Trial Outcomes & Findings for Extension Study of RA101495 for Patients With PNH Who Have Completed a Zilucoplan (RA101495) Clinical Study (NCT NCT03225287)
NCT ID: NCT03225287
Last Updated: 2023-09-28
Results Overview
TEAEs were defined as an AE that occurs after a participant's initial treatment zilucoplan start for this study (RA101495-01.202) that was not present at the time of treatment start, or an AE that increases in severity after treatment start in this study, if the event was present at the time of treatment start.
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
19 participants
Primary outcome timeframe
From Day 1 until the Final Study Visit (up to Month 49)
Results posted on
2023-09-28
Participant Flow
The study started to enroll participants in July 2017 and concluded in October 2021.
The Participant Flow refers to the All Enrolled Subjects.
Participant milestones
| Measure |
Zilucoplan-Cohort A (Eculizumab Naïve)
Cohort A (Eculizumab Naive) included all participants from study RA101495-01.201 (NCT03078582) who had not received eculizumab for treatment of paroxysmal nocturnal hemoglobinuria (PNH). Participants received a loading dose of 0.3 milligram/kilogram (mg/kg) zilucoplan administered by subcutaneous (SC) injection at Day 1 of this study. Following in-clinic education and training, all participants self-injected 0.1 mg/kg of zilucoplan by SC injection once daily for 12 months. From Week 2 onwards, if a participant had not achieved an adequate response, the zilucoplan dose was escalated to 0.3 mg/kg daily.
|
Zilucoplan-Cohort B (Eculizumab Switch)
Cohort B (Eculizumab Switch) included all participants from study RA101495-01.201 (NCT03078582) who had received eculizumab for treatment of PNH. Participants received a loading dose of 0.3 mg/kg zilucoplan administered by SC injection at Day 1 of this study. Following in-clinic education and training, all participants self-injected 0.1 mg/kg of zilucoplan by SC injection once daily for 12 months. From Week 2 onwards, if a participant had not achieved an adequate response, the zilucoplan dose was escalated to 0.3 mg/kg daily.
|
Zilucoplan (Inadequate Responder to Eculizumab)
Inadequate Responder cohort included participants from qualifying study RA101495-01.203 (NCT03030183) who had an inadequate response to eculizumab treatment for PNH. Participants received a loading dose of 0.3 mg/kg zilucoplan administered by SC injection at Day 1 of this study. Following in-clinic education and training, all participants self-injected 0.1 mg/kg of zilucoplan by SC injection once daily for 12 months. From Week 2 onwards, if a participant had not achieved an adequate response, the zilucoplan dose was escalated to 0.3 mg/kg daily.
|
|---|---|---|---|
|
Overall Study
STARTED
|
10
|
6
|
3
|
|
Overall Study
COMPLETED
|
0
|
0
|
0
|
|
Overall Study
NOT COMPLETED
|
10
|
6
|
3
|
Reasons for withdrawal
| Measure |
Zilucoplan-Cohort A (Eculizumab Naïve)
Cohort A (Eculizumab Naive) included all participants from study RA101495-01.201 (NCT03078582) who had not received eculizumab for treatment of paroxysmal nocturnal hemoglobinuria (PNH). Participants received a loading dose of 0.3 milligram/kilogram (mg/kg) zilucoplan administered by subcutaneous (SC) injection at Day 1 of this study. Following in-clinic education and training, all participants self-injected 0.1 mg/kg of zilucoplan by SC injection once daily for 12 months. From Week 2 onwards, if a participant had not achieved an adequate response, the zilucoplan dose was escalated to 0.3 mg/kg daily.
|
Zilucoplan-Cohort B (Eculizumab Switch)
Cohort B (Eculizumab Switch) included all participants from study RA101495-01.201 (NCT03078582) who had received eculizumab for treatment of PNH. Participants received a loading dose of 0.3 mg/kg zilucoplan administered by SC injection at Day 1 of this study. Following in-clinic education and training, all participants self-injected 0.1 mg/kg of zilucoplan by SC injection once daily for 12 months. From Week 2 onwards, if a participant had not achieved an adequate response, the zilucoplan dose was escalated to 0.3 mg/kg daily.
|
Zilucoplan (Inadequate Responder to Eculizumab)
Inadequate Responder cohort included participants from qualifying study RA101495-01.203 (NCT03030183) who had an inadequate response to eculizumab treatment for PNH. Participants received a loading dose of 0.3 mg/kg zilucoplan administered by SC injection at Day 1 of this study. Following in-clinic education and training, all participants self-injected 0.1 mg/kg of zilucoplan by SC injection once daily for 12 months. From Week 2 onwards, if a participant had not achieved an adequate response, the zilucoplan dose was escalated to 0.3 mg/kg daily.
|
|---|---|---|---|
|
Overall Study
Adverse Event
|
0
|
0
|
1
|
|
Overall Study
Protocol non-compliance
|
0
|
0
|
1
|
|
Overall Study
Subject withdraws consent
|
3
|
0
|
0
|
|
Overall Study
Sponsor, regulatory, or EC/IRB request
|
7
|
3
|
1
|
|
Overall Study
Trial drug not effective
|
0
|
1
|
0
|
|
Overall Study
Need of transfusions and signs of hemolysis
|
0
|
1
|
0
|
|
Overall Study
Stem cell transplantation
|
0
|
1
|
0
|
Baseline Characteristics
Extension Study of RA101495 for Patients With PNH Who Have Completed a Zilucoplan (RA101495) Clinical Study
Baseline characteristics by cohort
| Measure |
Zilucoplan-Cohort A (Eculizumab Naïve)
n=10 Participants
Cohort A (Eculizumab Naive) included all participants from study RA101495-01.201 (NCT03078582) who had not received eculizumab for treatment of paroxysmal nocturnal hemoglobinuria (PNH). Participants received a loading dose of 0.3 milligram/kilogram (mg/kg) zilucoplan administered by subcutaneous (SC) injection at Day 1 of this study. Following in-clinic education and training, all participants self-injected 0.1 mg/kg of zilucoplan by SC injection once daily for 12 months. From Week 2 onwards, if a participant had not achieved an adequate response, the zilucoplan dose was escalated to 0.3 mg/kg daily.
|
Zilucoplan-Cohort B (Eculizumab Switch)
n=6 Participants
Cohort B (Eculizumab Switch) included all participants from study RA101495-01.201 (NCT03078582) who had received eculizumab for treatment of PNH. Participants received a loading dose of 0.3 mg/kg zilucoplan administered by SC injection at Day 1 of this study. Following in-clinic education and training, all participants self-injected 0.1 mg/kg of zilucoplan by SC injection once daily for 12 months. From Week 2 onwards, if a participant had not achieved an adequate response, the zilucoplan dose was escalated to 0.3 mg/kg daily.
|
Zilucoplan (Inadequate Responder to Eculizumab)
n=3 Participants
Inadequate Responder cohort included participants from qualifying study RA101495-01.203 (NCT03030183) who had an inadequate response to eculizumab treatment for PNH. Participants received a loading dose of 0.3 mg/kg zilucoplan administered by SC injection at Day 1 of this study. Following in-clinic education and training, all participants self-injected 0.1 mg/kg of zilucoplan by SC injection once daily for 12 months. From Week 2 onwards, if a participant had not achieved an adequate response, the zilucoplan dose was escalated to 0.3 mg/kg daily.
|
Total
n=19 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
6 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
13 Participants
n=4 Participants
|
|
Age, Categorical
>=65 years
|
4 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
6 Participants
n=4 Participants
|
|
Age, Continuous
|
59.6 years
STANDARD_DEVIATION 14.5 • n=5 Participants
|
45.0 years
STANDARD_DEVIATION 23.0 • n=7 Participants
|
35.3 years
STANDARD_DEVIATION 16.3 • n=5 Participants
|
51.2 years
STANDARD_DEVIATION 19.4 • n=4 Participants
|
|
Sex: Female, Male
Female
|
6 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
8 Participants
n=4 Participants
|
|
Sex: Female, Male
Male
|
4 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
11 Participants
n=4 Participants
|
|
Race/Ethnicity, Customized
Black or African American
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
|
Race/Ethnicity, Customized
White
|
10 Participants
n=5 Participants
|
6 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
17 Participants
n=4 Participants
|
|
Race/Ethnicity, Customized
Not Reported
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
|
Race/Ethnicity, Customized
Not Hispanic or Latino
|
10 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
18 Participants
n=4 Participants
|
PRIMARY outcome
Timeframe: From Day 1 until the Final Study Visit (up to Month 49)Population: Safety population included all participants who received at least 1 injection of zilucoplan on or after Day 1 of this extension study.
TEAEs were defined as an AE that occurs after a participant's initial treatment zilucoplan start for this study (RA101495-01.202) that was not present at the time of treatment start, or an AE that increases in severity after treatment start in this study, if the event was present at the time of treatment start.
Outcome measures
| Measure |
Zilucoplan-Cohort A (Eculizumab Naïve)
n=10 Participants
Cohort A (Eculizumab Naive) included all participants from study RA101495-01.201 (NCT03078582) who had not received eculizumab for treatment of paroxysmal nocturnal hemoglobinuria (PNH). Participants received a loading dose of 0.3 milligram/kilogram (mg/kg) zilucoplan administered by subcutaneous (SC) injection at Day 1 of this study. Following in-clinic education and training, all participants self-injected 0.1 mg/kg of zilucoplan by SC injection once daily for 12 months. From Week 2 onwards, if a participant had not achieved an adequate response, the zilucoplan dose was escalated to 0.3 mg/kg daily.
|
Zilucoplan-Cohort B (Eculizumab Switch)
n=6 Participants
Cohort B (Eculizumab Switch) included all participants from study RA101495-01.201 (NCT03078582) who had received eculizumab for treatment of PNH. Participants received a loading dose of 0.3 mg/kg zilucoplan administered by SC injection at Day 1 of this study. Following in-clinic education and training, all participants self-injected 0.1 mg/kg of zilucoplan by SC injection once daily for 12 months. From Week 2 onwards, if a participant had not achieved an adequate response, the zilucoplan dose was escalated to 0.3 mg/kg daily.
|
Zilucoplan (Inadequate Responder to Eculizumab)
n=3 Participants
Inadequate Responder cohort included participants from qualifying study RA101495-01.203 (NCT03030183) who had an inadequate response to eculizumab treatment for PNH. Participants received a loading dose of 0.3 mg/kg zilucoplan administered by SC injection at Day 1 of this study. Following in-clinic education and training, all participants self-injected 0.1 mg/kg of zilucoplan by SC injection once daily for 12 months. From Week 2 onwards, if a participant had not achieved an adequate response, the zilucoplan dose was escalated to 0.3 mg/kg daily.
|
|---|---|---|---|
|
Percentage of Participants With Treatment Emergent Adverse Events (TEAEs)
|
90.0 percentage of participants
|
100 percentage of participants
|
66.7 percentage of participants
|
PRIMARY outcome
Timeframe: From Day 1 until the Final Study Visit (up to Month 49)Population: Safety population included all participants who received at least 1 injection of zilucoplan on or after Day 1 of this extension study.
Serious Adverse event (SAE) was defined as any untoward medical occurrence that:• results in death, • is life-threatening threatening (note that this refers to an event in which the participant was at risk of death at the time of the event; it does not refer to an event that hypothetically might have caused death if it were more severe), • requires hospitalization or prolongation of existing hospitalization, • results in persistent or significant disability/incapacity, and • results in a congenital anomaly/birth defect.
Outcome measures
| Measure |
Zilucoplan-Cohort A (Eculizumab Naïve)
n=10 Participants
Cohort A (Eculizumab Naive) included all participants from study RA101495-01.201 (NCT03078582) who had not received eculizumab for treatment of paroxysmal nocturnal hemoglobinuria (PNH). Participants received a loading dose of 0.3 milligram/kilogram (mg/kg) zilucoplan administered by subcutaneous (SC) injection at Day 1 of this study. Following in-clinic education and training, all participants self-injected 0.1 mg/kg of zilucoplan by SC injection once daily for 12 months. From Week 2 onwards, if a participant had not achieved an adequate response, the zilucoplan dose was escalated to 0.3 mg/kg daily.
|
Zilucoplan-Cohort B (Eculizumab Switch)
n=6 Participants
Cohort B (Eculizumab Switch) included all participants from study RA101495-01.201 (NCT03078582) who had received eculizumab for treatment of PNH. Participants received a loading dose of 0.3 mg/kg zilucoplan administered by SC injection at Day 1 of this study. Following in-clinic education and training, all participants self-injected 0.1 mg/kg of zilucoplan by SC injection once daily for 12 months. From Week 2 onwards, if a participant had not achieved an adequate response, the zilucoplan dose was escalated to 0.3 mg/kg daily.
|
Zilucoplan (Inadequate Responder to Eculizumab)
n=3 Participants
Inadequate Responder cohort included participants from qualifying study RA101495-01.203 (NCT03030183) who had an inadequate response to eculizumab treatment for PNH. Participants received a loading dose of 0.3 mg/kg zilucoplan administered by SC injection at Day 1 of this study. Following in-clinic education and training, all participants self-injected 0.1 mg/kg of zilucoplan by SC injection once daily for 12 months. From Week 2 onwards, if a participant had not achieved an adequate response, the zilucoplan dose was escalated to 0.3 mg/kg daily.
|
|---|---|---|---|
|
Percentage of Participants With Serious TEAEs
|
10.0 percentage of participants
|
50.0 percentage of participants
|
66.7 percentage of participants
|
SECONDARY outcome
Timeframe: At Day 1, Month 1, 2, 3, 6, 9, and 12Population: Safety population included all participants who received at least 1 injection of zilucoplan on or after Day 1 of this extension study. The planned analysis of immunogenicity (ADA) was not performed as the assay was considered not fit for purpose due to an insufficient level of drug tolerance; therefore, no samples were processed.
Blood samples collection were planned to analyze for the presence/absence of ADAs to zilucoplan for immunogenicity assessments.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Baseline, Month 1, 2, 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42, 45, and Final Study Visit (Month 49)Population: Safety population included all participants who received at least 1 injection of zilucoplan on or after Day 1 of this extension study. Baseline was generally the final visit of the qualifying study (RA101495-01.201 or RA101495-01.203). Here, Number of participants analyzed included those participants who were evaluable for the assessment and 'n' (Number analyzed) signifies participants who were evaluable at specified time points.
Serum LDH levels were measure of intravascular hemolysis. As high level of LDH in the blood was indicative of hemolysis in participants with PNH.
Outcome measures
| Measure |
Zilucoplan-Cohort A (Eculizumab Naïve)
n=9 Participants
Cohort A (Eculizumab Naive) included all participants from study RA101495-01.201 (NCT03078582) who had not received eculizumab for treatment of paroxysmal nocturnal hemoglobinuria (PNH). Participants received a loading dose of 0.3 milligram/kilogram (mg/kg) zilucoplan administered by subcutaneous (SC) injection at Day 1 of this study. Following in-clinic education and training, all participants self-injected 0.1 mg/kg of zilucoplan by SC injection once daily for 12 months. From Week 2 onwards, if a participant had not achieved an adequate response, the zilucoplan dose was escalated to 0.3 mg/kg daily.
|
Zilucoplan-Cohort B (Eculizumab Switch)
n=6 Participants
Cohort B (Eculizumab Switch) included all participants from study RA101495-01.201 (NCT03078582) who had received eculizumab for treatment of PNH. Participants received a loading dose of 0.3 mg/kg zilucoplan administered by SC injection at Day 1 of this study. Following in-clinic education and training, all participants self-injected 0.1 mg/kg of zilucoplan by SC injection once daily for 12 months. From Week 2 onwards, if a participant had not achieved an adequate response, the zilucoplan dose was escalated to 0.3 mg/kg daily.
|
Zilucoplan (Inadequate Responder to Eculizumab)
n=3 Participants
Inadequate Responder cohort included participants from qualifying study RA101495-01.203 (NCT03030183) who had an inadequate response to eculizumab treatment for PNH. Participants received a loading dose of 0.3 mg/kg zilucoplan administered by SC injection at Day 1 of this study. Following in-clinic education and training, all participants self-injected 0.1 mg/kg of zilucoplan by SC injection once daily for 12 months. From Week 2 onwards, if a participant had not achieved an adequate response, the zilucoplan dose was escalated to 0.3 mg/kg daily.
|
|---|---|---|---|
|
Change From Baseline in Serum Lactate Dehydrogenase (LDH) Levels at Each Time Point
Month 1
|
-3.7 Units per litre (U/L)
Standard Deviation 71.5
|
10.0 Units per litre (U/L)
Standard Deviation 16.4
|
336.7 Units per litre (U/L)
Standard Deviation 636.9
|
|
Change From Baseline in Serum Lactate Dehydrogenase (LDH) Levels at Each Time Point
Month 2
|
50.3 Units per litre (U/L)
Standard Deviation 156.2
|
-2.4 Units per litre (U/L)
Standard Deviation 22.2
|
676.5 Units per litre (U/L)
Standard Deviation 970.9
|
|
Change From Baseline in Serum Lactate Dehydrogenase (LDH) Levels at Each Time Point
Month 3
|
-6.5 Units per litre (U/L)
Standard Deviation 58.2
|
-1.6 Units per litre (U/L)
Standard Deviation 40.5
|
39.5 Units per litre (U/L)
Standard Deviation 40.3
|
|
Change From Baseline in Serum Lactate Dehydrogenase (LDH) Levels at Each Time Point
Month 6
|
32.4 Units per litre (U/L)
Standard Deviation 142.5
|
-28.4 Units per litre (U/L)
Standard Deviation 127.3
|
-3.5 Units per litre (U/L)
Standard Deviation 12.0
|
|
Change From Baseline in Serum Lactate Dehydrogenase (LDH) Levels at Each Time Point
Month 9
|
-18.1 Units per litre (U/L)
Standard Deviation 59.7
|
22.7 Units per litre (U/L)
Standard Deviation 23.6
|
-5.0 Units per litre (U/L)
Standard Deviation 35.4
|
|
Change From Baseline in Serum Lactate Dehydrogenase (LDH) Levels at Each Time Point
Month 12
|
-39.7 Units per litre (U/L)
Standard Deviation 54.9
|
8.7 Units per litre (U/L)
Standard Deviation 10.1
|
456.0 Units per litre (U/L)
Standard Deviation 640.6
|
|
Change From Baseline in Serum Lactate Dehydrogenase (LDH) Levels at Each Time Point
Month 15
|
-24.5 Units per litre (U/L)
Standard Deviation 70.8
|
4.0 Units per litre (U/L)
Standard Deviation 30.4
|
174.5 Units per litre (U/L)
Standard Deviation 215.7
|
|
Change From Baseline in Serum Lactate Dehydrogenase (LDH) Levels at Each Time Point
Month 18
|
-27.9 Units per litre (U/L)
Standard Deviation 54.8
|
5.7 Units per litre (U/L)
Standard Deviation 56.8
|
-56.0 Units per litre (U/L)
Standard Deviation NA
Standard Deviation (SD) could not be calculated for a single participant.
|
|
Change From Baseline in Serum Lactate Dehydrogenase (LDH) Levels at Each Time Point
Month 21
|
-12.0 Units per litre (U/L)
Standard Deviation 93.9
|
-7.3 Units per litre (U/L)
Standard Deviation 33.5
|
73.0 Units per litre (U/L)
Standard Deviation NA
SD could not be calculated for a single participant.
|
|
Change From Baseline in Serum Lactate Dehydrogenase (LDH) Levels at Each Time Point
Month 24
|
-18.7 Units per litre (U/L)
Standard Deviation 53.9
|
-27.0 Units per litre (U/L)
Standard Deviation 64.1
|
-29.0 Units per litre (U/L)
Standard Deviation NA
SD could not be calculated for a single participant.
|
|
Change From Baseline in Serum Lactate Dehydrogenase (LDH) Levels at Each Time Point
Month 27
|
-14.0 Units per litre (U/L)
Standard Deviation 99.3
|
58.0 Units per litre (U/L)
Standard Deviation 8.5
|
—
|
|
Change From Baseline in Serum Lactate Dehydrogenase (LDH) Levels at Each Time Point
Month 30
|
-28.0 Units per litre (U/L)
Standard Deviation 36.1
|
59.0 Units per litre (U/L)
Standard Deviation 140.0
|
1817.0 Units per litre (U/L)
Standard Deviation NA
SD could not be calculated for a single participant.
|
|
Change From Baseline in Serum Lactate Dehydrogenase (LDH) Levels at Each Time Point
Month 33
|
-39.0 Units per litre (U/L)
Standard Deviation 65.9
|
-16.3 Units per litre (U/L)
Standard Deviation 66.7
|
—
|
|
Change From Baseline in Serum Lactate Dehydrogenase (LDH) Levels at Each Time Point
Month 36
|
-61.6 Units per litre (U/L)
Standard Deviation 47.1
|
0.5 Units per litre (U/L)
Standard Deviation 4.9
|
—
|
|
Change From Baseline in Serum Lactate Dehydrogenase (LDH) Levels at Each Time Point
Month 39
|
-78.4 Units per litre (U/L)
Standard Deviation 80.9
|
-29.7 Units per litre (U/L)
Standard Deviation 83.5
|
—
|
|
Change From Baseline in Serum Lactate Dehydrogenase (LDH) Levels at Each Time Point
Month 42
|
-42.3 Units per litre (U/L)
Standard Deviation 78.3
|
35.5 Units per litre (U/L)
Standard Deviation 75.7
|
—
|
|
Change From Baseline in Serum Lactate Dehydrogenase (LDH) Levels at Each Time Point
Month 45
|
27.0 Units per litre (U/L)
Standard Deviation NA
SD could not be calculated for a single participant.
|
53.0 Units per litre (U/L)
Standard Deviation NA
SD could not be calculated for a single participant.
|
—
|
|
Change From Baseline in Serum Lactate Dehydrogenase (LDH) Levels at Each Time Point
Final Study Visit (Month 49)
|
41.6 Units per litre (U/L)
Standard Deviation 303.1
|
-93.2 Units per litre (U/L)
Standard Deviation 139.8
|
682.0 Units per litre (U/L)
Standard Deviation 548.7
|
SECONDARY outcome
Timeframe: Baseline, Month 1, 2, 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42, 45, and Final Study Visit (Month 49)Population: Safety population included all participants who received at least 1 injection of zilucoplan on or after Day 1 of this extension study. Baseline was generally the final visit of the qualifying study (RA101495-01.201 or RA101495-01.203). Here, Number of participants analyzed included those participants who were evaluable for the assessment and 'n' (Number analyzed) signifies participants who were evaluable at specified time points.
Total Bilirubin was monitored for signs and symptoms of hepatic or biliary dysfunction.
Outcome measures
| Measure |
Zilucoplan-Cohort A (Eculizumab Naïve)
n=9 Participants
Cohort A (Eculizumab Naive) included all participants from study RA101495-01.201 (NCT03078582) who had not received eculizumab for treatment of paroxysmal nocturnal hemoglobinuria (PNH). Participants received a loading dose of 0.3 milligram/kilogram (mg/kg) zilucoplan administered by subcutaneous (SC) injection at Day 1 of this study. Following in-clinic education and training, all participants self-injected 0.1 mg/kg of zilucoplan by SC injection once daily for 12 months. From Week 2 onwards, if a participant had not achieved an adequate response, the zilucoplan dose was escalated to 0.3 mg/kg daily.
|
Zilucoplan-Cohort B (Eculizumab Switch)
n=6 Participants
Cohort B (Eculizumab Switch) included all participants from study RA101495-01.201 (NCT03078582) who had received eculizumab for treatment of PNH. Participants received a loading dose of 0.3 mg/kg zilucoplan administered by SC injection at Day 1 of this study. Following in-clinic education and training, all participants self-injected 0.1 mg/kg of zilucoplan by SC injection once daily for 12 months. From Week 2 onwards, if a participant had not achieved an adequate response, the zilucoplan dose was escalated to 0.3 mg/kg daily.
|
Zilucoplan (Inadequate Responder to Eculizumab)
n=3 Participants
Inadequate Responder cohort included participants from qualifying study RA101495-01.203 (NCT03030183) who had an inadequate response to eculizumab treatment for PNH. Participants received a loading dose of 0.3 mg/kg zilucoplan administered by SC injection at Day 1 of this study. Following in-clinic education and training, all participants self-injected 0.1 mg/kg of zilucoplan by SC injection once daily for 12 months. From Week 2 onwards, if a participant had not achieved an adequate response, the zilucoplan dose was escalated to 0.3 mg/kg daily.
|
|---|---|---|---|
|
Change From Baseline in Total Bilirubin Values at Each Time Point
Month 1
|
-2.1 micromole per litre (umol/L)
Standard Deviation 7.7
|
1.7 micromole per litre (umol/L)
Standard Deviation 6.2
|
-0.3 micromole per litre (umol/L)
Standard Deviation 4.5
|
|
Change From Baseline in Total Bilirubin Values at Each Time Point
Month 2
|
0.1 micromole per litre (umol/L)
Standard Deviation 10.7
|
2.0 micromole per litre (umol/L)
Standard Deviation 7.2
|
6.0 micromole per litre (umol/L)
Standard Deviation 2.8
|
|
Change From Baseline in Total Bilirubin Values at Each Time Point
Month 3
|
1.4 micromole per litre (umol/L)
Standard Deviation 13.7
|
8.2 micromole per litre (umol/L)
Standard Deviation 13.1
|
3.5 micromole per litre (umol/L)
Standard Deviation 2.1
|
|
Change From Baseline in Total Bilirubin Values at Each Time Point
Month 6
|
4.6 micromole per litre (umol/L)
Standard Deviation 14.8
|
3.2 micromole per litre (umol/L)
Standard Deviation 7.2
|
2.5 micromole per litre (umol/L)
Standard Deviation 0.7
|
|
Change From Baseline in Total Bilirubin Values at Each Time Point
Month 9
|
-0.3 micromole per litre (umol/L)
Standard Deviation 7.4
|
6.0 micromole per litre (umol/L)
Standard Deviation 3.0
|
-1.0 micromole per litre (umol/L)
Standard Deviation 1.4
|
|
Change From Baseline in Total Bilirubin Values at Each Time Point
Month 12
|
3.9 micromole per litre (umol/L)
Standard Deviation 8.4
|
3.0 micromole per litre (umol/L)
Standard Deviation 2.0
|
2.5 micromole per litre (umol/L)
Standard Deviation 3.5
|
|
Change From Baseline in Total Bilirubin Values at Each Time Point
Month 15
|
-0.4 micromole per litre (umol/L)
Standard Deviation 6.4
|
6.0 micromole per litre (umol/L)
Standard Deviation 9.5
|
8.5 micromole per litre (umol/L)
Standard Deviation 9.2
|
|
Change From Baseline in Total Bilirubin Values at Each Time Point
Month 18
|
2.8 micromole per litre (umol/L)
Standard Deviation 9.5
|
2.0 micromole per litre (umol/L)
Standard Deviation 6.2
|
3.0 micromole per litre (umol/L)
Standard Deviation NA
SD could not be calculated for a single participant.
|
|
Change From Baseline in Total Bilirubin Values at Each Time Point
Month 21
|
3.9 micromole per litre (umol/L)
Standard Deviation 8.3
|
2.0 micromole per litre (umol/L)
Standard Deviation 7.8
|
7.0 micromole per litre (umol/L)
Standard Deviation NA
SD could not be calculated for a single participant.
|
|
Change From Baseline in Total Bilirubin Values at Each Time Point
Month 24
|
5.1 micromole per litre (umol/L)
Standard Deviation 16.0
|
-1.3 micromole per litre (umol/L)
Standard Deviation 3.1
|
8.0 micromole per litre (umol/L)
Standard Deviation NA
SD could not be calculated for a single participant.
|
|
Change From Baseline in Total Bilirubin Values at Each Time Point
Month 27
|
4.3 micromole per litre (umol/L)
Standard Deviation 12.2
|
-2.0 micromole per litre (umol/L)
Standard Deviation 5.7
|
—
|
|
Change From Baseline in Total Bilirubin Values at Each Time Point
Month 30
|
5.8 micromole per litre (umol/L)
Standard Deviation 14.6
|
3.0 micromole per litre (umol/L)
Standard Deviation 7.1
|
13.0 micromole per litre (umol/L)
Standard Deviation NA
SD could not be calculated for a single participant.
|
|
Change From Baseline in Total Bilirubin Values at Each Time Point
Month 33
|
2.6 micromole per litre (umol/L)
Standard Deviation 5.7
|
3.0 micromole per litre (umol/L)
Standard Deviation 2.6
|
—
|
|
Change From Baseline in Total Bilirubin Values at Each Time Point
Month 36
|
-3.8 micromole per litre (umol/L)
Standard Deviation 6.8
|
3.0 micromole per litre (umol/L)
Standard Deviation 0.0
|
—
|
|
Change From Baseline in Total Bilirubin Values at Each Time Point
Month 39
|
0.2 micromole per litre (umol/L)
Standard Deviation 3.1
|
0.7 micromole per litre (umol/L)
Standard Deviation 4.6
|
—
|
|
Change From Baseline in Total Bilirubin Values at Each Time Point
Month 42
|
4.8 micromole per litre (umol/L)
Standard Deviation 15.5
|
4.0 micromole per litre (umol/L)
Standard Deviation 15.6
|
—
|
|
Change From Baseline in Total Bilirubin Values at Each Time Point
Month 45
|
5.0 micromole per litre (umol/L)
Standard Deviation 17.0
|
-2.0 micromole per litre (umol/L)
Standard Deviation NA
SD could not be calculated for a single participant.
|
—
|
|
Change From Baseline in Total Bilirubin Values at Each Time Point
Final Study Visit (Month 49)
|
8.0 micromole per litre (umol/L)
Standard Deviation 17.3
|
0.4 micromole per litre (umol/L)
Standard Deviation 3.9
|
0.0 micromole per litre (umol/L)
Standard Deviation 9.9
|
SECONDARY outcome
Timeframe: Baseline, Month 1, 2, 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42, 45, and Final Study Visit (Month 49)Population: Safety population included all participants who received at least 1 injection of zilucoplan on or after Day 1 of this extension study. Baseline was generally the final visit of the qualifying study (RA101495-01.201 or RA101495-01.203). Here, Number of participants analyzed included those participants who were evaluable for the assessment and 'n' (Number analyzed) signifies participants who were evaluable at specified time points.
Total Hemoglobin Values were analyzed for hematology assessments.
Outcome measures
| Measure |
Zilucoplan-Cohort A (Eculizumab Naïve)
n=9 Participants
Cohort A (Eculizumab Naive) included all participants from study RA101495-01.201 (NCT03078582) who had not received eculizumab for treatment of paroxysmal nocturnal hemoglobinuria (PNH). Participants received a loading dose of 0.3 milligram/kilogram (mg/kg) zilucoplan administered by subcutaneous (SC) injection at Day 1 of this study. Following in-clinic education and training, all participants self-injected 0.1 mg/kg of zilucoplan by SC injection once daily for 12 months. From Week 2 onwards, if a participant had not achieved an adequate response, the zilucoplan dose was escalated to 0.3 mg/kg daily.
|
Zilucoplan-Cohort B (Eculizumab Switch)
n=6 Participants
Cohort B (Eculizumab Switch) included all participants from study RA101495-01.201 (NCT03078582) who had received eculizumab for treatment of PNH. Participants received a loading dose of 0.3 mg/kg zilucoplan administered by SC injection at Day 1 of this study. Following in-clinic education and training, all participants self-injected 0.1 mg/kg of zilucoplan by SC injection once daily for 12 months. From Week 2 onwards, if a participant had not achieved an adequate response, the zilucoplan dose was escalated to 0.3 mg/kg daily.
|
Zilucoplan (Inadequate Responder to Eculizumab)
n=3 Participants
Inadequate Responder cohort included participants from qualifying study RA101495-01.203 (NCT03030183) who had an inadequate response to eculizumab treatment for PNH. Participants received a loading dose of 0.3 mg/kg zilucoplan administered by SC injection at Day 1 of this study. Following in-clinic education and training, all participants self-injected 0.1 mg/kg of zilucoplan by SC injection once daily for 12 months. From Week 2 onwards, if a participant had not achieved an adequate response, the zilucoplan dose was escalated to 0.3 mg/kg daily.
|
|---|---|---|---|
|
Change From Baseline in Total Hemoglobin Values at Each Time Point
Month 1
|
0.0 grams per litre (g/L)
Standard Deviation 11.4
|
0.0 grams per litre (g/L)
Standard Deviation 8.2
|
-5.7 grams per litre (g/L)
Standard Deviation 3.8
|
|
Change From Baseline in Total Hemoglobin Values at Each Time Point
Month 2
|
1.1 grams per litre (g/L)
Standard Deviation 8.3
|
-5.0 grams per litre (g/L)
Standard Deviation 9.9
|
-2.0 grams per litre (g/L)
Standard Deviation 11.3
|
|
Change From Baseline in Total Hemoglobin Values at Each Time Point
Month 3
|
6.4 grams per litre (g/L)
Standard Deviation 6.4
|
3.0 grams per litre (g/L)
Standard Deviation 9.1
|
-3.5 grams per litre (g/L)
Standard Deviation 6.4
|
|
Change From Baseline in Total Hemoglobin Values at Each Time Point
Month 6
|
1.1 grams per litre (g/L)
Standard Deviation 11.2
|
2.0 grams per litre (g/L)
Standard Deviation 17.9
|
-5.0 grams per litre (g/L)
Standard Deviation 17.0
|
|
Change From Baseline in Total Hemoglobin Values at Each Time Point
Month 9
|
2.9 grams per litre (g/L)
Standard Deviation 7.1
|
-1.3 grams per litre (g/L)
Standard Deviation 4.0
|
-9.0 grams per litre (g/L)
Standard Deviation 5.7
|
|
Change From Baseline in Total Hemoglobin Values at Each Time Point
Month 12
|
2.9 grams per litre (g/L)
Standard Deviation 6.1
|
-1.7 grams per litre (g/L)
Standard Deviation 6.8
|
-5.0 grams per litre (g/L)
Standard Deviation 17.0
|
|
Change From Baseline in Total Hemoglobin Values at Each Time Point
Month 15
|
3.9 grams per litre (g/L)
Standard Deviation 8.4
|
-5.0 grams per litre (g/L)
Standard Deviation 5.3
|
-9.0 grams per litre (g/L)
Standard Deviation 28.3
|
|
Change From Baseline in Total Hemoglobin Values at Each Time Point
Month 18
|
5.0 grams per litre (g/L)
Standard Deviation 4.9
|
1.7 grams per litre (g/L)
Standard Deviation 9.5
|
-2.0 grams per litre (g/L)
Standard Deviation NA
SD could not be calculated for a single participant.
|
|
Change From Baseline in Total Hemoglobin Values at Each Time Point
Month 21
|
6.4 grams per litre (g/L)
Standard Deviation 6.3
|
-6.7 grams per litre (g/L)
Standard Deviation 7.6
|
-1.0 grams per litre (g/L)
Standard Deviation NA
SD could not be calculated for a single participant.
|
|
Change From Baseline in Total Hemoglobin Values at Each Time Point
Month 24
|
7.9 grams per litre (g/L)
Standard Deviation 12.6
|
-5.0 grams per litre (g/L)
Standard Deviation 2.6
|
—
|
|
Change From Baseline in Total Hemoglobin Values at Each Time Point
Month 27
|
7.0 grams per litre (g/L)
Standard Deviation 10.0
|
-5.0 grams per litre (g/L)
Standard Deviation 11.3
|
—
|
|
Change From Baseline in Total Hemoglobin Values at Each Time Point
Month 30
|
10.8 grams per litre (g/L)
Standard Deviation 7.0
|
-4.5 grams per litre (g/L)
Standard Deviation 12.0
|
-8.0 grams per litre (g/L)
Standard Deviation NA
SD could not be calculated for a single participant.
|
|
Change From Baseline in Total Hemoglobin Values at Each Time Point
Month 33
|
10.0 grams per litre (g/L)
Standard Deviation 8.8
|
-3.0 grams per litre (g/L)
Standard Deviation 4.4
|
—
|
|
Change From Baseline in Total Hemoglobin Values at Each Time Point
Month 36
|
8.4 grams per litre (g/L)
Standard Deviation 14.2
|
-2.0 grams per litre (g/L)
Standard Deviation 4.2
|
—
|
|
Change From Baseline in Total Hemoglobin Values at Each Time Point
Month 39
|
7.6 grams per litre (g/L)
Standard Deviation 10.1
|
4.7 grams per litre (g/L)
Standard Deviation 2.9
|
—
|
|
Change From Baseline in Total Hemoglobin Values at Each Time Point
Month 42
|
5.2 grams per litre (g/L)
Standard Deviation 11.2
|
5.5 grams per litre (g/L)
Standard Deviation 0.7
|
—
|
|
Change From Baseline in Total Hemoglobin Values at Each Time Point
Month 45
|
-1.3 grams per litre (g/L)
Standard Deviation 23.3
|
-4.0 grams per litre (g/L)
Standard Deviation NA
SD could not be calculated for a single participant.
|
—
|
|
Change From Baseline in Total Hemoglobin Values at Each Time Point
Final Study Visit (Month 49)
|
1.8 grams per litre (g/L)
Standard Deviation 10.6
|
-2.0 grams per litre (g/L)
Standard Deviation 4.1
|
-7.0 grams per litre (g/L)
Standard Deviation NA
SD could not be calculated for a single participant.
|
SECONDARY outcome
Timeframe: Baseline, Month 1, 2, 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42, 45, and Final Study Visit (Month 49)Population: Safety population included all participants who received at least 1 injection of zilucoplan on or after Day 1 of this extension study. Baseline was generally the final visit of the qualifying study (RA101495-01.201 or RA101495-01.203). Here, Number of participants analyzed included those participants who were evaluable for the assessment and 'n' (Number analyzed) signifies participants who were evaluable at specified time points.
Free Hemoglobin Values were analyzed for hematology assessments.
Outcome measures
| Measure |
Zilucoplan-Cohort A (Eculizumab Naïve)
n=7 Participants
Cohort A (Eculizumab Naive) included all participants from study RA101495-01.201 (NCT03078582) who had not received eculizumab for treatment of paroxysmal nocturnal hemoglobinuria (PNH). Participants received a loading dose of 0.3 milligram/kilogram (mg/kg) zilucoplan administered by subcutaneous (SC) injection at Day 1 of this study. Following in-clinic education and training, all participants self-injected 0.1 mg/kg of zilucoplan by SC injection once daily for 12 months. From Week 2 onwards, if a participant had not achieved an adequate response, the zilucoplan dose was escalated to 0.3 mg/kg daily.
|
Zilucoplan-Cohort B (Eculizumab Switch)
n=5 Participants
Cohort B (Eculizumab Switch) included all participants from study RA101495-01.201 (NCT03078582) who had received eculizumab for treatment of PNH. Participants received a loading dose of 0.3 mg/kg zilucoplan administered by SC injection at Day 1 of this study. Following in-clinic education and training, all participants self-injected 0.1 mg/kg of zilucoplan by SC injection once daily for 12 months. From Week 2 onwards, if a participant had not achieved an adequate response, the zilucoplan dose was escalated to 0.3 mg/kg daily.
|
Zilucoplan (Inadequate Responder to Eculizumab)
n=2 Participants
Inadequate Responder cohort included participants from qualifying study RA101495-01.203 (NCT03030183) who had an inadequate response to eculizumab treatment for PNH. Participants received a loading dose of 0.3 mg/kg zilucoplan administered by SC injection at Day 1 of this study. Following in-clinic education and training, all participants self-injected 0.1 mg/kg of zilucoplan by SC injection once daily for 12 months. From Week 2 onwards, if a participant had not achieved an adequate response, the zilucoplan dose was escalated to 0.3 mg/kg daily.
|
|---|---|---|---|
|
Change From Baseline in Free Hemoglobin Values at Each Time Point
Month 1
|
-0.20 milligrams per decilitre (mg/dL)
Standard Deviation 2.78
|
2.86 milligrams per decilitre (mg/dL)
Standard Deviation 4.30
|
0.80 milligrams per decilitre (mg/dL)
Standard Deviation 4.38
|
|
Change From Baseline in Free Hemoglobin Values at Each Time Point
Month 2
|
-1.14 milligrams per decilitre (mg/dL)
Standard Deviation 3.04
|
0.48 milligrams per decilitre (mg/dL)
Standard Deviation 1.77
|
6.10 milligrams per decilitre (mg/dL)
Standard Deviation NA
SD could not be calculated for a single participant.
|
|
Change From Baseline in Free Hemoglobin Values at Each Time Point
Month 3
|
1.34 milligrams per decilitre (mg/dL)
Standard Deviation 0.48
|
-0.02 milligrams per decilitre (mg/dL)
Standard Deviation 0.95
|
0.80 milligrams per decilitre (mg/dL)
Standard Deviation NA
SD could not be calculated for a single participant.
|
|
Change From Baseline in Free Hemoglobin Values at Each Time Point
Month 6
|
-0.43 milligrams per decilitre (mg/dL)
Standard Deviation 1.24
|
0.55 milligrams per decilitre (mg/dL)
Standard Deviation 2.45
|
1.20 milligrams per decilitre (mg/dL)
Standard Deviation NA
SD could not be calculated for a single participant.
|
|
Change From Baseline in Free Hemoglobin Values at Each Time Point
Month 9
|
-1.10 milligrams per decilitre (mg/dL)
Standard Deviation 2.11
|
-1.50 milligrams per decilitre (mg/dL)
Standard Deviation 1.57
|
0.00 milligrams per decilitre (mg/dL)
Standard Deviation NA
SD could not be calculated for a single participant.
|
|
Change From Baseline in Free Hemoglobin Values at Each Time Point
Month 12
|
-0.56 milligrams per decilitre (mg/dL)
Standard Deviation 2.80
|
0.80 milligrams per decilitre (mg/dL)
Standard Deviation 0.28
|
2.10 milligrams per decilitre (mg/dL)
Standard Deviation NA
SD could not be calculated for a single participant.
|
|
Change From Baseline in Free Hemoglobin Values at Each Time Point
Month 15
|
3.93 milligrams per decilitre (mg/dL)
Standard Deviation 11.61
|
-1.35 milligrams per decilitre (mg/dL)
Standard Deviation 2.19
|
1.60 milligrams per decilitre (mg/dL)
Standard Deviation NA
SD could not be calculated for a single participant.
|
|
Change From Baseline in Free Hemoglobin Values at Each Time Point
Month 18
|
1.98 milligrams per decilitre (mg/dL)
Standard Deviation 3.05
|
0.77 milligrams per decilitre (mg/dL)
Standard Deviation 1.56
|
—
|
|
Change From Baseline in Free Hemoglobin Values at Each Time Point
Month 21
|
3.15 milligrams per decilitre (mg/dL)
Standard Deviation 5.84
|
-1.30 milligrams per decilitre (mg/dL)
Standard Deviation 0.85
|
—
|
|
Change From Baseline in Free Hemoglobin Values at Each Time Point
Month 24
|
0.27 milligrams per decilitre (mg/dL)
Standard Deviation 0.35
|
-0.50 milligrams per decilitre (mg/dL)
Standard Deviation 0.99
|
—
|
|
Change From Baseline in Free Hemoglobin Values at Each Time Point
Month 30
|
0.33 milligrams per decilitre (mg/dL)
Standard Deviation 2.37
|
—
|
—
|
|
Change From Baseline in Free Hemoglobin Values at Each Time Point
Month 33
|
0.17 milligrams per decilitre (mg/dL)
Standard Deviation 2.66
|
5.80 milligrams per decilitre (mg/dL)
Standard Deviation NA
SD could not be calculated for a single participant.
|
—
|
|
Change From Baseline in Free Hemoglobin Values at Each Time Point
Month 39
|
-0.60 milligrams per decilitre (mg/dL)
Standard Deviation NA
SD could not be calculated for a single participant.
|
-0.95 milligrams per decilitre (mg/dL)
Standard Deviation 0.64
|
—
|
|
Change From Baseline in Free Hemoglobin Values at Each Time Point
Month 42
|
1.30 milligrams per decilitre (mg/dL)
Standard Deviation NA
SD could not be calculated for a single participant.
|
0.50 milligrams per decilitre (mg/dL)
Standard Deviation 3.68
|
—
|
|
Change From Baseline in Free Hemoglobin Values at Each Time Point
Final Study Visit (Month 49)
|
-2.70 milligrams per decilitre (mg/dL)
Standard Deviation NA
SD could not be calculated for a single participant.
|
-0.60 milligrams per decilitre (mg/dL)
Standard Deviation 1.13
|
-3.10 milligrams per decilitre (mg/dL)
Standard Deviation NA
SD could not be calculated for a single participant.
|
SECONDARY outcome
Timeframe: Baseline, Month 1, 2, 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42, 45, and Final Study Visit (Month 49)Population: Safety population included all participants who received at least 1 injection of zilucoplan on or after Day 1 of this extension study. Baseline was generally the final visit of the qualifying study (RA101495-01.201 or RA101495-01.203). Here, Number of participants analyzed included those participants who were evaluable for the assessment and 'n' (Number analyzed) signifies participants who were evaluable at specified time points.
Haptoglobin values were analyzed for hematology assessments.
Outcome measures
| Measure |
Zilucoplan-Cohort A (Eculizumab Naïve)
n=9 Participants
Cohort A (Eculizumab Naive) included all participants from study RA101495-01.201 (NCT03078582) who had not received eculizumab for treatment of paroxysmal nocturnal hemoglobinuria (PNH). Participants received a loading dose of 0.3 milligram/kilogram (mg/kg) zilucoplan administered by subcutaneous (SC) injection at Day 1 of this study. Following in-clinic education and training, all participants self-injected 0.1 mg/kg of zilucoplan by SC injection once daily for 12 months. From Week 2 onwards, if a participant had not achieved an adequate response, the zilucoplan dose was escalated to 0.3 mg/kg daily.
|
Zilucoplan-Cohort B (Eculizumab Switch)
n=6 Participants
Cohort B (Eculizumab Switch) included all participants from study RA101495-01.201 (NCT03078582) who had received eculizumab for treatment of PNH. Participants received a loading dose of 0.3 mg/kg zilucoplan administered by SC injection at Day 1 of this study. Following in-clinic education and training, all participants self-injected 0.1 mg/kg of zilucoplan by SC injection once daily for 12 months. From Week 2 onwards, if a participant had not achieved an adequate response, the zilucoplan dose was escalated to 0.3 mg/kg daily.
|
Zilucoplan (Inadequate Responder to Eculizumab)
n=3 Participants
Inadequate Responder cohort included participants from qualifying study RA101495-01.203 (NCT03030183) who had an inadequate response to eculizumab treatment for PNH. Participants received a loading dose of 0.3 mg/kg zilucoplan administered by SC injection at Day 1 of this study. Following in-clinic education and training, all participants self-injected 0.1 mg/kg of zilucoplan by SC injection once daily for 12 months. From Week 2 onwards, if a participant had not achieved an adequate response, the zilucoplan dose was escalated to 0.3 mg/kg daily.
|
|---|---|---|---|
|
Change From Baseline in Haptoglobin Values at Each Time Point
Month 1
|
0.032 g/L
Standard Deviation 0.097
|
0.000 g/L
Standard Deviation 0.000
|
0.000 g/L
Standard Deviation 0.000
|
|
Change From Baseline in Haptoglobin Values at Each Time Point
Month 2
|
0.053 g/L
Standard Deviation 0.160
|
0.000 g/L
Standard Deviation 0.000
|
0.000 g/L
Standard Deviation 0.000
|
|
Change From Baseline in Haptoglobin Values at Each Time Point
Month 3
|
0.021 g/L
Standard Deviation 0.060
|
0.000 g/L
Standard Deviation 0.000
|
0.000 g/L
Standard Deviation 0.000
|
|
Change From Baseline in Haptoglobin Values at Each Time Point
Month 6
|
0.031 g/L
Standard Deviation 0.088
|
0.036 g/L
Standard Deviation 0.080
|
0.020 g/L
Standard Deviation 0.028
|
|
Change From Baseline in Haptoglobin Values at Each Time Point
Month 9
|
0.008 g/L
Standard Deviation 0.021
|
0.000 g/L
Standard Deviation 0.000
|
0.000 g/L
Standard Deviation 0.000
|
|
Change From Baseline in Haptoglobin Values at Each Time Point
Month 12
|
0.000 g/L
Standard Deviation 0.000
|
0.000 g/L
Standard Deviation 0.000
|
0.000 g/L
Standard Deviation 0.000
|
|
Change From Baseline in Haptoglobin Values at Each Time Point
Month 15
|
0.000 g/L
Standard Deviation 0.000
|
0.000 g/L
Standard Deviation 0.000
|
0.000 g/L
Standard Deviation 0.000
|
|
Change From Baseline in Haptoglobin Values at Each Time Point
Month 18
|
0.000 g/L
Standard Deviation 0.000
|
0.000 g/L
Standard Deviation 0.000
|
0.000 g/L
Standard Deviation NA
SD could not be calculated for a single participant.
|
|
Change From Baseline in Haptoglobin Values at Each Time Point
Month 21
|
0.030 g/L
Standard Deviation 0.079
|
0.000 g/L
Standard Deviation 0.000
|
0.000 g/L
Standard Deviation NA
SD could not be calculated for a single participant.
|
|
Change From Baseline in Haptoglobin Values at Each Time Point
Month 24
|
0.030 g/L
Standard Deviation 0.079
|
0.000 g/L
Standard Deviation 0.000
|
0.000 g/L
Standard Deviation NA
SD could not be calculated for a single participant.
|
|
Change From Baseline in Haptoglobin Values at Each Time Point
Month 27
|
0.000 g/L
Standard Deviation 0.000
|
0.000 g/L
Standard Deviation 0.000
|
—
|
|
Change From Baseline in Haptoglobin Values at Each Time Point
Month 30
|
0.004 g/L
Standard Deviation 0.009
|
0.000 g/L
Standard Deviation 0.000
|
0.000 g/L
Standard Deviation NA
SD could not be calculated for a single participant.
|
|
Change From Baseline in Haptoglobin Values at Each Time Point
Month 33
|
0.002 g/L
Standard Deviation 0.004
|
0.000 g/L
Standard Deviation 0.000
|
—
|
|
Change From Baseline in Haptoglobin Values at Each Time Point
Month 36
|
0.036 g/L
Standard Deviation 0.080
|
0.000 g/L
Standard Deviation 0.000
|
—
|
|
Change From Baseline in Haptoglobin Values at Each Time Point
Month 39
|
0.020 g/L
Standard Deviation 0.045
|
0.000 g/L
Standard Deviation 0.000
|
—
|
|
Change From Baseline in Haptoglobin Values at Each Time Point
Month 42
|
0.034 g/L
Standard Deviation 0.076
|
0.000 g/L
Standard Deviation 0.000
|
—
|
|
Change From Baseline in Haptoglobin Values at Each Time Point
Month 45
|
0.080 g/L
Standard Deviation 0.113
|
0.000 g/L
Standard Deviation NA
SD could not be calculated for a single participant.
|
—
|
|
Change From Baseline in Haptoglobin Values at Each Time Point
Final Study Visit (Month 49)
|
0.042 g/L
Standard Deviation 0.127
|
0.006 g/L
Standard Deviation 0.013
|
0.000 g/L
Standard Deviation 0.000
|
SECONDARY outcome
Timeframe: Baseline, Month 1, 2, 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42, 45, and Final Study Visit (Month 49)Population: Safety population included all participants who received at least 1 injection of zilucoplan on or after Day 1 of this extension study. Here, Number of participants analyzed included those participants who were evaluable for the assessment and 'n' (Number analyzed) signifies participants who were evaluable at specified time points.
Reticulocytes values were analyzed for hematology assessments.
Outcome measures
| Measure |
Zilucoplan-Cohort A (Eculizumab Naïve)
n=9 Participants
Cohort A (Eculizumab Naive) included all participants from study RA101495-01.201 (NCT03078582) who had not received eculizumab for treatment of paroxysmal nocturnal hemoglobinuria (PNH). Participants received a loading dose of 0.3 milligram/kilogram (mg/kg) zilucoplan administered by subcutaneous (SC) injection at Day 1 of this study. Following in-clinic education and training, all participants self-injected 0.1 mg/kg of zilucoplan by SC injection once daily for 12 months. From Week 2 onwards, if a participant had not achieved an adequate response, the zilucoplan dose was escalated to 0.3 mg/kg daily.
|
Zilucoplan-Cohort B (Eculizumab Switch)
n=6 Participants
Cohort B (Eculizumab Switch) included all participants from study RA101495-01.201 (NCT03078582) who had received eculizumab for treatment of PNH. Participants received a loading dose of 0.3 mg/kg zilucoplan administered by SC injection at Day 1 of this study. Following in-clinic education and training, all participants self-injected 0.1 mg/kg of zilucoplan by SC injection once daily for 12 months. From Week 2 onwards, if a participant had not achieved an adequate response, the zilucoplan dose was escalated to 0.3 mg/kg daily.
|
Zilucoplan (Inadequate Responder to Eculizumab)
n=3 Participants
Inadequate Responder cohort included participants from qualifying study RA101495-01.203 (NCT03030183) who had an inadequate response to eculizumab treatment for PNH. Participants received a loading dose of 0.3 mg/kg zilucoplan administered by SC injection at Day 1 of this study. Following in-clinic education and training, all participants self-injected 0.1 mg/kg of zilucoplan by SC injection once daily for 12 months. From Week 2 onwards, if a participant had not achieved an adequate response, the zilucoplan dose was escalated to 0.3 mg/kg daily.
|
|---|---|---|---|
|
Change From Baseline in Reticulocytes at Each Time Point
Month 1
|
0.0104 10^12 reticulocytes (cells)/L
Standard Deviation 0.0484
|
0.0107 10^12 reticulocytes (cells)/L
Standard Deviation 0.0299
|
-0.0457 10^12 reticulocytes (cells)/L
Standard Deviation 0.0505
|
|
Change From Baseline in Reticulocytes at Each Time Point
Month 2
|
-0.0046 10^12 reticulocytes (cells)/L
Standard Deviation 0.0354
|
-0.0046 10^12 reticulocytes (cells)/L
Standard Deviation 0.0130
|
0.0115 10^12 reticulocytes (cells)/L
Standard Deviation 0.0049
|
|
Change From Baseline in Reticulocytes at Each Time Point
Month 3
|
-0.0249 10^12 reticulocytes (cells)/L
Standard Deviation 0.0310
|
-0.0112 10^12 reticulocytes (cells)/L
Standard Deviation 0.0251
|
0.0170 10^12 reticulocytes (cells)/L
Standard Deviation 0.0113
|
|
Change From Baseline in Reticulocytes at Each Time Point
Month 6
|
-0.0006 10^12 reticulocytes (cells)/L
Standard Deviation 0.0452
|
-0.0042 10^12 reticulocytes (cells)/L
Standard Deviation 0.0363
|
0.0200 10^12 reticulocytes (cells)/L
Standard Deviation 0.0382
|
|
Change From Baseline in Reticulocytes at Each Time Point
Month 9
|
0.0063 10^12 reticulocytes (cells)/L
Standard Deviation 0.0334
|
0.0070 10^12 reticulocytes (cells)/L
Standard Deviation 0.0171
|
0.0640 10^12 reticulocytes (cells)/L
Standard Deviation 0.0297
|
|
Change From Baseline in Reticulocytes at Each Time Point
Month 12
|
-0.0041 10^12 reticulocytes (cells)/L
Standard Deviation 0.0427
|
-0.0070 10^12 reticulocytes (cells)/L
Standard Deviation 0.0346
|
0.0240 10^12 reticulocytes (cells)/L
Standard Deviation 0.0113
|
|
Change From Baseline in Reticulocytes at Each Time Point
Month 15
|
-0.0264 10^12 reticulocytes (cells)/L
Standard Deviation 0.0601
|
-0.0343 10^12 reticulocytes (cells)/L
Standard Deviation 0.0191
|
0.0285 10^12 reticulocytes (cells)/L
Standard Deviation 0.0290
|
|
Change From Baseline in Reticulocytes at Each Time Point
Month 18
|
-0.0040 10^12 reticulocytes (cells)/L
Standard Deviation 0.0382
|
0.0027 10^12 reticulocytes (cells)/L
Standard Deviation 0.0380
|
0.0460 10^12 reticulocytes (cells)/L
Standard Deviation NA
SD could not be calculated for a single participant.
|
|
Change From Baseline in Reticulocytes at Each Time Point
Month 21
|
-0.0223 10^12 reticulocytes (cells)/L
Standard Deviation 0.0554
|
-0.0087 10^12 reticulocytes (cells)/L
Standard Deviation 0.0380
|
—
|
|
Change From Baseline in Reticulocytes at Each Time Point
Month 24
|
-0.0123 10^12 reticulocytes (cells)/L
Standard Deviation 0.0711
|
-0.0093 10^12 reticulocytes (cells)/L
Standard Deviation 0.0186
|
—
|
|
Change From Baseline in Reticulocytes at Each Time Point
Month 27
|
-0.0017 10^12 reticulocytes (cells)/L
Standard Deviation 0.0553
|
0.0150 10^12 reticulocytes (cells)/L
Standard Deviation 0.0127
|
—
|
|
Change From Baseline in Reticulocytes at Each Time Point
Month 30
|
0.0016 10^12 reticulocytes (cells)/L
Standard Deviation 0.0524
|
0.0060 10^12 reticulocytes (cells)/L
Standard Deviation 0.0594
|
0.0300 10^12 reticulocytes (cells)/L
Standard Deviation NA
SD could not be calculated for a single participant.
|
|
Change From Baseline in Reticulocytes at Each Time Point
Month 33
|
-0.0302 10^12 reticulocytes (cells)/L
Standard Deviation 0.0446
|
-0.0093 10^12 reticulocytes (cells)/L
Standard Deviation 0.0234
|
—
|
|
Change From Baseline in Reticulocytes at Each Time Point
Month 36
|
-0.0630 10^12 reticulocytes (cells)/L
Standard Deviation 0.0621
|
-0.0215 10^12 reticulocytes (cells)/L
Standard Deviation 0.0078
|
—
|
|
Change From Baseline in Reticulocytes at Each Time Point
Month 39
|
-0.0340 10^12 reticulocytes (cells)/L
Standard Deviation 0.0860
|
-0.0133 10^12 reticulocytes (cells)/L
Standard Deviation 0.0291
|
—
|
|
Change From Baseline in Reticulocytes at Each Time Point
Month 42
|
-0.0270 10^12 reticulocytes (cells)/L
Standard Deviation 0.0721
|
-0.0015 10^12 reticulocytes (cells)/L
Standard Deviation 0.0078
|
—
|
|
Change From Baseline in Reticulocytes at Each Time Point
Month 45
|
-0.0423 10^12 reticulocytes (cells)/L
Standard Deviation 0.1189
|
-0.0050 10^12 reticulocytes (cells)/L
Standard Deviation NA
SD could not be calculated for a single participant.
|
—
|
|
Change From Baseline in Reticulocytes at Each Time Point
Final Study Visit (Month 49)
|
-0.0433 10^12 reticulocytes (cells)/L
Standard Deviation 0.0480
|
-0.0333 10^12 reticulocytes (cells)/L
Standard Deviation 0.0116
|
0.0100 10^12 reticulocytes (cells)/L
Standard Deviation NA
SD could not be calculated for a single participant.
|
SECONDARY outcome
Timeframe: Baseline, Month 1, 2, 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42, 45, 48 and Final Study Visit (Month 49)Population: Safety population included all participants who received at least 1 injection of zilucoplan on or after Day 1 of this extension study. Baseline was generally the final visit of the qualifying study (RA101495-01.201 or RA101495-01.203). Here, Number of participants analyzed included those participants who were evaluable for the assessment and 'n' (Number analyzed) signifies participants who were evaluable at specified time points.
Hemoglobinuria was assessed using a urine colorimetric scoring system with a score of 1 through 10 where 1 represents no hemoglobinuria and 10 represents maximum hemoglobinuria.
Outcome measures
| Measure |
Zilucoplan-Cohort A (Eculizumab Naïve)
n=9 Participants
Cohort A (Eculizumab Naive) included all participants from study RA101495-01.201 (NCT03078582) who had not received eculizumab for treatment of paroxysmal nocturnal hemoglobinuria (PNH). Participants received a loading dose of 0.3 milligram/kilogram (mg/kg) zilucoplan administered by subcutaneous (SC) injection at Day 1 of this study. Following in-clinic education and training, all participants self-injected 0.1 mg/kg of zilucoplan by SC injection once daily for 12 months. From Week 2 onwards, if a participant had not achieved an adequate response, the zilucoplan dose was escalated to 0.3 mg/kg daily.
|
Zilucoplan-Cohort B (Eculizumab Switch)
n=6 Participants
Cohort B (Eculizumab Switch) included all participants from study RA101495-01.201 (NCT03078582) who had received eculizumab for treatment of PNH. Participants received a loading dose of 0.3 mg/kg zilucoplan administered by SC injection at Day 1 of this study. Following in-clinic education and training, all participants self-injected 0.1 mg/kg of zilucoplan by SC injection once daily for 12 months. From Week 2 onwards, if a participant had not achieved an adequate response, the zilucoplan dose was escalated to 0.3 mg/kg daily.
|
Zilucoplan (Inadequate Responder to Eculizumab)
n=3 Participants
Inadequate Responder cohort included participants from qualifying study RA101495-01.203 (NCT03030183) who had an inadequate response to eculizumab treatment for PNH. Participants received a loading dose of 0.3 mg/kg zilucoplan administered by SC injection at Day 1 of this study. Following in-clinic education and training, all participants self-injected 0.1 mg/kg of zilucoplan by SC injection once daily for 12 months. From Week 2 onwards, if a participant had not achieved an adequate response, the zilucoplan dose was escalated to 0.3 mg/kg daily.
|
|---|---|---|---|
|
Change From Baseline in Hemoglobinuria Values at Each Time Point
Month 1
|
0.1 score on a scale
Standard Deviation 1.0
|
0.2 score on a scale
Standard Deviation 1.5
|
1.7 score on a scale
Standard Deviation 1.5
|
|
Change From Baseline in Hemoglobinuria Values at Each Time Point
Month 2
|
0.9 score on a scale
Standard Deviation 1.4
|
0.8 score on a scale
Standard Deviation 1.3
|
1.0 score on a scale
Standard Deviation 1.4
|
|
Change From Baseline in Hemoglobinuria Values at Each Time Point
Month 3
|
0.4 score on a scale
Standard Deviation 1.2
|
0.8 score on a scale
Standard Deviation 1.3
|
0.0 score on a scale
Standard Deviation NA
SD could not be calculated for a single participant.
|
|
Change From Baseline in Hemoglobinuria Values at Each Time Point
Month 6
|
0.4 score on a scale
Standard Deviation 0.9
|
0.8 score on a scale
Standard Deviation 1.3
|
0.0 score on a scale
Standard Deviation NA
SD could not be calculated for a single participant.
|
|
Change From Baseline in Hemoglobinuria Values at Each Time Point
Month 9
|
0.4 score on a scale
Standard Deviation 0.9
|
1.5 score on a scale
Standard Deviation 2.1
|
0.0 score on a scale
Standard Deviation NA
SD could not be calculated for a single participant.
|
|
Change From Baseline in Hemoglobinuria Values at Each Time Point
Month 12
|
0.9 score on a scale
Standard Deviation 1.4
|
1.0 score on a scale
Standard Deviation 1.7
|
1.0 score on a scale
Standard Deviation 1.4
|
|
Change From Baseline in Hemoglobinuria Values at Each Time Point
Month 15
|
0.9 score on a scale
Standard Deviation 1.1
|
1.0 score on a scale
Standard Deviation 1.7
|
1.0 score on a scale
Standard Deviation 1.4
|
|
Change From Baseline in Hemoglobinuria Values at Each Time Point
Month 18
|
0.1 score on a scale
Standard Deviation 0.9
|
0.3 score on a scale
Standard Deviation 0.6
|
0.0 score on a scale
Standard Deviation NA
SD could not be calculated for a single participant.
|
|
Change From Baseline in Hemoglobinuria Values at Each Time Point
Month 21
|
0.7 score on a scale
Standard Deviation 1.6
|
1.0 score on a scale
Standard Deviation 1.7
|
0.0 score on a scale
Standard Deviation NA
SD could not be calculated for a single participant.
|
|
Change From Baseline in Hemoglobinuria Values at Each Time Point
Month 24
|
0.6 score on a scale
Standard Deviation 1.6
|
0.3 score on a scale
Standard Deviation 0.6
|
0.0 score on a scale
Standard Deviation NA
SD could not be calculated for a single participant.
|
|
Change From Baseline in Hemoglobinuria Values at Each Time Point
Month 27
|
0.7 score on a scale
Standard Deviation 1.5
|
1.5 score on a scale
Standard Deviation 2.1
|
0.0 score on a scale
Standard Deviation NA
SD could not be calculated for a single participant.
|
|
Change From Baseline in Hemoglobinuria Values at Each Time Point
Month 30
|
0.6 score on a scale
Standard Deviation 1.3
|
1.5 score on a scale
Standard Deviation 2.1
|
0.0 score on a scale
Standard Deviation NA
SD could not be calculated for a single participant.
|
|
Change From Baseline in Hemoglobinuria Values at Each Time Point
Month 33
|
1.4 score on a scale
Standard Deviation 0.9
|
1.0 score on a scale
Standard Deviation 1.7
|
—
|
|
Change From Baseline in Hemoglobinuria Values at Each Time Point
Month 36
|
1.4 score on a scale
Standard Deviation 1.9
|
1.5 score on a scale
Standard Deviation 2.1
|
—
|
|
Change From Baseline in Hemoglobinuria Values at Each Time Point
Month 39
|
0.4 score on a scale
Standard Deviation 1.7
|
1.0 score on a scale
Standard Deviation 1.7
|
—
|
|
Change From Baseline in Hemoglobinuria Values at Each Time Point
Month 42
|
1.0 score on a scale
Standard Deviation 1.2
|
1.5 score on a scale
Standard Deviation 2.1
|
—
|
|
Change From Baseline in Hemoglobinuria Values at Each Time Point
Month 45
|
1.0 score on a scale
Standard Deviation 1.0
|
-1.0 score on a scale
Standard Deviation NA
SD could not be calculated for a single participant.
|
—
|
|
Change From Baseline in Hemoglobinuria Values at Each Time Point
Final Study Visit (Month 49)
|
0.4 score on a scale
Standard Deviation 1.1
|
0.6 score on a scale
Standard Deviation 0.9
|
2.5 score on a scale
Standard Deviation 3.5
|
SECONDARY outcome
Timeframe: Predose: At Day 1 (Screening), Month 1, 2, 3, 6, 9, 12, and Final Study Visit (Month 49)Population: Pharmacokinetic (PK) population included all participants in the Safety population who had at least 1 plasma sample obtained for PK assessment. Here, 'n' (Number analyzed) signifies participants who were evaluable at specified time points.
Blood samples of RA101495 (zilucoplan) and its metabolites (RA102758 and RA103488) were collected for Plasma concentration analysis.
Outcome measures
| Measure |
Zilucoplan-Cohort A (Eculizumab Naïve)
n=9 Participants
Cohort A (Eculizumab Naive) included all participants from study RA101495-01.201 (NCT03078582) who had not received eculizumab for treatment of paroxysmal nocturnal hemoglobinuria (PNH). Participants received a loading dose of 0.3 milligram/kilogram (mg/kg) zilucoplan administered by subcutaneous (SC) injection at Day 1 of this study. Following in-clinic education and training, all participants self-injected 0.1 mg/kg of zilucoplan by SC injection once daily for 12 months. From Week 2 onwards, if a participant had not achieved an adequate response, the zilucoplan dose was escalated to 0.3 mg/kg daily.
|
Zilucoplan-Cohort B (Eculizumab Switch)
n=6 Participants
Cohort B (Eculizumab Switch) included all participants from study RA101495-01.201 (NCT03078582) who had received eculizumab for treatment of PNH. Participants received a loading dose of 0.3 mg/kg zilucoplan administered by SC injection at Day 1 of this study. Following in-clinic education and training, all participants self-injected 0.1 mg/kg of zilucoplan by SC injection once daily for 12 months. From Week 2 onwards, if a participant had not achieved an adequate response, the zilucoplan dose was escalated to 0.3 mg/kg daily.
|
Zilucoplan (Inadequate Responder to Eculizumab)
n=3 Participants
Inadequate Responder cohort included participants from qualifying study RA101495-01.203 (NCT03030183) who had an inadequate response to eculizumab treatment for PNH. Participants received a loading dose of 0.3 mg/kg zilucoplan administered by SC injection at Day 1 of this study. Following in-clinic education and training, all participants self-injected 0.1 mg/kg of zilucoplan by SC injection once daily for 12 months. From Week 2 onwards, if a participant had not achieved an adequate response, the zilucoplan dose was escalated to 0.3 mg/kg daily.
|
|---|---|---|---|
|
Plasma Concentrations of RA101495 and Its Major Metabolite(s)
RA101495- Day 1
|
10999.99 micrograms per litre (ug/L)
Standard Deviation 2698.29
|
13616.08 micrograms per litre (ug/L)
Standard Deviation 1490.81
|
6976.50 micrograms per litre (ug/L)
Standard Deviation 5663.23
|
|
Plasma Concentrations of RA101495 and Its Major Metabolite(s)
RA101495- Month 1
|
10879.13 micrograms per litre (ug/L)
Standard Deviation 2405.19
|
12645.38 micrograms per litre (ug/L)
Standard Deviation 1620.31
|
7630.03 micrograms per litre (ug/L)
Standard Deviation 7060.56
|
|
Plasma Concentrations of RA101495 and Its Major Metabolite(s)
RA101495- Month 2
|
11276.17 micrograms per litre (ug/L)
Standard Deviation 2896.98
|
12220.10 micrograms per litre (ug/L)
Standard Deviation 2188.78
|
6865.45 micrograms per litre (ug/L)
Standard Deviation 8108.18
|
|
Plasma Concentrations of RA101495 and Its Major Metabolite(s)
RA101495- Month 3
|
10806.17 micrograms per litre (ug/L)
Standard Deviation 3418.92
|
12315.28 micrograms per litre (ug/L)
Standard Deviation 2515.84
|
12430.00 micrograms per litre (ug/L)
Standard Deviation 16.55
|
|
Plasma Concentrations of RA101495 and Its Major Metabolite(s)
RA101495- Month 6
|
9393.42 micrograms per litre (ug/L)
Standard Deviation 2224.52
|
12218.72 micrograms per litre (ug/L)
Standard Deviation 2166.05
|
12254.00 micrograms per litre (ug/L)
Standard Deviation NA
SD could not be calculated for a single participant.
|
|
Plasma Concentrations of RA101495 and Its Major Metabolite(s)
RA101495- Month 9
|
11848.67 micrograms per litre (ug/L)
Standard Deviation 2685.92
|
12282.83 micrograms per litre (ug/L)
Standard Deviation 3064.42
|
15193.35 micrograms per litre (ug/L)
Standard Deviation 2756.66
|
|
Plasma Concentrations of RA101495 and Its Major Metabolite(s)
RA101495- Month 12
|
11495.99 micrograms per litre (ug/L)
Standard Deviation 2885.81
|
13279.97 micrograms per litre (ug/L)
Standard Deviation 2148.60
|
12038.65 micrograms per litre (ug/L)
Standard Deviation 7934.94
|
|
Plasma Concentrations of RA101495 and Its Major Metabolite(s)
RA101495- Final Study Visit (Month 49)
|
—
|
—
|
7174.20 micrograms per litre (ug/L)
Standard Deviation NA
SD could not be calculated for a single participant.
|
|
Plasma Concentrations of RA101495 and Its Major Metabolite(s)
RA102758- Day 1
|
1883.49 micrograms per litre (ug/L)
Standard Deviation 593.93
|
2443.62 micrograms per litre (ug/L)
Standard Deviation 560.97
|
1280.07 micrograms per litre (ug/L)
Standard Deviation 1109.03
|
|
Plasma Concentrations of RA101495 and Its Major Metabolite(s)
RA102758- Month 1
|
1865.06 micrograms per litre (ug/L)
Standard Deviation 594.31
|
2185.00 micrograms per litre (ug/L)
Standard Deviation 631.37
|
757.30 micrograms per litre (ug/L)
Standard Deviation 997.44
|
|
Plasma Concentrations of RA101495 and Its Major Metabolite(s)
RA102758- Month 2
|
1894.86 micrograms per litre (ug/L)
Standard Deviation 608.46
|
2090.04 micrograms per litre (ug/L)
Standard Deviation 831.80
|
757.85 micrograms per litre (ug/L)
Standard Deviation 1018.59
|
|
Plasma Concentrations of RA101495 and Its Major Metabolite(s)
RA102758- Month 3
|
1758.55 micrograms per litre (ug/L)
Standard Deviation 689.03
|
1990.54 micrograms per litre (ug/L)
Standard Deviation 700.70
|
1954.05 micrograms per litre (ug/L)
Standard Deviation 160.58
|
|
Plasma Concentrations of RA101495 and Its Major Metabolite(s)
RA102758- Month 6
|
1467.85 micrograms per litre (ug/L)
Standard Deviation 563.68
|
1819.20 micrograms per litre (ug/L)
Standard Deviation 613.14
|
1725.50 micrograms per litre (ug/L)
Standard Deviation NA
SD could not be calculated for a single participant.
|
|
Plasma Concentrations of RA101495 and Its Major Metabolite(s)
RA102758- Month 9
|
1686.80 micrograms per litre (ug/L)
Standard Deviation 554.87
|
1881.73 micrograms per litre (ug/L)
Standard Deviation 583.87
|
1238.55 micrograms per litre (ug/L)
Standard Deviation 166.24
|
|
Plasma Concentrations of RA101495 and Its Major Metabolite(s)
RA102758- Month 12
|
1646.99 micrograms per litre (ug/L)
Standard Deviation 536.00
|
1954.23 micrograms per litre (ug/L)
Standard Deviation 725.54
|
10.00 micrograms per litre (ug/L)
Standard Deviation NA
SD could not be calculated for a single participant.
|
|
Plasma Concentrations of RA101495 and Its Major Metabolite(s)
RA102758- Final Study Visit (Month 49)
|
—
|
—
|
1094.60 micrograms per litre (ug/L)
Standard Deviation NA
SD could not be calculated for a single participant.
|
|
Plasma Concentrations of RA101495 and Its Major Metabolite(s)
RA103488- Day 1
|
3587.68 micrograms per litre (ug/L)
Standard Deviation 1747.12
|
4887.98 micrograms per litre (ug/L)
Standard Deviation 1969.22
|
2084.83 micrograms per litre (ug/L)
Standard Deviation 1791.35
|
|
Plasma Concentrations of RA101495 and Its Major Metabolite(s)
RA103488- Month 1
|
4344.99 micrograms per litre (ug/L)
Standard Deviation 2319.10
|
4400.68 micrograms per litre (ug/L)
Standard Deviation 1823.93
|
1703.73 micrograms per litre (ug/L)
Standard Deviation 1605.96
|
|
Plasma Concentrations of RA101495 and Its Major Metabolite(s)
RA103488- Month 2
|
4799.29 micrograms per litre (ug/L)
Standard Deviation 3113.57
|
4365.84 micrograms per litre (ug/L)
Standard Deviation 1537.67
|
1591.35 micrograms per litre (ug/L)
Standard Deviation 1552.03
|
|
Plasma Concentrations of RA101495 and Its Major Metabolite(s)
RA103488- Month 3
|
4191.36 micrograms per litre (ug/L)
Standard Deviation 2347.65
|
4541.78 micrograms per litre (ug/L)
Standard Deviation 1706.07
|
2715.60 micrograms per litre (ug/L)
Standard Deviation 1345.48
|
|
Plasma Concentrations of RA101495 and Its Major Metabolite(s)
RA103488- Month 6
|
4027.53 micrograms per litre (ug/L)
Standard Deviation 2730.92
|
4436.46 micrograms per litre (ug/L)
Standard Deviation 2341.16
|
1929.60 micrograms per litre (ug/L)
Standard Deviation NA
SD could not be calculated for a single participant.
|
|
Plasma Concentrations of RA101495 and Its Major Metabolite(s)
RA103488- Month 9
|
3132.54 micrograms per litre (ug/L)
Standard Deviation 1320.61
|
4217.97 micrograms per litre (ug/L)
Standard Deviation 2015.36
|
2954.85 micrograms per litre (ug/L)
Standard Deviation 1754.97
|
|
Plasma Concentrations of RA101495 and Its Major Metabolite(s)
RA103488- Month 12
|
3903.41 micrograms per litre (ug/L)
Standard Deviation 1960.30
|
4334.47 micrograms per litre (ug/L)
Standard Deviation 1976.47
|
2319.20 micrograms per litre (ug/L)
Standard Deviation 2534.84
|
|
Plasma Concentrations of RA101495 and Its Major Metabolite(s)
RA103488- Final Study Visit (Month 49)
|
—
|
—
|
2210.00 micrograms per litre (ug/L)
Standard Deviation NA
SD could not be calculated for a single participant.
|
SECONDARY outcome
Timeframe: At Day 1, Month 1, 2, 3, 6, 9, and 12Population: PK population included all participants in the Safety population who had at least 1 plasma sample obtained for PK assessment. Data was not collected and analyzed for this outcome measure due to change in planned analysis.
Cmax is the maximum plasma concentration.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: At Day 1, Month 1, 2, 3, 6, 9, and 12Population: PK population included all participants in the Safety population who had at least 1 plasma sample obtained for PK assessment. Data was not collected and analyzed for this outcome measure due to change in planned analysis.
tmax is the time to corresponding Cmax.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: At Day 1, Month 1, 2, 3, 6, 9, and 12Population: PK population included all participants in the Safety population who had at least 1 plasma sample obtained for PK assessment. As per the change in planned analyses, samples/data were not collected, and AUC0-t not calculated.
AUC0-t is area under the drug concentration-time curves.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: At Day 1, Month 1, 2, 3, 6, 9, and 12Population: Pharmacodynamic (PD) population included all participants in the Safety population who had at least 1 plasma sample obtained for PD assessment. Data was not collected and analyzed for this outcome measure due to change in planned analysis.
Blood samples collection were planned to assess complement (CH50) levels. The planned analysis of CH50 was not performed because the CH50 assay was not able to be validated due to lack of reproducibility of the manufacturer's kits.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Baseline, Month 1, 2, 3, 6, 9, 12 and Final Study Visit (Month 49)Population: PD population included all participants in the Safety population who had at least 1 plasma sample obtained for PD assessment. Here, Number of participants analyzed included those participants who were evaluable for the assessment and 'n' (Number analyzed) signifies participants who were evaluable at specified time points.
Blood samples were collected for measurement of sRBC lysis for the Classical Complement Pathways.
Outcome measures
| Measure |
Zilucoplan-Cohort A (Eculizumab Naïve)
n=9 Participants
Cohort A (Eculizumab Naive) included all participants from study RA101495-01.201 (NCT03078582) who had not received eculizumab for treatment of paroxysmal nocturnal hemoglobinuria (PNH). Participants received a loading dose of 0.3 milligram/kilogram (mg/kg) zilucoplan administered by subcutaneous (SC) injection at Day 1 of this study. Following in-clinic education and training, all participants self-injected 0.1 mg/kg of zilucoplan by SC injection once daily for 12 months. From Week 2 onwards, if a participant had not achieved an adequate response, the zilucoplan dose was escalated to 0.3 mg/kg daily.
|
Zilucoplan-Cohort B (Eculizumab Switch)
n=6 Participants
Cohort B (Eculizumab Switch) included all participants from study RA101495-01.201 (NCT03078582) who had received eculizumab for treatment of PNH. Participants received a loading dose of 0.3 mg/kg zilucoplan administered by SC injection at Day 1 of this study. Following in-clinic education and training, all participants self-injected 0.1 mg/kg of zilucoplan by SC injection once daily for 12 months. From Week 2 onwards, if a participant had not achieved an adequate response, the zilucoplan dose was escalated to 0.3 mg/kg daily.
|
Zilucoplan (Inadequate Responder to Eculizumab)
n=1 Participants
Inadequate Responder cohort included participants from qualifying study RA101495-01.203 (NCT03030183) who had an inadequate response to eculizumab treatment for PNH. Participants received a loading dose of 0.3 mg/kg zilucoplan administered by SC injection at Day 1 of this study. Following in-clinic education and training, all participants self-injected 0.1 mg/kg of zilucoplan by SC injection once daily for 12 months. From Week 2 onwards, if a participant had not achieved an adequate response, the zilucoplan dose was escalated to 0.3 mg/kg daily.
|
|---|---|---|---|
|
Change From Baseline in Sheep Red Blood Cell (sRBC) Values at Each Time Point
Month 1
|
1.378 percent lysis of sheep erythrocytes
Standard Deviation 2.277
|
0.523 percent lysis of sheep erythrocytes
Standard Deviation 0.772
|
93.870 percent lysis of sheep erythrocytes
Standard Deviation NA
SD could not be calculated for a single participant.
|
|
Change From Baseline in Sheep Red Blood Cell (sRBC) Values at Each Time Point
Month 2
|
1.248 percent lysis of sheep erythrocytes
Standard Deviation 1.789
|
-0.308 percent lysis of sheep erythrocytes
Standard Deviation 0.452
|
93.870 percent lysis of sheep erythrocytes
Standard Deviation NA
SD could not be calculated for a single participant.
|
|
Change From Baseline in Sheep Red Blood Cell (sRBC) Values at Each Time Point
Month 3
|
1.256 percent lysis of sheep erythrocytes
Standard Deviation 1.799
|
0.576 percent lysis of sheep erythrocytes
Standard Deviation 0.972
|
-2.620 percent lysis of sheep erythrocytes
Standard Deviation NA
SD could not be calculated for a single participant.
|
|
Change From Baseline in Sheep Red Blood Cell (sRBC) Values at Each Time Point
Month 6
|
1.493 percent lysis of sheep erythrocytes
Standard Deviation 2.838
|
0.458 percent lysis of sheep erythrocytes
Standard Deviation 1.657
|
-2.300 percent lysis of sheep erythrocytes
Standard Deviation NA
SD could not be calculated for a single participant.
|
|
Change From Baseline in Sheep Red Blood Cell (sRBC) Values at Each Time Point
Month 9
|
0.683 percent lysis of sheep erythrocytes
Standard Deviation 1.055
|
0.757 percent lysis of sheep erythrocytes
Standard Deviation 0.673
|
-1.410 percent lysis of sheep erythrocytes
Standard Deviation NA
SD could not be calculated for a single participant.
|
|
Change From Baseline in Sheep Red Blood Cell (sRBC) Values at Each Time Point
Month 12
|
0.759 percent lysis of sheep erythrocytes
Standard Deviation 0.557
|
0.773 percent lysis of sheep erythrocytes
Standard Deviation 0.415
|
8.390 percent lysis of sheep erythrocytes
Standard Deviation NA
SD could not be calculated for a single participant.
|
|
Change From Baseline in Sheep Red Blood Cell (sRBC) Values at Each Time Point
Final Study Visit (Month 49)
|
—
|
25.230 percent lysis of sheep erythrocytes
Standard Deviation 35.200
|
—
|
SECONDARY outcome
Timeframe: Baseline, Month 1, 2, 3, 6, 9, 12 and Final Study Visit (Month 49)Population: PD population included all participants in the Safety population who had at least 1 plasma sample obtained for PD assessment. Here, Number of participants analyzed included those participants who were evaluable for the assessment and 'n' (Number analyzed) signifies participants who were evaluable at specified time points.
Blood samples were collected for measurement of membrane attack complex (MAC) by Wieslab ELISA for alternative complement pathway.
Outcome measures
| Measure |
Zilucoplan-Cohort A (Eculizumab Naïve)
n=9 Participants
Cohort A (Eculizumab Naive) included all participants from study RA101495-01.201 (NCT03078582) who had not received eculizumab for treatment of paroxysmal nocturnal hemoglobinuria (PNH). Participants received a loading dose of 0.3 milligram/kilogram (mg/kg) zilucoplan administered by subcutaneous (SC) injection at Day 1 of this study. Following in-clinic education and training, all participants self-injected 0.1 mg/kg of zilucoplan by SC injection once daily for 12 months. From Week 2 onwards, if a participant had not achieved an adequate response, the zilucoplan dose was escalated to 0.3 mg/kg daily.
|
Zilucoplan-Cohort B (Eculizumab Switch)
n=6 Participants
Cohort B (Eculizumab Switch) included all participants from study RA101495-01.201 (NCT03078582) who had received eculizumab for treatment of PNH. Participants received a loading dose of 0.3 mg/kg zilucoplan administered by SC injection at Day 1 of this study. Following in-clinic education and training, all participants self-injected 0.1 mg/kg of zilucoplan by SC injection once daily for 12 months. From Week 2 onwards, if a participant had not achieved an adequate response, the zilucoplan dose was escalated to 0.3 mg/kg daily.
|
Zilucoplan (Inadequate Responder to Eculizumab)
n=2 Participants
Inadequate Responder cohort included participants from qualifying study RA101495-01.203 (NCT03030183) who had an inadequate response to eculizumab treatment for PNH. Participants received a loading dose of 0.3 mg/kg zilucoplan administered by SC injection at Day 1 of this study. Following in-clinic education and training, all participants self-injected 0.1 mg/kg of zilucoplan by SC injection once daily for 12 months. From Week 2 onwards, if a participant had not achieved an adequate response, the zilucoplan dose was escalated to 0.3 mg/kg daily.
|
|---|---|---|---|
|
Change From Baseline in Wieslab Enzyme-linked Immunosorbent Assay (ELISA) Values for Alternative Complement Pathway at Each Time Point
Month 1
|
1.7 percentage of activity
Standard Deviation 5.8
|
1.0 percentage of activity
Standard Deviation 1.7
|
48.0 percentage of activity
Standard Deviation 67.9
|
|
Change From Baseline in Wieslab Enzyme-linked Immunosorbent Assay (ELISA) Values for Alternative Complement Pathway at Each Time Point
Month 2
|
0.1 percentage of activity
Standard Deviation 1.9
|
-1.0 percentage of activity
Standard Deviation 1.0
|
51.5 percentage of activity
Standard Deviation 74.2
|
|
Change From Baseline in Wieslab Enzyme-linked Immunosorbent Assay (ELISA) Values for Alternative Complement Pathway at Each Time Point
Month 3
|
0.1 percentage of activity
Standard Deviation 2.0
|
0.0 percentage of activity
Standard Deviation 0.7
|
2.0 percentage of activity
Standard Deviation 1.4
|
|
Change From Baseline in Wieslab Enzyme-linked Immunosorbent Assay (ELISA) Values for Alternative Complement Pathway at Each Time Point
Month 6
|
1.0 percentage of activity
Standard Deviation 4.5
|
-0.2 percentage of activity
Standard Deviation 1.9
|
5.0 percentage of activity
Standard Deviation NA
SD could not be calculated for a single participant.
|
|
Change From Baseline in Wieslab Enzyme-linked Immunosorbent Assay (ELISA) Values for Alternative Complement Pathway at Each Time Point
Month 9
|
-0.9 percentage of activity
Standard Deviation 0.8
|
-0.7 percentage of activity
Standard Deviation 0.6
|
-2.0 percentage of activity
Standard Deviation NA
SD could not be calculated for a single participant.
|
|
Change From Baseline in Wieslab Enzyme-linked Immunosorbent Assay (ELISA) Values for Alternative Complement Pathway at Each Time Point
Month 12
|
-1.4 percentage of activity
Standard Deviation 1.4
|
-1.3 percentage of activity
Standard Deviation 0.6
|
4.5 percentage of activity
Standard Deviation 6.4
|
|
Change From Baseline in Wieslab Enzyme-linked Immunosorbent Assay (ELISA) Values for Alternative Complement Pathway at Each Time Point
Final Study Visit (Month 49)
|
—
|
1.0 percentage of activity
Standard Deviation 2.8
|
—
|
SECONDARY outcome
Timeframe: Baseline, Month 1, 2, 3, 6, 9, 12 and Final Study Visit (Month 49)Population: PD population included all participants in the Safety population who had at least 1 plasma sample obtained for PD assessment. Here, Number of participants analyzed included those participants who were evaluable for the assessment and 'n' (Number analyzed) signifies participants who were evaluable at specified time points.
Blood samples were collected for measurement of Complement component 5 (C5) levels.
Outcome measures
| Measure |
Zilucoplan-Cohort A (Eculizumab Naïve)
n=9 Participants
Cohort A (Eculizumab Naive) included all participants from study RA101495-01.201 (NCT03078582) who had not received eculizumab for treatment of paroxysmal nocturnal hemoglobinuria (PNH). Participants received a loading dose of 0.3 milligram/kilogram (mg/kg) zilucoplan administered by subcutaneous (SC) injection at Day 1 of this study. Following in-clinic education and training, all participants self-injected 0.1 mg/kg of zilucoplan by SC injection once daily for 12 months. From Week 2 onwards, if a participant had not achieved an adequate response, the zilucoplan dose was escalated to 0.3 mg/kg daily.
|
Zilucoplan-Cohort B (Eculizumab Switch)
n=6 Participants
Cohort B (Eculizumab Switch) included all participants from study RA101495-01.201 (NCT03078582) who had received eculizumab for treatment of PNH. Participants received a loading dose of 0.3 mg/kg zilucoplan administered by SC injection at Day 1 of this study. Following in-clinic education and training, all participants self-injected 0.1 mg/kg of zilucoplan by SC injection once daily for 12 months. From Week 2 onwards, if a participant had not achieved an adequate response, the zilucoplan dose was escalated to 0.3 mg/kg daily.
|
Zilucoplan (Inadequate Responder to Eculizumab)
n=2 Participants
Inadequate Responder cohort included participants from qualifying study RA101495-01.203 (NCT03030183) who had an inadequate response to eculizumab treatment for PNH. Participants received a loading dose of 0.3 mg/kg zilucoplan administered by SC injection at Day 1 of this study. Following in-clinic education and training, all participants self-injected 0.1 mg/kg of zilucoplan by SC injection once daily for 12 months. From Week 2 onwards, if a participant had not achieved an adequate response, the zilucoplan dose was escalated to 0.3 mg/kg daily.
|
|---|---|---|---|
|
Change From Baseline in Complement Component 5 (C5) Values at Each Time Point
Month 1
|
-11.231 ug/mL
Standard Deviation 24.960
|
20.488 ug/mL
Standard Deviation 31.024
|
-27.505 ug/mL
Standard Deviation 90.092
|
|
Change From Baseline in Complement Component 5 (C5) Values at Each Time Point
Month 2
|
-15.914 ug/mL
Standard Deviation 32.737
|
17.716 ug/mL
Standard Deviation 33.125
|
16.310 ug/mL
Standard Deviation 36.077
|
|
Change From Baseline in Complement Component 5 (C5) Values at Each Time Point
Month 3
|
-24.404 ug/mL
Standard Deviation 50.084
|
-2.898 ug/mL
Standard Deviation 49.747
|
22.355 ug/mL
Standard Deviation 19.764
|
|
Change From Baseline in Complement Component 5 (C5) Values at Each Time Point
Month 6
|
-6.823 ug/mL
Standard Deviation 44.787
|
9.572 ug/mL
Standard Deviation 24.927
|
91.640 ug/mL
Standard Deviation NA
SD could not be calculated for a single participant.
|
|
Change From Baseline in Complement Component 5 (C5) Values at Each Time Point
Month 9
|
-0.019 ug/mL
Standard Deviation 53.475
|
1.177 ug/mL
Standard Deviation 27.654
|
89.670 ug/mL
Standard Deviation NA
SD could not be calculated for a single participant.
|
|
Change From Baseline in Complement Component 5 (C5) Values at Each Time Point
Month 12
|
-40.591 ug/mL
Standard Deviation 34.977
|
-23.487 ug/mL
Standard Deviation 30.911
|
-24.905 ug/mL
Standard Deviation 17.685
|
|
Change From Baseline in Complement Component 5 (C5) Values at Each Time Point
Final Study Visit (Month 49)
|
-28.663 ug/mL
Standard Deviation 46.526
|
-19.770 ug/mL
Standard Deviation 74.604
|
—
|
Adverse Events
Zilucoplan-Cohort A (Eculizumab Naïve)
Serious events: 1 serious events
Other events: 9 other events
Deaths: 0 deaths
Zilucoplan-Cohort B (Eculizumab Switch)
Serious events: 3 serious events
Other events: 6 other events
Deaths: 0 deaths
Zilucoplan (Inadequate Responder to Eculizumab)
Serious events: 2 serious events
Other events: 2 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Zilucoplan-Cohort A (Eculizumab Naïve)
n=10 participants at risk
Cohort A (Eculizumab Naive) included all participants from study RA101495-01.201 (NCT03078582) who had not received eculizumab for treatment of paroxysmal nocturnal hemoglobinuria (PNH). Participants received a loading dose of 0.3 milligram/kilogram (mg/kg) zilucoplan administered by subcutaneous (SC) injection at Day 1 of this study. Following in-clinic education and training, all participants self-injected 0.1 mg/kg of zilucoplan by SC injection once daily for 12 months. From Week 2 onwards, if a participant had not achieved an adequate response, the zilucoplan dose was escalated to 0.3 mg/kg daily.
|
Zilucoplan-Cohort B (Eculizumab Switch)
n=6 participants at risk
Cohort B (Eculizumab Switch) included all participants from study RA101495-01.201 (NCT03078582) who had received eculizumab for treatment of PNH. Participants received a loading dose of 0.3 mg/kg zilucoplan administered by SC injection at Day 1 of this study. Following in-clinic education and training, all participants self-injected 0.1 mg/kg of zilucoplan by SC injection once daily for 12 months. From Week 2 onwards, if a participant had not achieved an adequate response, the zilucoplan dose was escalated to 0.3 mg/kg daily.
|
Zilucoplan (Inadequate Responder to Eculizumab)
n=3 participants at risk
Inadequate Responder cohort included participants from qualifying study RA101495-01.203 (NCT03030183) who had an inadequate response to eculizumab treatment for PNH. Participants received a loading dose of 0.3 mg/kg zilucoplan administered by SC injection at Day 1 of this study. Following in-clinic education and training, all participants self-injected 0.1 mg/kg of zilucoplan by SC injection once daily for 12 months. From Week 2 onwards, if a participant had not achieved an adequate response, the zilucoplan dose was escalated to 0.3 mg/kg daily.
|
|---|---|---|---|
|
Blood and lymphatic system disorders
Anaemia
|
10.0%
1/10 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
33.3%
1/3 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Gastrointestinal disorders
Nausea
|
10.0%
1/10 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Gastrointestinal disorders
Tongue haematoma
|
0.00%
0/10 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
16.7%
1/6 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Infections and infestations
Enterocolitis infectious
|
0.00%
0/10 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
16.7%
1/6 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Infections and infestations
Pneumonia pneumococcal
|
0.00%
0/10 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
33.3%
1/3 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Musculoskeletal and connective tissue disorders
Osteoarthritis
|
10.0%
1/10 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Musculoskeletal and connective tissue disorders
Rotator cuff syndrome
|
10.0%
1/10 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Nervous system disorders
Encephalopathy
|
0.00%
0/10 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
33.3%
1/3 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Nervous system disorders
Headache
|
10.0%
1/10 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Psychiatric disorders
Suicide attempt
|
0.00%
0/10 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
33.3%
1/3 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Vascular disorders
Deep vein thrombosis
|
0.00%
0/10 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
16.7%
1/6 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
Other adverse events
| Measure |
Zilucoplan-Cohort A (Eculizumab Naïve)
n=10 participants at risk
Cohort A (Eculizumab Naive) included all participants from study RA101495-01.201 (NCT03078582) who had not received eculizumab for treatment of paroxysmal nocturnal hemoglobinuria (PNH). Participants received a loading dose of 0.3 milligram/kilogram (mg/kg) zilucoplan administered by subcutaneous (SC) injection at Day 1 of this study. Following in-clinic education and training, all participants self-injected 0.1 mg/kg of zilucoplan by SC injection once daily for 12 months. From Week 2 onwards, if a participant had not achieved an adequate response, the zilucoplan dose was escalated to 0.3 mg/kg daily.
|
Zilucoplan-Cohort B (Eculizumab Switch)
n=6 participants at risk
Cohort B (Eculizumab Switch) included all participants from study RA101495-01.201 (NCT03078582) who had received eculizumab for treatment of PNH. Participants received a loading dose of 0.3 mg/kg zilucoplan administered by SC injection at Day 1 of this study. Following in-clinic education and training, all participants self-injected 0.1 mg/kg of zilucoplan by SC injection once daily for 12 months. From Week 2 onwards, if a participant had not achieved an adequate response, the zilucoplan dose was escalated to 0.3 mg/kg daily.
|
Zilucoplan (Inadequate Responder to Eculizumab)
n=3 participants at risk
Inadequate Responder cohort included participants from qualifying study RA101495-01.203 (NCT03030183) who had an inadequate response to eculizumab treatment for PNH. Participants received a loading dose of 0.3 mg/kg zilucoplan administered by SC injection at Day 1 of this study. Following in-clinic education and training, all participants self-injected 0.1 mg/kg of zilucoplan by SC injection once daily for 12 months. From Week 2 onwards, if a participant had not achieved an adequate response, the zilucoplan dose was escalated to 0.3 mg/kg daily.
|
|---|---|---|---|
|
Gastrointestinal disorders
Constipation
|
10.0%
1/10 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
33.3%
1/3 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Gastrointestinal disorders
Abdominal distension
|
10.0%
1/10 • Number of events 2 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Blood and lymphatic system disorders
Anaemia
|
20.0%
2/10 • Number of events 4 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Blood and lymphatic system disorders
Haemolysis
|
10.0%
1/10 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
16.7%
1/6 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Blood and lymphatic system disorders
Aplastic anaemia
|
0.00%
0/10 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
16.7%
1/6 • Number of events 2 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Blood and lymphatic system disorders
Pancytopenia
|
0.00%
0/10 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
16.7%
1/6 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Cardiac disorders
Bradycardia
|
10.0%
1/10 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Ear and labyrinth disorders
Tinnitus
|
10.0%
1/10 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Eye disorders
Lacrimation increased
|
0.00%
0/10 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
16.7%
1/6 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Gastrointestinal disorders
Diarrhoea
|
50.0%
5/10 • Number of events 8 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
16.7%
1/6 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Gastrointestinal disorders
Nausea
|
40.0%
4/10 • Number of events 5 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Gastrointestinal disorders
Abdominal pain
|
30.0%
3/10 • Number of events 7 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Gastrointestinal disorders
Abdominal discomfort
|
10.0%
1/10 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Gastrointestinal disorders
Abdominal pain upper
|
10.0%
1/10 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Gastrointestinal disorders
Gastrooesophageal reflux disease
|
10.0%
1/10 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Gastrointestinal disorders
Large intestine polyp
|
10.0%
1/10 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Gastrointestinal disorders
Oral discomfort
|
10.0%
1/10 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Gastrointestinal disorders
Oral pain
|
10.0%
1/10 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Gastrointestinal disorders
Rectal haemorrhage
|
10.0%
1/10 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Gastrointestinal disorders
Rectal polyp
|
10.0%
1/10 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Gastrointestinal disorders
Retching
|
10.0%
1/10 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Gastrointestinal disorders
Vomiting
|
0.00%
0/10 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
33.3%
1/3 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
General disorders
Fatigue
|
40.0%
4/10 • Number of events 9 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
33.3%
2/6 • Number of events 3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
33.3%
1/3 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
General disorders
Injection site bruising
|
20.0%
2/10 • Number of events 2 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
16.7%
1/6 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
General disorders
Pyrexia
|
30.0%
3/10 • Number of events 3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
General disorders
Chest discomfort
|
20.0%
2/10 • Number of events 2 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
General disorders
Chest pain
|
0.00%
0/10 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
33.3%
1/3 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
General disorders
Chills
|
10.0%
1/10 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
General disorders
Influenza like illness
|
0.00%
0/10 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
33.3%
1/3 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
General disorders
Injection site haematoma
|
10.0%
1/10 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Hepatobiliary disorders
Cholelithiasis
|
10.0%
1/10 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Immune system disorders
Seasonal allergy
|
20.0%
2/10 • Number of events 3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Immune system disorders
Alloimmunisation
|
10.0%
1/10 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Immune system disorders
Drug hypersensitivity
|
0.00%
0/10 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
16.7%
1/6 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Infections and infestations
Nasopharyngitis
|
40.0%
4/10 • Number of events 10 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
16.7%
1/6 • Number of events 2 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Infections and infestations
Upper respiratory tract infection
|
40.0%
4/10 • Number of events 5 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
16.7%
1/6 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Infections and infestations
Pharyngitis
|
10.0%
1/10 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
16.7%
1/6 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Infections and infestations
Sinusitis
|
10.0%
1/10 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
33.3%
1/3 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Infections and infestations
Viral infection
|
10.0%
1/10 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
16.7%
1/6 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Infections and infestations
Bronchitis
|
10.0%
1/10 • Number of events 2 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Infections and infestations
Influenza
|
10.0%
1/10 • Number of events 2 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Infections and infestations
Rhinitis
|
10.0%
1/10 • Number of events 2 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Infections and infestations
Cystitis
|
10.0%
1/10 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Infections and infestations
Ear infection
|
10.0%
1/10 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Infections and infestations
Gastroenteritis
|
10.0%
1/10 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Infections and infestations
Gastrointestinal infection
|
10.0%
1/10 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Infections and infestations
Hordeolum
|
10.0%
1/10 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Infections and infestations
Infection
|
10.0%
1/10 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Infections and infestations
Oral candidiasis
|
10.0%
1/10 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Infections and infestations
Oral herpes
|
10.0%
1/10 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Infections and infestations
Respiratory tract infection
|
0.00%
0/10 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
33.3%
1/3 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Infections and infestations
Skin bacterial infection
|
10.0%
1/10 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Infections and infestations
Skin infection
|
10.0%
1/10 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Infections and infestations
Tooth abscess
|
10.0%
1/10 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Infections and infestations
Urinary tract infection
|
10.0%
1/10 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Injury, poisoning and procedural complications
Contusion
|
20.0%
2/10 • Number of events 3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Injury, poisoning and procedural complications
Post vaccination syndrome
|
10.0%
1/10 • Number of events 2 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Injury, poisoning and procedural complications
Animal bite
|
10.0%
1/10 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Injury, poisoning and procedural complications
Bone contusion
|
10.0%
1/10 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Injury, poisoning and procedural complications
Eye contusion
|
0.00%
0/10 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
16.7%
1/6 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Injury, poisoning and procedural complications
Fall
|
0.00%
0/10 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
16.7%
1/6 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Injury, poisoning and procedural complications
Foot fracture
|
10.0%
1/10 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Injury, poisoning and procedural complications
Limb injury
|
10.0%
1/10 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Injury, poisoning and procedural complications
Post-traumatic pain
|
0.00%
0/10 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
16.7%
1/6 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Injury, poisoning and procedural complications
Tooth fracture
|
10.0%
1/10 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Injury, poisoning and procedural complications
Traumatic haematoma
|
0.00%
0/10 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
16.7%
1/6 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Investigations
Alanine aminotransferase increased
|
0.00%
0/10 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
33.3%
1/3 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Investigations
Aspartate aminotransferase increased
|
0.00%
0/10 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
33.3%
1/3 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Investigations
Grip strength decreased
|
10.0%
1/10 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Investigations
International normalised ratio increased
|
0.00%
0/10 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
33.3%
1/3 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Investigations
Weight decreased
|
10.0%
1/10 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Metabolism and nutrition disorders
Hyperkalaemia
|
10.0%
1/10 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Metabolism and nutrition disorders
Hypokalaemia
|
0.00%
0/10 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
33.3%
1/3 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Metabolism and nutrition disorders
Vitamin D deficiency
|
0.00%
0/10 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
16.7%
1/6 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
30.0%
3/10 • Number of events 9 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
33.3%
1/3 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Musculoskeletal and connective tissue disorders
Muscle spasms
|
20.0%
2/10 • Number of events 6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
10.0%
1/10 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
33.3%
1/3 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
10.0%
1/10 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
16.7%
1/6 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
10.0%
1/10 • Number of events 2 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Musculoskeletal and connective tissue disorders
Arthritis
|
10.0%
1/10 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Musculoskeletal and connective tissue disorders
Joint swelling
|
0.00%
0/10 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
16.7%
1/6 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Musculoskeletal and connective tissue disorders
Limb discomfort
|
10.0%
1/10 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Musculoskeletal and connective tissue disorders
Muscle tightness
|
10.0%
1/10 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal pain
|
0.00%
0/10 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
16.7%
1/6 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Musculoskeletal and connective tissue disorders
Plantar fasciitis
|
10.0%
1/10 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Musculoskeletal and connective tissue disorders
Rotator cuff syndrome
|
10.0%
1/10 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Basal cell carcinoma
|
20.0%
2/10 • Number of events 3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Nervous system disorders
Headache
|
20.0%
2/10 • Number of events 4 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
33.3%
2/6 • Number of events 6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
33.3%
1/3 • Number of events 2 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Nervous system disorders
Dizziness
|
20.0%
2/10 • Number of events 4 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Nervous system disorders
Amnesia
|
0.00%
0/10 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
33.3%
1/3 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Nervous system disorders
Sensory disturbance
|
10.0%
1/10 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Psychiatric disorders
Depression
|
0.00%
0/10 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
16.7%
1/6 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
33.3%
1/3 • Number of events 2 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Psychiatric disorders
Insomnia
|
10.0%
1/10 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Renal and urinary disorders
Paroxysmal nocturnal haemoglobinuria
|
10.0%
1/10 • Number of events 3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Renal and urinary disorders
Chromaturia
|
10.0%
1/10 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
30.0%
3/10 • Number of events 4 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
20.0%
2/10 • Number of events 2 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
33.3%
1/3 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
10.0%
1/10 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Respiratory, thoracic and mediastinal disorders
Laryngospasm
|
10.0%
1/10 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Respiratory, thoracic and mediastinal disorders
Pleural effusion
|
10.0%
1/10 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
10.0%
1/10 • Number of events 2 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Skin and subcutaneous tissue disorders
Actinic keratosis
|
10.0%
1/10 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Skin and subcutaneous tissue disorders
Psoriasis
|
0.00%
0/10 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
16.7%
1/6 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Skin and subcutaneous tissue disorders
Rash
|
10.0%
1/10 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Skin and subcutaneous tissue disorders
Rash pruritic
|
10.0%
1/10 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Skin and subcutaneous tissue disorders
Urticaria
|
10.0%
1/10 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Vascular disorders
Hypertension
|
10.0%
1/10 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
|
Vascular disorders
Lymphoedema
|
10.0%
1/10 • Number of events 1 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/6 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
0.00%
0/3 • From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were reported for Safety population. Deaths and TEAEs were monitored together for each cohort regardless of dose administered.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: GT60