Trial Outcomes & Findings for Study of ALXN1840 Versus Standard of Care in Pediatric Participants With Wilson Disease (NCT NCT05047523)
NCT ID: NCT05047523
Last Updated: 2024-10-23
Results Overview
Plasma samples were planned to be collected to measure ceruloplasmin-bound copper. Due to the early termination of the study, data for this Outcome Measure were not collected for any of the cohorts.
Recruitment status
TERMINATED
Study phase
PHASE3
Target enrollment
40 participants
Primary outcome timeframe
Baseline, Week 48
Results posted on
2024-10-23
Participant Flow
The study consisted of 2 periods: Primary Evaluation Period (PEP) and Open-label Extension (OLE) Period. Participants who completed the 48-week PEP were offered the opportunity to continue treatment in an up to 24-week OLE Period.
Per prespecified analysis, participants were randomized, stratified by cohort, in 1:1 ratio to ALXN1840 or continued treatment with Standard of Care (SoC) in Cohort 1 or as continued or initial therapy in Cohort 2 for the PEP. The stratification was not applicable to the OLE Period. Data was collected for the OLE Period by dose received irrespective of the cohort assigned during randomization.
Participant milestones
| Measure |
Cohort 1: ALXN1840
Participants in Cohort 1 (who received SoC therapy, that is, chelation therapy with penicillamine or trientine, zinc therapy, or a combination of both chelation and zinc therapy for \>28 days) received titrated doses of ALXN1840 orally for up to 48 weeks in the PEP.
|
Cohort 1: SoC Therapy
Participants in Cohort 1 (who received SoC therapy, that is, chelation therapy with penicillamine or trientine, zinc therapy, or a combination of both chelation and zinc therapy for \>28 days) continued to receive SoC therapy according to the local package label for up to 48 weeks in the PEP.
|
Cohort 2: ALXN1840
Participants in Cohort 2 (who were treatment naïve or who received SoC therapy for ≤28 days) received titrated doses of ALXN1840 orally for up to 48 weeks in the PEP.
|
Cohort 2: SoC Therapy
Participants in Cohort 2 (who were treatment naïve or who received SoC therapy for ≤28 days) received SoC therapy according to the local package label for up to 48 weeks in the PEP.
|
ALXN1840/ALXN1840
Participants who were randomized to the ALXN1840 group during the PEP and who completed the 48-week PEP were offered the opportunity to participate in the OLE Period and continued to receive ALXN1840 for up to 24 additional weeks.
|
SoC Therapy/ALXN1840
Participants who were randomized to the SoC group during the PEP and who completed the 48-week PEP were offered the opportunity to participate in the OLE Period and received ALXN1840 for up to 24 additional weeks.
|
|---|---|---|---|---|---|---|
|
Primary Evaluation Period (48 Weeks)
STARTED
|
15
|
16
|
4
|
5
|
0
|
0
|
|
Primary Evaluation Period (48 Weeks)
Received at Least 1 Dose of Study Drug
|
15
|
16
|
4
|
5
|
0
|
0
|
|
Primary Evaluation Period (48 Weeks)
COMPLETED
|
12
|
11
|
0
|
1
|
0
|
0
|
|
Primary Evaluation Period (48 Weeks)
NOT COMPLETED
|
3
|
5
|
4
|
4
|
0
|
0
|
|
Extension Period (24 Weeks)
STARTED
|
0
|
0
|
0
|
0
|
12
|
12
|
|
Extension Period (24 Weeks)
Received at Least 1 Dose of Study Drug
|
0
|
0
|
0
|
0
|
12
|
12
|
|
Extension Period (24 Weeks)
COMPLETED
|
0
|
0
|
0
|
0
|
3
|
3
|
|
Extension Period (24 Weeks)
NOT COMPLETED
|
0
|
0
|
0
|
0
|
9
|
9
|
Reasons for withdrawal
| Measure |
Cohort 1: ALXN1840
Participants in Cohort 1 (who received SoC therapy, that is, chelation therapy with penicillamine or trientine, zinc therapy, or a combination of both chelation and zinc therapy for \>28 days) received titrated doses of ALXN1840 orally for up to 48 weeks in the PEP.
|
Cohort 1: SoC Therapy
Participants in Cohort 1 (who received SoC therapy, that is, chelation therapy with penicillamine or trientine, zinc therapy, or a combination of both chelation and zinc therapy for \>28 days) continued to receive SoC therapy according to the local package label for up to 48 weeks in the PEP.
|
Cohort 2: ALXN1840
Participants in Cohort 2 (who were treatment naïve or who received SoC therapy for ≤28 days) received titrated doses of ALXN1840 orally for up to 48 weeks in the PEP.
|
Cohort 2: SoC Therapy
Participants in Cohort 2 (who were treatment naïve or who received SoC therapy for ≤28 days) received SoC therapy according to the local package label for up to 48 weeks in the PEP.
|
ALXN1840/ALXN1840
Participants who were randomized to the ALXN1840 group during the PEP and who completed the 48-week PEP were offered the opportunity to participate in the OLE Period and continued to receive ALXN1840 for up to 24 additional weeks.
|
SoC Therapy/ALXN1840
Participants who were randomized to the SoC group during the PEP and who completed the 48-week PEP were offered the opportunity to participate in the OLE Period and received ALXN1840 for up to 24 additional weeks.
|
|---|---|---|---|---|---|---|
|
Primary Evaluation Period (48 Weeks)
Physician Decision
|
0
|
0
|
1
|
0
|
0
|
0
|
|
Primary Evaluation Period (48 Weeks)
Withdrawal by Subject
|
0
|
1
|
0
|
0
|
0
|
0
|
|
Primary Evaluation Period (48 Weeks)
Study Terminated by Sponsor
|
2
|
4
|
3
|
4
|
0
|
0
|
|
Primary Evaluation Period (48 Weeks)
Adverse Event
|
1
|
0
|
0
|
0
|
0
|
0
|
|
Extension Period (24 Weeks)
Study Terminated by Sponsor
|
0
|
0
|
0
|
0
|
8
|
8
|
|
Extension Period (24 Weeks)
Adverse Event
|
0
|
0
|
0
|
0
|
1
|
1
|
Baseline Characteristics
Study of ALXN1840 Versus Standard of Care in Pediatric Participants With Wilson Disease
Baseline characteristics by cohort
| Measure |
Cohort 1: ALXN1840
n=15 Participants
Participants in Cohort 1 (who received SoC therapy, that is, chelation therapy with penicillamine or trientine, zinc therapy, or a combination of both chelation and zinc therapy for \>28 days) received titrated doses of ALXN1840 orally for up to 48 weeks in PEP.
|
Cohort 1: SoC Therapy
n=16 Participants
Participants in Cohort 1 (who received SoC therapy, that is, chelation therapy with penicillamine or trientine, zinc therapy, or a combination of both chelation and zinc therapy for \>28 days) continued to receive SoC therapy according to the local package label for up to 48 weeks in PEP.
|
Cohort 2: ALXN1840
n=4 Participants
Participants in Cohort 2 (who were treatment naïve or who received SoC therapy for ≤28 days) received titrated doses of ALXN1840 orally for up to 48 weeks in PEP.
|
Cohort 2: SoC Therapy
n=5 Participants
Participants in Cohort 2 (who were treatment naïve or who received SoC therapy for ≤28 days) received SoC therapy according to the local package label for up to 48 weeks in PEP.
|
Total
n=40 Participants
Total of all reporting groups
|
|---|---|---|---|---|---|
|
Age, Customized
Children (2-11 years)
|
6 Participants
n=93 Participants
|
6 Participants
n=4 Participants
|
3 Participants
n=27 Participants
|
3 Participants
n=483 Participants
|
18 Participants
n=36 Participants
|
|
Age, Customized
Adolescents (12-17 years)
|
9 Participants
n=93 Participants
|
10 Participants
n=4 Participants
|
1 Participants
n=27 Participants
|
2 Participants
n=483 Participants
|
22 Participants
n=36 Participants
|
|
Sex: Female, Male
Female
|
5 Participants
n=93 Participants
|
4 Participants
n=4 Participants
|
3 Participants
n=27 Participants
|
3 Participants
n=483 Participants
|
15 Participants
n=36 Participants
|
|
Sex: Female, Male
Male
|
10 Participants
n=93 Participants
|
12 Participants
n=4 Participants
|
1 Participants
n=27 Participants
|
2 Participants
n=483 Participants
|
25 Participants
n=36 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
5 Participants
n=93 Participants
|
4 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
0 Participants
n=483 Participants
|
9 Participants
n=36 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
10 Participants
n=93 Participants
|
11 Participants
n=4 Participants
|
4 Participants
n=27 Participants
|
5 Participants
n=483 Participants
|
30 Participants
n=36 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=93 Participants
|
1 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
0 Participants
n=483 Participants
|
1 Participants
n=36 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
0 Participants
n=483 Participants
|
0 Participants
n=36 Participants
|
|
Race (NIH/OMB)
Asian
|
5 Participants
n=93 Participants
|
6 Participants
n=4 Participants
|
1 Participants
n=27 Participants
|
3 Participants
n=483 Participants
|
15 Participants
n=36 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
0 Participants
n=483 Participants
|
0 Participants
n=36 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
0 Participants
n=483 Participants
|
0 Participants
n=36 Participants
|
|
Race (NIH/OMB)
White
|
9 Participants
n=93 Participants
|
9 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
2 Participants
n=483 Participants
|
20 Participants
n=36 Participants
|
|
Race (NIH/OMB)
More than one race
|
1 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
2 Participants
n=27 Participants
|
0 Participants
n=483 Participants
|
3 Participants
n=36 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=93 Participants
|
1 Participants
n=4 Participants
|
1 Participants
n=27 Participants
|
0 Participants
n=483 Participants
|
2 Participants
n=36 Participants
|
PRIMARY outcome
Timeframe: Baseline, Week 48Population: FAS included all participants who received at least 1 dose of ALXN1840 or SoC treatment. Due to the early termination of the study, data for this Outcome Measure were not collected for any of the cohorts.
Plasma samples were planned to be collected to measure ceruloplasmin-bound copper. Due to the early termination of the study, data for this Outcome Measure were not collected for any of the cohorts.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Baseline up to Week 48Population: Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
An adverse event (AE) was any untoward medical occurrence in a participant administered the study drug and which did not necessarily have a causal relationship with this treatment. TEAEs were defined as AEs with onset after the first dose of study intervention or existing events that worsened in severity after the first dose of study intervention. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section.
Outcome measures
| Measure |
Cohort 1: ALXN1840
n=15 Participants
Participants in Cohort 1 (who received SoC therapy, that is, chelation therapy with penicillamine or trientine, zinc therapy, or a combination of both chelation and zinc therapy for \>28 days) received titrated doses of ALXN1840 orally for up to 48 weeks in the PEP.
|
Cohort 1: SoC Therapy
n=16 Participants
Participants in Cohort 1 (who received SoC therapy, that is, chelation therapy with penicillamine or trientine, zinc therapy, or a combination of both chelation and zinc therapy for \>28 days) continued to receive SoC therapy according to the local package label for up to 48 weeks in the PEP.
|
Cohort 2: ALXN1840
n=4 Participants
Participants in Cohort 2 (who were treatment naïve or who received SoC therapy for ≤28 days) received titrated doses of ALXN1840 orally for up to 48 weeks in the PEP.
|
Cohort 2: SoC Therapy
n=5 Participants
Participants in Cohort 2 (who were treatment naïve or who received SoC therapy for ≤28 days) received SoC therapy according to the local package label for up to 48 weeks in the PEP.
|
|---|---|---|---|---|
|
Number of Participants With Treatment-emergent Adverse Events (TEAEs) During the Primary Evaluation Period
|
13 Participants
|
13 Participants
|
3 Participants
|
4 Participants
|
SECONDARY outcome
Timeframe: Week 48Population: Pharmacodynamic (PD) analysis set included all participants who had sufficient plasma samples to enable the calculation of pharmacokinetic (PK) parameters and provide PK/PD profiles. Here, 'Overall number of participants analyzed' = participants evaluable for this Outcome Measure. No participants were evaluable for this Outcome Measure for Cohort 2: ALXN1840.
Outcome measures
| Measure |
Cohort 1: ALXN1840
n=11 Participants
Participants in Cohort 1 (who received SoC therapy, that is, chelation therapy with penicillamine or trientine, zinc therapy, or a combination of both chelation and zinc therapy for \>28 days) received titrated doses of ALXN1840 orally for up to 48 weeks in the PEP.
|
Cohort 1: SoC Therapy
n=11 Participants
Participants in Cohort 1 (who received SoC therapy, that is, chelation therapy with penicillamine or trientine, zinc therapy, or a combination of both chelation and zinc therapy for \>28 days) continued to receive SoC therapy according to the local package label for up to 48 weeks in the PEP.
|
Cohort 2: ALXN1840
Participants in Cohort 2 (who were treatment naïve or who received SoC therapy for ≤28 days) received titrated doses of ALXN1840 orally for up to 48 weeks in the PEP.
|
Cohort 2: SoC Therapy
n=1 Participants
Participants in Cohort 2 (who were treatment naïve or who received SoC therapy for ≤28 days) received SoC therapy according to the local package label for up to 48 weeks in the PEP.
|
|---|---|---|---|---|
|
Area Under the Effect Versus Time Curve (AUEC) for Plasma Total Copper and Direct NCC
Total Copper
|
6342.909 nanograms (ng)*hours (hr)/milliliter(mL)
Standard Deviation 3038.3317
|
7528.864 nanograms (ng)*hours (hr)/milliliter(mL)
Standard Deviation 4167.2110
|
—
|
16369.000 nanograms (ng)*hours (hr)/milliliter(mL)
Standard Deviation NA
Data could not be calculated due to single participant.
|
|
Area Under the Effect Versus Time Curve (AUEC) for Plasma Total Copper and Direct NCC
Direct NCC
|
3882.473 nanograms (ng)*hours (hr)/milliliter(mL)
Standard Deviation 1716.4565
|
3067.864 nanograms (ng)*hours (hr)/milliliter(mL)
Standard Deviation 1524.9183
|
—
|
4214.000 nanograms (ng)*hours (hr)/milliliter(mL)
Standard Deviation NA
Data could not be calculated due to single participant.
|
SECONDARY outcome
Timeframe: Week 48Population: PK analysis set included all participants who had sufficient plasma samples to enable the calculation of PK parameters and provide PK/PD profiles. Here, 'Overall number of participants analyzed' = participants evaluable for this Outcome Measure. No participants were evaluable for this Outcome Measure for Cohort 2: ALXN1840.
Outcome measures
| Measure |
Cohort 1: ALXN1840
n=12 Participants
Participants in Cohort 1 (who received SoC therapy, that is, chelation therapy with penicillamine or trientine, zinc therapy, or a combination of both chelation and zinc therapy for \>28 days) received titrated doses of ALXN1840 orally for up to 48 weeks in the PEP.
|
Cohort 1: SoC Therapy
n=11 Participants
Participants in Cohort 1 (who received SoC therapy, that is, chelation therapy with penicillamine or trientine, zinc therapy, or a combination of both chelation and zinc therapy for \>28 days) continued to receive SoC therapy according to the local package label for up to 48 weeks in the PEP.
|
Cohort 2: ALXN1840
Participants in Cohort 2 (who were treatment naïve or who received SoC therapy for ≤28 days) received titrated doses of ALXN1840 orally for up to 48 weeks in the PEP.
|
Cohort 2: SoC Therapy
n=1 Participants
Participants in Cohort 2 (who were treatment naïve or who received SoC therapy for ≤28 days) received SoC therapy according to the local package label for up to 48 weeks in the PEP.
|
|---|---|---|---|---|
|
Maximum Observed Concentration (Cmax) of ALXN1840 for Plasma Total Molybdenum and Plasma Ultrafiltrate Molybdenum Concentrations
Plasma Total Molybdenum
|
183.150 ng/mL
Standard Deviation 110.4736
|
209.355 ng/mL
Standard Deviation 114.3741
|
—
|
307.000 ng/mL
Standard Deviation NA
Data could not be calculated due to single participant.
|
|
Maximum Observed Concentration (Cmax) of ALXN1840 for Plasma Total Molybdenum and Plasma Ultrafiltrate Molybdenum Concentrations
Plasma Ultrafiltrate Molybdenum
|
13.733 ng/mL
Standard Deviation 8.6186
|
17.708 ng/mL
Standard Deviation 27.9548
|
—
|
9.910 ng/mL
Standard Deviation NA
Data could not be calculated due to single participant.
|
SECONDARY outcome
Timeframe: Week 48Population: PK analysis set included all participants who had sufficient plasma samples to enable the calculation of PK parameters and provide PK/PD profiles. Here, 'Overall number of participants analyzed' = participants evaluable for this Outcome Measure. No participants were evaluable for this Outcome Measure for Cohort 2: ALXN1840.
Outcome measures
| Measure |
Cohort 1: ALXN1840
n=11 Participants
Participants in Cohort 1 (who received SoC therapy, that is, chelation therapy with penicillamine or trientine, zinc therapy, or a combination of both chelation and zinc therapy for \>28 days) received titrated doses of ALXN1840 orally for up to 48 weeks in the PEP.
|
Cohort 1: SoC Therapy
n=11 Participants
Participants in Cohort 1 (who received SoC therapy, that is, chelation therapy with penicillamine or trientine, zinc therapy, or a combination of both chelation and zinc therapy for \>28 days) continued to receive SoC therapy according to the local package label for up to 48 weeks in the PEP.
|
Cohort 2: ALXN1840
Participants in Cohort 2 (who were treatment naïve or who received SoC therapy for ≤28 days) received titrated doses of ALXN1840 orally for up to 48 weeks in the PEP.
|
Cohort 2: SoC Therapy
n=1 Participants
Participants in Cohort 2 (who were treatment naïve or who received SoC therapy for ≤28 days) received SoC therapy according to the local package label for up to 48 weeks in the PEP.
|
|---|---|---|---|---|
|
Area Under the Plasma Concentration Versus Time Curve From Time 0 to the End of the Dosing Interval (AUCtau) of ALXN1840 for Plasma Total Molybdenum and Plasma Ultrafiltrate Molybdenum
Plasma Total Molybdenum
|
4498.082 ng*hr/mL
Standard Deviation 3054.8300
|
2931.045 ng*hr/mL
Standard Deviation 1694.3512
|
—
|
4261.100 ng*hr/mL
Standard Deviation NA
Data could not be calculated due to single participant.
|
|
Area Under the Plasma Concentration Versus Time Curve From Time 0 to the End of the Dosing Interval (AUCtau) of ALXN1840 for Plasma Total Molybdenum and Plasma Ultrafiltrate Molybdenum
Plasma Ultrafiltrate Molybdenum
|
233.154 ng*hr/mL
Standard Deviation 145.7960
|
144.627 ng*hr/mL
Standard Deviation 88.6710
|
—
|
129.370 ng*hr/mL
Standard Deviation NA
Data could not be calculated due to single participant.
|
Adverse Events
Cohort 1: ALXN1840
Serious events: 0 serious events
Other events: 13 other events
Deaths: 0 deaths
Cohort 1: SoC Therapy
Serious events: 2 serious events
Other events: 12 other events
Deaths: 0 deaths
Cohort 2: ALXN1840
Serious events: 0 serious events
Other events: 2 other events
Deaths: 0 deaths
Cohort 2: SoC Therapy
Serious events: 0 serious events
Other events: 4 other events
Deaths: 0 deaths
ALXN1840/ALXN1840
Serious events: 0 serious events
Other events: 6 other events
Deaths: 0 deaths
SoC Therapy/ALXN1840
Serious events: 0 serious events
Other events: 8 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Cohort 1: ALXN1840
n=15 participants at risk
Participants in Cohort 1 (who received SoC therapy, that is, chelation therapy with penicillamine or trientine, zinc therapy, or a combination of both chelation and zinc therapy for \>28 days) received titrated doses of ALXN1840 orally for up to 48 weeks in the PEP.
|
Cohort 1: SoC Therapy
n=16 participants at risk
Participants in Cohort 1 (who received SoC therapy, that is, chelation therapy with penicillamine or trientine, zinc therapy, or a combination of both chelation and zinc therapy for \>28 days) continued to receive SoC therapy according to the local package label for up to 48 weeks in the PEP.
|
Cohort 2: ALXN1840
n=4 participants at risk
Participants in Cohort 2 (who were treatment naïve or who received SoC therapy for ≤28 days) received titrated doses of ALXN1840 orally for up to 48 weeks in the PEP.
|
Cohort 2: SoC Therapy
n=5 participants at risk
Participants in Cohort 2 (who were treatment naïve or who received SoC therapy for ≤28 days) received SoC therapy according to the local package label for up to 48 weeks in the PEP.
|
ALXN1840/ALXN1840
n=12 participants at risk
Participants who were randomized to the ALXN1840 group during the PEP and who completed the 48-week PEP were offered the opportunity to participate in the OLE Period and continued to receive ALXN1840 for up to 24 additional weeks.
|
SoC Therapy/ALXN1840
n=12 participants at risk
Participants who were randomized to the SoC group during the PEP and who completed the 48-week PEP were offered the opportunity to participate in the OLE Period and received ALXN1840 for up to 24 additional weeks.
|
|---|---|---|---|---|---|---|
|
Gastrointestinal disorders
Duodenal perforation
|
0.00%
0/15 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
6.2%
1/16 • Number of events 1 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/4 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/5 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/12 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/12 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
|
Infections and infestations
Gastrointestinal infection
|
0.00%
0/15 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
6.2%
1/16 • Number of events 1 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/4 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/5 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/12 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/12 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
Other adverse events
| Measure |
Cohort 1: ALXN1840
n=15 participants at risk
Participants in Cohort 1 (who received SoC therapy, that is, chelation therapy with penicillamine or trientine, zinc therapy, or a combination of both chelation and zinc therapy for \>28 days) received titrated doses of ALXN1840 orally for up to 48 weeks in the PEP.
|
Cohort 1: SoC Therapy
n=16 participants at risk
Participants in Cohort 1 (who received SoC therapy, that is, chelation therapy with penicillamine or trientine, zinc therapy, or a combination of both chelation and zinc therapy for \>28 days) continued to receive SoC therapy according to the local package label for up to 48 weeks in the PEP.
|
Cohort 2: ALXN1840
n=4 participants at risk
Participants in Cohort 2 (who were treatment naïve or who received SoC therapy for ≤28 days) received titrated doses of ALXN1840 orally for up to 48 weeks in the PEP.
|
Cohort 2: SoC Therapy
n=5 participants at risk
Participants in Cohort 2 (who were treatment naïve or who received SoC therapy for ≤28 days) received SoC therapy according to the local package label for up to 48 weeks in the PEP.
|
ALXN1840/ALXN1840
n=12 participants at risk
Participants who were randomized to the ALXN1840 group during the PEP and who completed the 48-week PEP were offered the opportunity to participate in the OLE Period and continued to receive ALXN1840 for up to 24 additional weeks.
|
SoC Therapy/ALXN1840
n=12 participants at risk
Participants who were randomized to the SoC group during the PEP and who completed the 48-week PEP were offered the opportunity to participate in the OLE Period and received ALXN1840 for up to 24 additional weeks.
|
|---|---|---|---|---|---|---|
|
Blood and lymphatic system disorders
Neutropenia
|
6.7%
1/15 • Number of events 1 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
6.2%
1/16 • Number of events 1 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
25.0%
1/4 • Number of events 1 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/5 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/12 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/12 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
|
Gastrointestinal disorders
Abdominal pain
|
20.0%
3/15 • Number of events 5 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/16 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/4 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/5 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
8.3%
1/12 • Number of events 1 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
8.3%
1/12 • Number of events 1 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
|
Gastrointestinal disorders
Abdominal pain upper
|
6.7%
1/15 • Number of events 1 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/16 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/4 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
20.0%
1/5 • Number of events 1 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/12 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/12 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
|
Gastrointestinal disorders
Aphthous ulcer
|
0.00%
0/15 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
6.2%
1/16 • Number of events 1 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
25.0%
1/4 • Number of events 1 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/5 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/12 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/12 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
|
Gastrointestinal disorders
Dyspepsia
|
6.7%
1/15 • Number of events 1 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
6.2%
1/16 • Number of events 1 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/4 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/5 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/12 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/12 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
|
Gastrointestinal disorders
Nausea
|
6.7%
1/15 • Number of events 2 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/16 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/4 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
20.0%
1/5 • Number of events 1 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
16.7%
2/12 • Number of events 2 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
8.3%
1/12 • Number of events 1 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
|
Gastrointestinal disorders
Odynophagia
|
0.00%
0/15 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
6.2%
1/16 • Number of events 1 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
25.0%
1/4 • Number of events 1 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/5 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/12 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/12 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
|
Gastrointestinal disorders
Constipation
|
0.00%
0/15 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
12.5%
2/16 • Number of events 2 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/4 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/5 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/12 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/12 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
|
Gastrointestinal disorders
Vomiting
|
0.00%
0/15 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
12.5%
2/16 • Number of events 2 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/4 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/5 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/12 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/12 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
|
General disorders
Pyrexia
|
6.7%
1/15 • Number of events 1 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
6.2%
1/16 • Number of events 1 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
25.0%
1/4 • Number of events 3 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/5 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/12 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/12 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
|
Hepatobiliary disorders
Hypertransaminasaemia
|
20.0%
3/15 • Number of events 3 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/16 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/4 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
20.0%
1/5 • Number of events 1 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/12 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/12 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
|
Infections and infestations
COVID-19
|
40.0%
6/15 • Number of events 6 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
25.0%
4/16 • Number of events 4 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/4 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/5 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/12 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/12 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
|
Infections and infestations
Nasopharyngitis
|
6.7%
1/15 • Number of events 2 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
6.2%
1/16 • Number of events 1 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
25.0%
1/4 • Number of events 2 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
20.0%
1/5 • Number of events 2 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/12 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/12 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
|
Infections and infestations
Gastroenteritis
|
13.3%
2/15 • Number of events 2 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
6.2%
1/16 • Number of events 1 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/4 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/5 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/12 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/12 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
|
Infections and infestations
Upper respiratory tract infection
|
6.7%
1/15 • Number of events 2 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
18.8%
3/16 • Number of events 3 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/4 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
20.0%
1/5 • Number of events 2 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/12 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
33.3%
4/12 • Number of events 5 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
|
Infections and infestations
Respiratory tract infection
|
6.7%
1/15 • Number of events 1 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
18.8%
3/16 • Number of events 4 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/4 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/5 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/12 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/12 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
|
Infections and infestations
Viral infection
|
0.00%
0/15 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
12.5%
2/16 • Number of events 2 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/4 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
20.0%
1/5 • Number of events 1 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/12 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/12 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
|
Infections and infestations
Gastrointestinal infection
|
0.00%
0/15 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
6.2%
1/16 • Number of events 1 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/4 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/5 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/12 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/12 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
|
Investigations
Alanine aminotransferase increased
|
40.0%
6/15 • Number of events 7 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/16 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
25.0%
1/4 • Number of events 2 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/5 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/12 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
16.7%
2/12 • Number of events 2 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
|
Investigations
Aspartate aminotransferase increased
|
13.3%
2/15 • Number of events 2 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/16 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
25.0%
1/4 • Number of events 2 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/5 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/12 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/12 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
|
Investigations
Gamma-glutamyltransferase increased
|
20.0%
3/15 • Number of events 3 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/16 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/4 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/5 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/12 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/12 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
|
Investigations
Blood cholesterol increased
|
13.3%
2/15 • Number of events 2 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/16 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/4 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/5 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/12 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/12 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
|
Investigations
Blood triglycerides increased
|
13.3%
2/15 • Number of events 2 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/16 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/4 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/5 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/12 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
16.7%
2/12 • Number of events 2 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
|
Investigations
Electrocardiogram abnormal
|
6.7%
1/15 • Number of events 1 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
6.2%
1/16 • Number of events 1 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/4 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/5 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/12 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/12 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
13.3%
2/15 • Number of events 2 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/16 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
25.0%
1/4 • Number of events 1 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/5 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/12 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/12 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
13.3%
2/15 • Number of events 2 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/16 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
25.0%
1/4 • Number of events 1 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/5 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/12 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/12 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
0.00%
0/15 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
12.5%
2/16 • Number of events 2 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/4 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/5 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/12 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/12 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
|
Nervous system disorders
Headache
|
13.3%
2/15 • Number of events 2 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
6.2%
1/16 • Number of events 1 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
25.0%
1/4 • Number of events 1 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/5 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/12 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
16.7%
2/12 • Number of events 3 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
6.7%
1/15 • Number of events 1 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
6.2%
1/16 • Number of events 1 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/4 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/5 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/12 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/12 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
0.00%
0/15 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
12.5%
2/16 • Number of events 2 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/4 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/5 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/12 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/12 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
|
Skin and subcutaneous tissue disorders
Rash
|
6.7%
1/15 • Number of events 1 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
6.2%
1/16 • Number of events 2 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/4 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/5 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/12 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/12 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
|
Skin and subcutaneous tissue disorders
Acne
|
0.00%
0/15 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
6.2%
1/16 • Number of events 1 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/4 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
20.0%
1/5 • Number of events 1 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/12 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/12 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
|
Gastrointestinal disorders
Diarrhoea
|
0.00%
0/15 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/16 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/4 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/5 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
8.3%
1/12 • Number of events 1 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
16.7%
2/12 • Number of events 2 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
|
Infections and infestations
Influenza
|
0.00%
0/15 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/16 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/4 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/5 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
8.3%
1/12 • Number of events 1 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
16.7%
2/12 • Number of events 2 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
|
Metabolism and nutrition disorders
Hypertriglyceridaemia
|
0.00%
0/15 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/16 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/4 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/5 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
8.3%
1/12 • Number of events 1 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
16.7%
2/12 • Number of events 2 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhoea
|
0.00%
0/15 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/16 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/4 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
0.00%
0/5 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
8.3%
1/12 • Number of events 1 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
8.3%
1/12 • Number of events 1 • Baseline up to Week 76
Safety analysis set included all participants who received at least 1 dose of ALXN1840 or SoC treatment.
|
Additional Information
Alexion Pharmaceuticals, Inc.
Alexion Pharmaceuticals, Inc.
Phone: +1.855.752.2356
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place