 95.3
|
—
|
SECONDARY outcome
Timeframe: Up to 6 hours after first infusionPopulation: SP. This endpoint was analyzed for PUPs only.
Vital signs assessments include heart rate, blood pressure, and body temperature. Clinical significance of an abnormality will be assessed by the investigator.
Outcome measures
| Measure |
CSL627: Previously Treated Patients (PTPs)
n=24 Participants
The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule for the subject based upon the subject's pharmacokinetic (PK) profile, rVIII-SingleChain PK data, previous FVIII treatment regimen, and bleeding phenotype, if available.
rVIII-SingleChain: Recombinant single-chain coagulation factor VIII
|
CSL627: Previously Untreated Patients (PUPs)
The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule at their discretion, taking into consideration the World Federation of Hemophilia (WFH) guidelines, the type of bleeding episode, location of the bleeding, subject's age, and other disease characteristics.
rVIII-SingleChain: Recombinant single-chain coagulation factor VIII
|
|---|---|---|
|
Percentage of PUPs With Clinically Significant Abnormal Vital Signs Values After First Infusion of CSL627
|
0 percentage of participants
|
—
|
SECONDARY outcome
Timeframe: Up to 5 yearsPopulation: SP. This endpoint was analyzed for PUPs only.
Vital signs assessments include heart rate, blood pressure, and body temperature. Clinical significance of an abnormality will be assessed by the investigator.
Outcome measures
| Measure |
CSL627: Previously Treated Patients (PTPs)
n=24 Participants
The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule for the subject based upon the subject's pharmacokinetic (PK) profile, rVIII-SingleChain PK data, previous FVIII treatment regimen, and bleeding phenotype, if available.
rVIII-SingleChain: Recombinant single-chain coagulation factor VIII
|
CSL627: Previously Untreated Patients (PUPs)
The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule at their discretion, taking into consideration the World Federation of Hemophilia (WFH) guidelines, the type of bleeding episode, location of the bleeding, subject's age, and other disease characteristics.
rVIII-SingleChain: Recombinant single-chain coagulation factor VIII
|
|---|---|---|
|
Percentage of PUPs With Treatment-emergent Clinically Significant Abnormal Vital Signs Values
|
0 percentage of participants
|
—
|
Adverse Events
CSL627: Previously Treated Patients (PTPs)
Serious events: 23 serious events
Other events: 130 other events
Deaths: 1 deaths
CSL627: Previously Untreated Patients (PUPs)
Serious events: 14 serious events
Other events: 23 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
CSL627: Previously Treated Patients (PTPs)
n=222 participants at risk
The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule for the subject based upon the subject's pharmacokinetic (PK) profile, rVIII-SingleChain PK data, previous FVIII treatment regimen, and bleeding phenotype, if available.
rVIII-SingleChain: Recombinant single-chain coagulation factor VIII
|
CSL627: Previously Untreated Patients (PUPs)
n=24 participants at risk
The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule at their discretion, taking into consideration the World Federation of Hemophilia (WFH) guidelines, the type of bleeding episode, location of the bleeding, subject's age, and other disease characteristics.
rVIII-SingleChain: Recombinant single-chain coagulation factor VIII
|
|---|---|---|
|
Injury, poisoning and procedural complications
Wound haemorrhage
|
0.00%
0/222 • Up to 5 years
|
4.2%
1/24 • Number of events 1 • Up to 5 years
|
|
Investigations
Anti factor VIII antibody positive
|
0.00%
0/222 • Up to 5 years
|
25.0%
6/24 • Number of events 6 • Up to 5 years
|
|
Investigations
Inhibiting antibodies positive
|
0.00%
0/222 • Up to 5 years
|
20.8%
5/24 • Number of events 5 • Up to 5 years
|
|
Blood and lymphatic system disorders
Factor VIII inhibition
|
0.00%
0/222 • Up to 5 years
|
4.2%
1/24 • Number of events 1 • Up to 5 years
|
|
Social circumstances
Pregnancy of partner
|
0.45%
1/222 • Number of events 1 • Up to 5 years
|
0.00%
0/24 • Up to 5 years
|
|
Nervous system disorders
Generalised tonic-clonic seizure
|
0.45%
1/222 • Number of events 1 • Up to 5 years
|
0.00%
0/24 • Up to 5 years
|
|
Vascular disorders
Shock
|
0.45%
1/222 • Number of events 1 • Up to 5 years
|
0.00%
0/24 • Up to 5 years
|
|
Vascular disorders
Vasospasm
|
0.45%
1/222 • Number of events 1 • Up to 5 years
|
0.00%
0/24 • Up to 5 years
|
|
Vascular disorders
Haemorrhage
|
0.00%
0/222 • Up to 5 years
|
4.2%
1/24 • Number of events 1 • Up to 5 years
|
|
Injury, poisoning and procedural complications
Road traffic accident
|
0.90%
2/222 • Number of events 2 • Up to 5 years
|
0.00%
0/24 • Up to 5 years
|
|
Injury, poisoning and procedural complications
Abdominal injury
|
0.45%
1/222 • Number of events 1 • Up to 5 years
|
0.00%
0/24 • Up to 5 years
|
|
Injury, poisoning and procedural complications
Periprosthetic fracture
|
0.45%
1/222 • Number of events 1 • Up to 5 years
|
0.00%
0/24 • Up to 5 years
|
|
Injury, poisoning and procedural complications
Skin laceration
|
0.45%
1/222 • Number of events 1 • Up to 5 years
|
0.00%
0/24 • Up to 5 years
|
|
Injury, poisoning and procedural complications
Fall
|
0.00%
0/222 • Up to 5 years
|
8.3%
2/24 • Number of events 2 • Up to 5 years
|
|
Nervous system disorders
Lennox-Gastaut syndrome
|
0.45%
1/222 • Number of events 1 • Up to 5 years
|
0.00%
0/24 • Up to 5 years
|
|
Nervous system disorders
Seizure
|
0.45%
1/222 • Number of events 1 • Up to 5 years
|
0.00%
0/24 • Up to 5 years
|
|
Eye disorders
Blindness transient
|
0.45%
1/222 • Number of events 1 • Up to 5 years
|
0.00%
0/24 • Up to 5 years
|
|
General disorders
Chest pain
|
0.45%
1/222 • Number of events 1 • Up to 5 years
|
0.00%
0/24 • Up to 5 years
|
|
Gastrointestinal disorders
Diarrhoea
|
0.45%
1/222 • Number of events 1 • Up to 5 years
|
0.00%
0/24 • Up to 5 years
|
|
Gastrointestinal disorders
Duodenal ulcer
|
0.45%
1/222 • Number of events 1 • Up to 5 years
|
0.00%
0/24 • Up to 5 years
|
|
Gastrointestinal disorders
Gastritis erosive
|
0.45%
1/222 • Number of events 1 • Up to 5 years
|
0.00%
0/24 • Up to 5 years
|
|
Gastrointestinal disorders
Gastrointestinal haemorrhage
|
0.45%
1/222 • Number of events 1 • Up to 5 years
|
0.00%
0/24 • Up to 5 years
|
|
Gastrointestinal disorders
Haemorrhoids
|
0.45%
1/222 • Number of events 1 • Up to 5 years
|
0.00%
0/24 • Up to 5 years
|
|
Gastrointestinal disorders
Mouth haemorrhage
|
0.00%
0/222 • Up to 5 years
|
4.2%
1/24 • Number of events 1 • Up to 5 years
|
|
Renal and urinary disorders
Nephritis
|
0.45%
1/222 • Number of events 1 • Up to 5 years
|
0.00%
0/24 • Up to 5 years
|
|
Musculoskeletal and connective tissue disorders
Haemophilic arthropathy
|
1.4%
3/222 • Number of events 3 • Up to 5 years
|
0.00%
0/24 • Up to 5 years
|
|
Musculoskeletal and connective tissue disorders
Muscle haemorrhage
|
0.45%
1/222 • Number of events 1 • Up to 5 years
|
0.00%
0/24 • Up to 5 years
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal stiffness
|
0.45%
1/222 • Number of events 1 • Up to 5 years
|
0.00%
0/24 • Up to 5 years
|
|
Musculoskeletal and connective tissue disorders
Tendinous contracture
|
0.45%
1/222 • Number of events 1 • Up to 5 years
|
0.00%
0/24 • Up to 5 years
|
|
Musculoskeletal and connective tissue disorders
Muscle spasms
|
0.00%
0/222 • Up to 5 years
|
4.2%
1/24 • Number of events 1 • Up to 5 years
|
|
Infections and infestations
Appendicitis
|
0.90%
2/222 • Number of events 2 • Up to 5 years
|
0.00%
0/24 • Up to 5 years
|
|
Infections and infestations
Cellulitis
|
0.45%
1/222 • Number of events 1 • Up to 5 years
|
0.00%
0/24 • Up to 5 years
|
|
Infections and infestations
Lower respiratory tract infection
|
0.45%
1/222 • Number of events 1 • Up to 5 years
|
0.00%
0/24 • Up to 5 years
|
|
Infections and infestations
Pneumonia
|
0.45%
1/222 • Number of events 1 • Up to 5 years
|
0.00%
0/24 • Up to 5 years
|
|
Infections and infestations
Wound infection
|
0.45%
1/222 • Number of events 1 • Up to 5 years
|
0.00%
0/24 • Up to 5 years
|
|
Infections and infestations
Device related infection
|
0.00%
0/222 • Up to 5 years
|
4.2%
1/24 • Number of events 1 • Up to 5 years
|
|
Infections and infestations
Acinetobacter infection
|
0.00%
0/222 • Up to 5 years
|
4.2%
1/24 • Number of events 1 • Up to 5 years
|
|
Infections and infestations
Influenza
|
0.45%
1/222 • Number of events 1 • Up to 5 years
|
0.00%
0/24 • Up to 5 years
|
Other adverse events
| Measure |
CSL627: Previously Treated Patients (PTPs)
n=222 participants at risk
The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule for the subject based upon the subject's pharmacokinetic (PK) profile, rVIII-SingleChain PK data, previous FVIII treatment regimen, and bleeding phenotype, if available.
rVIII-SingleChain: Recombinant single-chain coagulation factor VIII
|
CSL627: Previously Untreated Patients (PUPs)
n=24 participants at risk
The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule at their discretion, taking into consideration the World Federation of Hemophilia (WFH) guidelines, the type of bleeding episode, location of the bleeding, subject's age, and other disease characteristics.
rVIII-SingleChain: Recombinant single-chain coagulation factor VIII
|
|---|---|---|
|
Vascular disorders
Haematoma
|
0.90%
2/222 • Number of events 2 • Up to 5 years
|
8.3%
2/24 • Number of events 3 • Up to 5 years
|
|
Immune system disorders
Allergy to arthropod sting
|
0.00%
0/222 • Up to 5 years
|
8.3%
2/24 • Number of events 5 • Up to 5 years
|
|
General disorders
Pyrexia
|
6.3%
14/222 • Number of events 18 • Up to 5 years
|
62.5%
15/24 • Number of events 44 • Up to 5 years
|
|
General disorders
Malaise
|
0.00%
0/222 • Up to 5 years
|
8.3%
2/24 • Number of events 4 • Up to 5 years
|
|
Injury, poisoning and procedural complications
Fall
|
5.0%
11/222 • Number of events 12 • Up to 5 years
|
12.5%
3/24 • Number of events 5 • Up to 5 years
|
|
Injury, poisoning and procedural complications
Head injury
|
5.0%
11/222 • Number of events 11 • Up to 5 years
|
4.2%
1/24 • Number of events 3 • Up to 5 years
|
|
Injury, poisoning and procedural complications
Contusion
|
5.0%
11/222 • Number of events 13 • Up to 5 years
|
0.00%
0/24 • Up to 5 years
|
|
Investigations
Coronavirus test positive
|
0.00%
0/222 • Up to 5 years
|
8.3%
2/24 • Number of events 2 • Up to 5 years
|
|
Investigations
Inhibiting antibodies positive
|
0.00%
0/222 • Up to 5 years
|
8.3%
2/24 • Number of events 2 • Up to 5 years
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
5.9%
13/222 • Number of events 19 • Up to 5 years
|
25.0%
6/24 • Number of events 13 • Up to 5 years
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhoea
|
1.4%
3/222 • Number of events 3 • Up to 5 years
|
8.3%
2/24 • Number of events 3 • Up to 5 years
|
|
Respiratory, thoracic and mediastinal disorders
Bronchospasm
|
0.00%
0/222 • Up to 5 years
|
8.3%
2/24 • Number of events 4 • Up to 5 years
|
|
Blood and lymphatic system disorders
Iron deficiency anaemia
|
0.90%
2/222 • Number of events 3 • Up to 5 years
|
12.5%
3/24 • Number of events 3 • Up to 5 years
|
|
Nervous system disorders
Headache
|
9.0%
20/222 • Number of events 29 • Up to 5 years
|
0.00%
0/24 • Up to 5 years
|
|
Ear and labyrinth disorders
Ear pain
|
1.8%
4/222 • Number of events 4 • Up to 5 years
|
8.3%
2/24 • Number of events 2 • Up to 5 years
|
|
Gastrointestinal disorders
Dental caries
|
5.0%
11/222 • Number of events 11 • Up to 5 years
|
0.00%
0/24 • Up to 5 years
|
|
Gastrointestinal disorders
Diarrhoea
|
2.3%
5/222 • Number of events 5 • Up to 5 years
|
20.8%
5/24 • Number of events 6 • Up to 5 years
|
|
Gastrointestinal disorders
Vomiting
|
1.8%
4/222 • Number of events 5 • Up to 5 years
|
12.5%
3/24 • Number of events 8 • Up to 5 years
|
|
Skin and subcutaneous tissue disorders
Rash
|
2.7%
6/222 • Number of events 6 • Up to 5 years
|
12.5%
3/24 • Number of events 3 • Up to 5 years
|
|
Skin and subcutaneous tissue disorders
Eczema
|
0.45%
1/222 • Number of events 1 • Up to 5 years
|
12.5%
3/24 • Number of events 3 • Up to 5 years
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
9.0%
20/222 • Number of events 28 • Up to 5 years
|
4.2%
1/24 • Number of events 1 • Up to 5 years
|
|
Musculoskeletal and connective tissue disorders
Joint swelling
|
1.8%
4/222 • Number of events 5 • Up to 5 years
|
8.3%
2/24 • Number of events 2 • Up to 5 years
|
|
Musculoskeletal and connective tissue disorders
Haemarthrosis
|
0.90%
2/222 • Number of events 3 • Up to 5 years
|
8.3%
2/24 • Number of events 2 • Up to 5 years
|
|
Infections and infestations
Nasopharyngitis
|
18.0%
40/222 • Number of events 73 • Up to 5 years
|
37.5%
9/24 • Number of events 15 • Up to 5 years
|
|
Infections and infestations
Upper respiratory tract infection
|
11.3%
25/222 • Number of events 37 • Up to 5 years
|
29.2%
7/24 • Number of events 18 • Up to 5 years
|
|
Infections and infestations
Influenza
|
6.3%
14/222 • Number of events 16 • Up to 5 years
|
16.7%
4/24 • Number of events 6 • Up to 5 years
|
|
Infections and infestations
Tonsillitis
|
5.9%
13/222 • Number of events 17 • Up to 5 years
|
20.8%
5/24 • Number of events 5 • Up to 5 years
|
|
Infections and infestations
Rhinitis
|
3.2%
7/222 • Number of events 7 • Up to 5 years
|
25.0%
6/24 • Number of events 10 • Up to 5 years
|
|
Infections and infestations
Ear infection
|
2.3%
5/222 • Number of events 8 • Up to 5 years
|
20.8%
5/24 • Number of events 9 • Up to 5 years
|
|
Infections and infestations
Conjunctivitis
|
2.7%
6/222 • Number of events 6 • Up to 5 years
|
12.5%
3/24 • Number of events 4 • Up to 5 years
|
|
Infections and infestations
Bronchitis
|
2.3%
5/222 • Number of events 7 • Up to 5 years
|
12.5%
3/24 • Number of events 3 • Up to 5 years
|
|
Infections and infestations
Varicella
|
0.90%
2/222 • Number of events 2 • Up to 5 years
|
25.0%
6/24 • Number of events 7 • Up to 5 years
|
|
Infections and infestations
Otitis media
|
1.4%
3/222 • Number of events 5 • Up to 5 years
|
16.7%
4/24 • Number of events 5 • Up to 5 years
|
|
Infections and infestations
Viral rhinitis
|
0.45%
1/222 • Number of events 1 • Up to 5 years
|
8.3%
2/24 • Number of events 2 • Up to 5 years
|
|
Infections and infestations
Scarlet fever
|
0.00%
0/222 • Up to 5 years
|
8.3%
2/24 • Number of events 2 • Up to 5 years
|
|
Infections and infestations
Tinea capitis
|
0.00%
0/222 • Up to 5 years
|
8.3%
2/24 • Number of events 2 • Up to 5 years
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee CSL agreements and restrictions on publishing may vary with individual investigators; however, CSL will not prohibit any investigator from publishing. CSL supports the publication of results from all centers of a multi-center trial and generally requires that reports based on single-site data not precede the primary publication of the entire clinical trial.
- Publication restrictions are in place
Restriction type: OTHER