Trial Outcomes & Findings for Safety, Tolerability, and Efficacy of Zilucoplan in Subjects With Generalized Myasthenia Gravis (NCT NCT04115293)

NCT ID: NCT04115293

Last Updated: 2025-07-16

Results Overview

The MG-ADL is an 8-item patient-reported outcome measure assessing MG symptoms and their effects on daily activities. Each item in the scale scored 0 to 3 (0=None, 3=severe disease) point scale. The total score was the sum of all individual item scores and ranged from 0 to 24. Higher scores indicated more severe disability due to MG. A decrease from Baseline score indicated improvement.

• Recruitment status: COMPLETED
• Study phase: PHASE3
• Target enrollment: 174 participants
• Primary outcome timeframe: From Baseline to End of Treatment (Week 12)
• Results posted on: 2025-07-16

Participant Flow

The study started to enroll participants in September 2019 and concluded in December 2021.

The Participant flow refers to the Randomized Set.

Participant milestones

Measure	Placebo Participants self-administered zilucoplan (RA101495) matching placebo as subcutaneous (SC) injection during 12-week Treatment Period.	Zilucoplan 0.3 mg/kg Participants self-administered zilucoplan (RA101495) 0.3 milligrams/kilogram/day (mg/kg/day) SC injection during 12-week Treatment Period.
Overall Study STARTED	88	86
Overall Study COMPLETED	84	82
Overall Study NOT COMPLETED	4	4

Reasons for withdrawal

Measure	Placebo Participants self-administered zilucoplan (RA101495) matching placebo as subcutaneous (SC) injection during 12-week Treatment Period.	Zilucoplan 0.3 mg/kg Participants self-administered zilucoplan (RA101495) 0.3 milligrams/kilogram/day (mg/kg/day) SC injection during 12-week Treatment Period.
Overall Study Adverse Event	0	2
Overall Study Withdrawal by Subject	2	1
Overall Study Physician Decision	1	0
Overall Study Death	1	1

Baseline Characteristics

Safety, Tolerability, and Efficacy of Zilucoplan in Subjects With Generalized Myasthenia Gravis

Baseline characteristics by cohort

Measure	Placebo n=88 Participants Participants self-administered zilucoplan (RA101495) matching placebo as subcutaneous (SC) injection during 12-week Treatment Period.	Zilucoplan 0.3 mg/kg n=86 Participants Participants self-administered zilucoplan (RA101495) 0.3 milligrams/kilogram/day (mg/kg/day) SC injection during 12-week Treatment Period.	Total n=174 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
Age, Categorical Between 18 and 65 years	62 Participants n=5 Participants	64 Participants n=7 Participants	126 Participants n=5 Participants
Age, Categorical >=65 years	26 Participants n=5 Participants	22 Participants n=7 Participants	48 Participants n=5 Participants
Age, Continuous	53.3 years STANDARD_DEVIATION 15.7 • n=5 Participants	52.6 years STANDARD_DEVIATION 14.6 • n=7 Participants	53.0 years STANDARD_DEVIATION 15.1 • n=5 Participants
Sex: Female, Male Female	47 Participants n=5 Participants	52 Participants n=7 Participants	99 Participants n=5 Participants
Sex: Female, Male Male	41 Participants n=5 Participants	34 Participants n=7 Participants	75 Participants n=5 Participants
Race/Ethnicity, Customized American Indian/Alaska native	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants
Race/Ethnicity, Customized Asian	14 Participants n=5 Participants	7 Participants n=7 Participants	21 Participants n=5 Participants
Race/Ethnicity, Customized Black	7 Participants n=5 Participants	6 Participants n=7 Participants	13 Participants n=5 Participants
Race/Ethnicity, Customized Native Hawaiian or other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race/Ethnicity, Customized White	62 Participants n=5 Participants	66 Participants n=7 Participants	128 Participants n=5 Participants
Race/Ethnicity, Customized Other/Mixed	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race/Ethnicity, Customized Missing	4 Participants n=5 Participants	7 Participants n=7 Participants	11 Participants n=5 Participants
Race/Ethnicity, Customized Hispanic or Latino	5 Participants n=5 Participants	7 Participants n=7 Participants	12 Participants n=5 Participants
Race/Ethnicity, Customized Not Hispanic or Latino	79 Participants n=5 Participants	72 Participants n=7 Participants	151 Participants n=5 Participants

PRIMARY outcome

Timeframe: From Baseline to End of Treatment (Week 12)

Population: The modified Intention-to-Treat (mITT) population included all randomized participants who received at least 1 dose of study drug and had at least 1 post-dosing MG-ADL score.

The MG-ADL is an 8-item patient-reported outcome measure assessing MG symptoms and their effects on daily activities. Each item in the scale scored 0 to 3 (0=None, 3=severe disease) point scale. The total score was the sum of all individual item scores and ranged from 0 to 24. Higher scores indicated more severe disability due to MG. A decrease from Baseline score indicated improvement.

Outcome measures

Measure	Placebo n=88 Participants Participants self-administered zilucoplan (RA101495) matching placebo as subcutaneous (SC) injection during 12-week Treatment Period.	Zilucoplan 0.3 mg/kg n=86 Participants Participants self-administered zilucoplan (RA101495) 0.3 milligrams/kilogram/day (mg/kg/day) SC injection during 12-week Treatment Period.
Change From Baseline (CFB) to Week 12 in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Total Score	-2.30 score on a scale Interval -3.17 to -1.43	-4.39 score on a scale Interval -5.28 to -3.5

SECONDARY outcome

Timeframe: From Baseline to End of Treatment (Week 12)

Population: The modified Intention-to-Treat (mITT) population included all randomized participants who received at least 1 dose of study drug and had at least 1 post-dosing MG-ADL score.

The QMG is a standardized and validated quantitative strength scoring system that was developed specifically for MG. The scale consisted of 13 items. Each item in the scale scored on a 0 to 3-point scale, ranging from 0 (no weakness) to 3 (severe weakness), summing up to the overall score range from 0 to 39. Higher scores indicated more severe impairment. A decrease from Baseline score indicated improvement.

Outcome measures

Measure	Placebo n=88 Participants Participants self-administered zilucoplan (RA101495) matching placebo as subcutaneous (SC) injection during 12-week Treatment Period.	Zilucoplan 0.3 mg/kg n=86 Participants Participants self-administered zilucoplan (RA101495) 0.3 milligrams/kilogram/day (mg/kg/day) SC injection during 12-week Treatment Period.
Change From Baseline to Week 12 in the Quantitative Myasthenia Gravis (QMG) Total Score	-3.25 score on a scale Interval -4.32 to -2.17	-6.19 score on a scale Interval -7.29 to -5.08

SECONDARY outcome

Timeframe: From Baseline to End of Treatment (Week 12)

Population: The modified Intention-to-Treat (mITT) population included all randomized participants who received at least 1 dose of study drug and had at least 1 post-dosing MG-ADL score.

The total MGC score was sum of responses to 10 individual items : 1. Ptosis upward gaze (0 to 3), 2. Double vision on lateral gaze, left or right (0, to 4), 3. Eye closure (0 to 2), 4. Talking (0 to 6), 5. Chewing (0 to 6), 6. Swallowing [0 to 6], 7. Breathing (0 to 9), 8. Neck flexion or extension (0 to 4), 9. Shoulder abduction (0 to 5), 10. Hip flexion (0 to 5). The higher score for each item indicated severity. The total score ranged 0 to 50 with higher score indicative of severe disease activity). A decrease from Baseline score showed improvement.

Outcome measures

Measure	Placebo n=88 Participants Participants self-administered zilucoplan (RA101495) matching placebo as subcutaneous (SC) injection during 12-week Treatment Period.	Zilucoplan 0.3 mg/kg n=86 Participants Participants self-administered zilucoplan (RA101495) 0.3 milligrams/kilogram/day (mg/kg/day) SC injection during 12-week Treatment Period.
Change From Baseline to Week 12 in the Myasthenia Gravis Composite (MGC) Scale Total Score	-5.42 score on a scale Interval -6.98 to -3.86	-8.62 score on a scale Interval -10.22 to -7.01

SECONDARY outcome

Timeframe: From Baseline to End of Treatment (Week 12)

Population: The modified Intention-to-Treat (mITT) population included all randomized participants who received at least 1 dose of study drug and had at least 1 post-dosing MG-ADL score.

The MG-QoL15r is a 15-item patient-reported outcome measure designed to assess quality of life in patients with MG. Each item in the scale scored on a 0 to 2-point scale (0=Not much at all, 1=Somewhat, 2=Very much). The total score was the sum of the 15 individual item scores, ranging from 0 to 30. Higher scores indicated more severe impact of the disease on aspects of the patient's life. A decrease from Baseline score indicated improvement.

Outcome measures

Measure	Placebo n=88 Participants Participants self-administered zilucoplan (RA101495) matching placebo as subcutaneous (SC) injection during 12-week Treatment Period.	Zilucoplan 0.3 mg/kg n=86 Participants Participants self-administered zilucoplan (RA101495) 0.3 milligrams/kilogram/day (mg/kg/day) SC injection during 12-week Treatment Period.
Change From Baseline to Week 12 in the Myasthenia Gravis - Quality of Life Revised (MG-QoL15r) Scale Total Score	-3.16 score on a scale Interval -4.65 to -1.67	-5.65 score on a scale Interval -7.17 to -4.12

SECONDARY outcome

Timeframe: From Baseline to End of Treatment (Week 12)

Population: The modified Intention-to-Treat (mITT) population included all randomized participants who received at least 1 dose of study drug and had at least 1 post-dosing MG-ADL score.

Time to first receipt of rescue therapy over the 12-week treatment period (in days) was defined as the date of first rescue therapy use minus date of first Investigational Medicinal Product (IMP) + 1.

Outcome measures

Measure	Placebo n=88 Participants Participants self-administered zilucoplan (RA101495) matching placebo as subcutaneous (SC) injection during 12-week Treatment Period.	Zilucoplan 0.3 mg/kg n=86 Participants Participants self-administered zilucoplan (RA101495) 0.3 milligrams/kilogram/day (mg/kg/day) SC injection during 12-week Treatment Period.
Time to First Receipt of Rescue Therapy Over the 12-week Treatment Period	NA Days The median time to first receipt of rescue therapy was not estimated due to the less number of events.	NA Days The median time to first receipt of rescue therapy was not estimated due to the less number of events.

SECONDARY outcome

Timeframe: End of Treatment (Week 12)

Population: The mITT population included randomized participants who received at least 1 dose of study drug and had at least 1 post-dosing MG-ADL score.

Percentage of Participants achieving MSE was defined as achieving a MG-ADL value of a 0 (No MG symptoms) or 1 (Mild MG symptoms) at Week 12 and not having taken rescue therapy. Any participant with an event of death, myasthenic crisis or rescue therapy was considered as non-responders. Any other missing data was imputed using the missing at Random (MAR) assumption.

Outcome measures

Measure	Placebo n=88 Participants Participants self-administered zilucoplan (RA101495) matching placebo as subcutaneous (SC) injection during 12-week Treatment Period.	Zilucoplan 0.3 mg/kg n=86 Participants Participants self-administered zilucoplan (RA101495) 0.3 milligrams/kilogram/day (mg/kg/day) SC injection during 12-week Treatment Period.
Percentage of Participants Achieving Minimal Symptom Expression (MSE) at Week 12 Without Rescue Therapy	5.8 percentage of participants	14.0 percentage of participants

SECONDARY outcome

Timeframe: End of Treatment (Week 12)

Population: The mITT population included randomized participants who received at least 1 dose of study drug and had at least 1 post-dosing MG-ADL score.

Percentage of participants achieving a ≥ 3-point reduction in MG-ADL Score at Week 12 without rescue therapy were reported. The MG-ADL is an 8-item patient-reported outcome measure assessing MG symptoms and their effects on daily activities. Each item in the scale scored on a 0 to 3 (0=None, 3=severe disease) point scale. The total score was the sum of all individual item scores and ranged from 0 to 24. Higher scores indicated more severe disability due to MG. Any participant with an event of death, myasthenic crisis or rescue therapy was considered as non-responders. Any other missing data was imputed using the MAR assumption.

Outcome measures

Measure	Placebo n=88 Participants Participants self-administered zilucoplan (RA101495) matching placebo as subcutaneous (SC) injection during 12-week Treatment Period.	Zilucoplan 0.3 mg/kg n=86 Participants Participants self-administered zilucoplan (RA101495) 0.3 milligrams/kilogram/day (mg/kg/day) SC injection during 12-week Treatment Period.
Percentage of Participants Achieving a ≥ 3-point Reduction in MG-ADL Score at Week 12 Without Rescue Therapy	46.1 percentage of participants	73.1 percentage of participants

SECONDARY outcome

Timeframe: End of Treatment (Week 12)

Population: The mITT population included randomized participants who received at least 1 dose of study drug and had at least 1 post-dosing MG-ADL score.

Percentage of participants achieving a ≥5-point reduction in QMG Score without rescue therapy at Week 12 were reported. The QMG is a standardized and validated quantitative strength scoring system that was developed specifically for MG. The scale consisted of 13 items. Each item in the scale scored on a 0 to 3-point scale, ranging from 0 (no weakness) to 3 (severe weakness), summing up to the overall score range from 0 to 39. Higher scores indicated more severe impairment. Any participant with an event of death, myasthenic crisis or rescue therapy was considered as non-responders. Any other missing data was imputed using the MAR assumption.

Outcome measures

Measure	Placebo n=88 Participants Participants self-administered zilucoplan (RA101495) matching placebo as subcutaneous (SC) injection during 12-week Treatment Period.	Zilucoplan 0.3 mg/kg n=86 Participants Participants self-administered zilucoplan (RA101495) 0.3 milligrams/kilogram/day (mg/kg/day) SC injection during 12-week Treatment Period.
Percentage of Participants Achieving a ≥5-point Reduction in QMG Score Without Rescue Therapy at Week 12	33.0 percentage of participants	58.0 percentage of participants

SECONDARY outcome

Timeframe: From Baseline (Day 1) to Safety Follow-up visit (19 Weeks [12 weeks Treatment Period plus up to 7 weeks Follow-up])

Population: The Safety Set (SS) included all participants who received at least 1 dose of study drug based on the actual study treatment received.

A TEAE is defined as an AE starting on or after the time of first administration of IMP and up to and including 40 days after the final dose (or last contact depending on which occurs first). Adverse events starting before the date of the first administration of IMP were not considered TEAEs.

Outcome measures

Measure	Placebo n=88 Participants Participants self-administered zilucoplan (RA101495) matching placebo as subcutaneous (SC) injection during 12-week Treatment Period.	Zilucoplan 0.3 mg/kg n=86 Participants Participants self-administered zilucoplan (RA101495) 0.3 milligrams/kilogram/day (mg/kg/day) SC injection during 12-week Treatment Period.
Percentage of Participants With Treatment-emergent Adverse Events (TEAEs)	70.5 percentage of participants	76.7 percentage of participants

Adverse Events

Placebo

Serious events: 13 serious events

Other events: 34 other events

Deaths: 1 deaths

Zilucoplan 0.3 mg/kg

Serious events: 11 serious events

Other events: 41 other events

Deaths: 1 deaths

Serious adverse events

Measure	Placebo n=88 participants at risk Participants self-administered zilucoplan (RA101495) matching placebo as subcutaneous (SC) injection during 12-week Treatment Period.	Zilucoplan 0.3 mg/kg n=86 participants at risk Participants self-administered zilucoplan (RA101495) 0.3 milligrams/kilogram/day (mg/kg/day) SC injection during 12-week Treatment Period.
Blood and lymphatic system disorders Anaemia	0.00% 0/88 • From Baseline (Day 1) to Safety follow-up visit (19 Weeks [12 weeks Treatment Period plus up to 7 weeks follow-up]) A TEAE is defined as an AE starting on or after the time of first administration of IMP and up to and including 40 days after the final dose (or last contact depending on which occurs first). Adverse events starting before the date of the first administration of IMP were not considered TEAEs.	1.2% 1/86 • Number of events 1 • From Baseline (Day 1) to Safety follow-up visit (19 Weeks [12 weeks Treatment Period plus up to 7 weeks follow-up]) A TEAE is defined as an AE starting on or after the time of first administration of IMP and up to and including 40 days after the final dose (or last contact depending on which occurs first). Adverse events starting before the date of the first administration of IMP were not considered TEAEs.
Gastrointestinal disorders Vomiting	1.1% 1/88 • Number of events 1 • From Baseline (Day 1) to Safety follow-up visit (19 Weeks [12 weeks Treatment Period plus up to 7 weeks follow-up]) A TEAE is defined as an AE starting on or after the time of first administration of IMP and up to and including 40 days after the final dose (or last contact depending on which occurs first). Adverse events starting before the date of the first administration of IMP were not considered TEAEs.	0.00% 0/86 • From Baseline (Day 1) to Safety follow-up visit (19 Weeks [12 weeks Treatment Period plus up to 7 weeks follow-up]) A TEAE is defined as an AE starting on or after the time of first administration of IMP and up to and including 40 days after the final dose (or last contact depending on which occurs first). Adverse events starting before the date of the first administration of IMP were not considered TEAEs.
Gastrointestinal disorders Aphthous ulcer	0.00% 0/88 • From Baseline (Day 1) to Safety follow-up visit (19 Weeks [12 weeks Treatment Period plus up to 7 weeks follow-up]) A TEAE is defined as an AE starting on or after the time of first administration of IMP and up to and including 40 days after the final dose (or last contact depending on which occurs first). Adverse events starting before the date of the first administration of IMP were not considered TEAEs.	1.2% 1/86 • Number of events 1 • From Baseline (Day 1) to Safety follow-up visit (19 Weeks [12 weeks Treatment Period plus up to 7 weeks follow-up]) A TEAE is defined as an AE starting on or after the time of first administration of IMP and up to and including 40 days after the final dose (or last contact depending on which occurs first). Adverse events starting before the date of the first administration of IMP were not considered TEAEs.
Infections and infestations Oesophageal candidiasis	0.00% 0/88 • From Baseline (Day 1) to Safety follow-up visit (19 Weeks [12 weeks Treatment Period plus up to 7 weeks follow-up]) A TEAE is defined as an AE starting on or after the time of first administration of IMP and up to and including 40 days after the final dose (or last contact depending on which occurs first). Adverse events starting before the date of the first administration of IMP were not considered TEAEs.	1.2% 1/86 • Number of events 1 • From Baseline (Day 1) to Safety follow-up visit (19 Weeks [12 weeks Treatment Period plus up to 7 weeks follow-up]) A TEAE is defined as an AE starting on or after the time of first administration of IMP and up to and including 40 days after the final dose (or last contact depending on which occurs first). Adverse events starting before the date of the first administration of IMP were not considered TEAEs.
Infections and infestations Oral candidiasis	0.00% 0/88 • From Baseline (Day 1) to Safety follow-up visit (19 Weeks [12 weeks Treatment Period plus up to 7 weeks follow-up]) A TEAE is defined as an AE starting on or after the time of first administration of IMP and up to and including 40 days after the final dose (or last contact depending on which occurs first). Adverse events starting before the date of the first administration of IMP were not considered TEAEs.	1.2% 1/86 • Number of events 1 • From Baseline (Day 1) to Safety follow-up visit (19 Weeks [12 weeks Treatment Period plus up to 7 weeks follow-up]) A TEAE is defined as an AE starting on or after the time of first administration of IMP and up to and including 40 days after the final dose (or last contact depending on which occurs first). Adverse events starting before the date of the first administration of IMP were not considered TEAEs.
Infections and infestations COVID-19	2.3% 2/88 • Number of events 2 • From Baseline (Day 1) to Safety follow-up visit (19 Weeks [12 weeks Treatment Period plus up to 7 weeks follow-up]) A TEAE is defined as an AE starting on or after the time of first administration of IMP and up to and including 40 days after the final dose (or last contact depending on which occurs first). Adverse events starting before the date of the first administration of IMP were not considered TEAEs.	1.2% 1/86 • Number of events 1 • From Baseline (Day 1) to Safety follow-up visit (19 Weeks [12 weeks Treatment Period plus up to 7 weeks follow-up]) A TEAE is defined as an AE starting on or after the time of first administration of IMP and up to and including 40 days after the final dose (or last contact depending on which occurs first). Adverse events starting before the date of the first administration of IMP were not considered TEAEs.
Infections and infestations COVID-19 pneumonia	2.3% 2/88 • Number of events 2 • From Baseline (Day 1) to Safety follow-up visit (19 Weeks [12 weeks Treatment Period plus up to 7 weeks follow-up]) A TEAE is defined as an AE starting on or after the time of first administration of IMP and up to and including 40 days after the final dose (or last contact depending on which occurs first). Adverse events starting before the date of the first administration of IMP were not considered TEAEs.	1.2% 1/86 • Number of events 1 • From Baseline (Day 1) to Safety follow-up visit (19 Weeks [12 weeks Treatment Period plus up to 7 weeks follow-up]) A TEAE is defined as an AE starting on or after the time of first administration of IMP and up to and including 40 days after the final dose (or last contact depending on which occurs first). Adverse events starting before the date of the first administration of IMP were not considered TEAEs.
Infections and infestations Herpes simplex meningoencephalitis	1.1% 1/88 • Number of events 2 • From Baseline (Day 1) to Safety follow-up visit (19 Weeks [12 weeks Treatment Period plus up to 7 weeks follow-up]) A TEAE is defined as an AE starting on or after the time of first administration of IMP and up to and including 40 days after the final dose (or last contact depending on which occurs first). Adverse events starting before the date of the first administration of IMP were not considered TEAEs.	0.00% 0/86 • From Baseline (Day 1) to Safety follow-up visit (19 Weeks [12 weeks Treatment Period plus up to 7 weeks follow-up]) A TEAE is defined as an AE starting on or after the time of first administration of IMP and up to and including 40 days after the final dose (or last contact depending on which occurs first). Adverse events starting before the date of the first administration of IMP were not considered TEAEs.
Infections and infestations Pneumonia	0.00% 0/88 • From Baseline (Day 1) to Safety follow-up visit (19 Weeks [12 weeks Treatment Period plus up to 7 weeks follow-up]) A TEAE is defined as an AE starting on or after the time of first administration of IMP and up to and including 40 days after the final dose (or last contact depending on which occurs first). Adverse events starting before the date of the first administration of IMP were not considered TEAEs.	1.2% 1/86 • Number of events 1 • From Baseline (Day 1) to Safety follow-up visit (19 Weeks [12 weeks Treatment Period plus up to 7 weeks follow-up]) A TEAE is defined as an AE starting on or after the time of first administration of IMP and up to and including 40 days after the final dose (or last contact depending on which occurs first). Adverse events starting before the date of the first administration of IMP were not considered TEAEs.
Infections and infestations Sepsis	0.00% 0/88 • From Baseline (Day 1) to Safety follow-up visit (19 Weeks [12 weeks Treatment Period plus up to 7 weeks follow-up]) A TEAE is defined as an AE starting on or after the time of first administration of IMP and up to and including 40 days after the final dose (or last contact depending on which occurs first). Adverse events starting before the date of the first administration of IMP were not considered TEAEs.	1.2% 1/86 • Number of events 1 • From Baseline (Day 1) to Safety follow-up visit (19 Weeks [12 weeks Treatment Period plus up to 7 weeks follow-up]) A TEAE is defined as an AE starting on or after the time of first administration of IMP and up to and including 40 days after the final dose (or last contact depending on which occurs first). Adverse events starting before the date of the first administration of IMP were not considered TEAEs.
Investigations Lipase increased	0.00% 0/88 • From Baseline (Day 1) to Safety follow-up visit (19 Weeks [12 weeks Treatment Period plus up to 7 weeks follow-up]) A TEAE is defined as an AE starting on or after the time of first administration of IMP and up to and including 40 days after the final dose (or last contact depending on which occurs first). Adverse events starting before the date of the first administration of IMP were not considered TEAEs.	1.2% 1/86 • Number of events 1 • From Baseline (Day 1) to Safety follow-up visit (19 Weeks [12 weeks Treatment Period plus up to 7 weeks follow-up]) A TEAE is defined as an AE starting on or after the time of first administration of IMP and up to and including 40 days after the final dose (or last contact depending on which occurs first). Adverse events starting before the date of the first administration of IMP were not considered TEAEs.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Metastases to meninges	1.1% 1/88 • Number of events 1 • From Baseline (Day 1) to Safety follow-up visit (19 Weeks [12 weeks Treatment Period plus up to 7 weeks follow-up]) A TEAE is defined as an AE starting on or after the time of first administration of IMP and up to and including 40 days after the final dose (or last contact depending on which occurs first). Adverse events starting before the date of the first administration of IMP were not considered TEAEs.	0.00% 0/86 • From Baseline (Day 1) to Safety follow-up visit (19 Weeks [12 weeks Treatment Period plus up to 7 weeks follow-up]) A TEAE is defined as an AE starting on or after the time of first administration of IMP and up to and including 40 days after the final dose (or last contact depending on which occurs first). Adverse events starting before the date of the first administration of IMP were not considered TEAEs.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Basal cell carcinoma	0.00% 0/88 • From Baseline (Day 1) to Safety follow-up visit (19 Weeks [12 weeks Treatment Period plus up to 7 weeks follow-up]) A TEAE is defined as an AE starting on or after the time of first administration of IMP and up to and including 40 days after the final dose (or last contact depending on which occurs first). Adverse events starting before the date of the first administration of IMP were not considered TEAEs.	1.2% 1/86 • Number of events 1 • From Baseline (Day 1) to Safety follow-up visit (19 Weeks [12 weeks Treatment Period plus up to 7 weeks follow-up]) A TEAE is defined as an AE starting on or after the time of first administration of IMP and up to and including 40 days after the final dose (or last contact depending on which occurs first). Adverse events starting before the date of the first administration of IMP were not considered TEAEs.
Nervous system disorders Cerebral haemorrhage	1.1% 1/88 • Number of events 1 • From Baseline (Day 1) to Safety follow-up visit (19 Weeks [12 weeks Treatment Period plus up to 7 weeks follow-up]) A TEAE is defined as an AE starting on or after the time of first administration of IMP and up to and including 40 days after the final dose (or last contact depending on which occurs first). Adverse events starting before the date of the first administration of IMP were not considered TEAEs.	0.00% 0/86 • From Baseline (Day 1) to Safety follow-up visit (19 Weeks [12 weeks Treatment Period plus up to 7 weeks follow-up]) A TEAE is defined as an AE starting on or after the time of first administration of IMP and up to and including 40 days after the final dose (or last contact depending on which occurs first). Adverse events starting before the date of the first administration of IMP were not considered TEAEs.
Nervous system disorders Cerebrovascular accident	1.1% 1/88 • Number of events 1 • From Baseline (Day 1) to Safety follow-up visit (19 Weeks [12 weeks Treatment Period plus up to 7 weeks follow-up]) A TEAE is defined as an AE starting on or after the time of first administration of IMP and up to and including 40 days after the final dose (or last contact depending on which occurs first). Adverse events starting before the date of the first administration of IMP were not considered TEAEs.	0.00% 0/86 • From Baseline (Day 1) to Safety follow-up visit (19 Weeks [12 weeks Treatment Period plus up to 7 weeks follow-up]) A TEAE is defined as an AE starting on or after the time of first administration of IMP and up to and including 40 days after the final dose (or last contact depending on which occurs first). Adverse events starting before the date of the first administration of IMP were not considered TEAEs.
Nervous system disorders Myasthenia gravis	5.7% 5/88 • Number of events 6 • From Baseline (Day 1) to Safety follow-up visit (19 Weeks [12 weeks Treatment Period plus up to 7 weeks follow-up]) A TEAE is defined as an AE starting on or after the time of first administration of IMP and up to and including 40 days after the final dose (or last contact depending on which occurs first). Adverse events starting before the date of the first administration of IMP were not considered TEAEs.	2.3% 2/86 • Number of events 3 • From Baseline (Day 1) to Safety follow-up visit (19 Weeks [12 weeks Treatment Period plus up to 7 weeks follow-up]) A TEAE is defined as an AE starting on or after the time of first administration of IMP and up to and including 40 days after the final dose (or last contact depending on which occurs first). Adverse events starting before the date of the first administration of IMP were not considered TEAEs.
Pregnancy, puerperium and perinatal conditions Hyperemesis gravidarum	1.1% 1/88 • Number of events 1 • From Baseline (Day 1) to Safety follow-up visit (19 Weeks [12 weeks Treatment Period plus up to 7 weeks follow-up]) A TEAE is defined as an AE starting on or after the time of first administration of IMP and up to and including 40 days after the final dose (or last contact depending on which occurs first). Adverse events starting before the date of the first administration of IMP were not considered TEAEs.	0.00% 0/86 • From Baseline (Day 1) to Safety follow-up visit (19 Weeks [12 weeks Treatment Period plus up to 7 weeks follow-up]) A TEAE is defined as an AE starting on or after the time of first administration of IMP and up to and including 40 days after the final dose (or last contact depending on which occurs first). Adverse events starting before the date of the first administration of IMP were not considered TEAEs.
Respiratory, thoracic and mediastinal disorders Chronic obstructive pulmonary disease	1.1% 1/88 • Number of events 1 • From Baseline (Day 1) to Safety follow-up visit (19 Weeks [12 weeks Treatment Period plus up to 7 weeks follow-up]) A TEAE is defined as an AE starting on or after the time of first administration of IMP and up to and including 40 days after the final dose (or last contact depending on which occurs first). Adverse events starting before the date of the first administration of IMP were not considered TEAEs.	0.00% 0/86 • From Baseline (Day 1) to Safety follow-up visit (19 Weeks [12 weeks Treatment Period plus up to 7 weeks follow-up]) A TEAE is defined as an AE starting on or after the time of first administration of IMP and up to and including 40 days after the final dose (or last contact depending on which occurs first). Adverse events starting before the date of the first administration of IMP were not considered TEAEs.
Respiratory, thoracic and mediastinal disorders Pulmonary embolism	0.00% 0/88 • From Baseline (Day 1) to Safety follow-up visit (19 Weeks [12 weeks Treatment Period plus up to 7 weeks follow-up]) A TEAE is defined as an AE starting on or after the time of first administration of IMP and up to and including 40 days after the final dose (or last contact depending on which occurs first). Adverse events starting before the date of the first administration of IMP were not considered TEAEs.	1.2% 1/86 • Number of events 1 • From Baseline (Day 1) to Safety follow-up visit (19 Weeks [12 weeks Treatment Period plus up to 7 weeks follow-up]) A TEAE is defined as an AE starting on or after the time of first administration of IMP and up to and including 40 days after the final dose (or last contact depending on which occurs first). Adverse events starting before the date of the first administration of IMP were not considered TEAEs.
Skin and subcutaneous tissue disorders Angioedema	0.00% 0/88 • From Baseline (Day 1) to Safety follow-up visit (19 Weeks [12 weeks Treatment Period plus up to 7 weeks follow-up]) A TEAE is defined as an AE starting on or after the time of first administration of IMP and up to and including 40 days after the final dose (or last contact depending on which occurs first). Adverse events starting before the date of the first administration of IMP were not considered TEAEs.	1.2% 1/86 • Number of events 1 • From Baseline (Day 1) to Safety follow-up visit (19 Weeks [12 weeks Treatment Period plus up to 7 weeks follow-up]) A TEAE is defined as an AE starting on or after the time of first administration of IMP and up to and including 40 days after the final dose (or last contact depending on which occurs first). Adverse events starting before the date of the first administration of IMP were not considered TEAEs.

Other adverse events

Measure	Placebo n=88 participants at risk Participants self-administered zilucoplan (RA101495) matching placebo as subcutaneous (SC) injection during 12-week Treatment Period.	Zilucoplan 0.3 mg/kg n=86 participants at risk Participants self-administered zilucoplan (RA101495) 0.3 milligrams/kilogram/day (mg/kg/day) SC injection during 12-week Treatment Period.
Gastrointestinal disorders Diarrhoea	2.3% 2/88 • Number of events 2 • From Baseline (Day 1) to Safety follow-up visit (19 Weeks [12 weeks Treatment Period plus up to 7 weeks follow-up]) A TEAE is defined as an AE starting on or after the time of first administration of IMP and up to and including 40 days after the final dose (or last contact depending on which occurs first). Adverse events starting before the date of the first administration of IMP were not considered TEAEs.	10.5% 9/86 • Number of events 9 • From Baseline (Day 1) to Safety follow-up visit (19 Weeks [12 weeks Treatment Period plus up to 7 weeks follow-up]) A TEAE is defined as an AE starting on or after the time of first administration of IMP and up to and including 40 days after the final dose (or last contact depending on which occurs first). Adverse events starting before the date of the first administration of IMP were not considered TEAEs.
Gastrointestinal disorders Vomiting	5.7% 5/88 • Number of events 5 • From Baseline (Day 1) to Safety follow-up visit (19 Weeks [12 weeks Treatment Period plus up to 7 weeks follow-up]) A TEAE is defined as an AE starting on or after the time of first administration of IMP and up to and including 40 days after the final dose (or last contact depending on which occurs first). Adverse events starting before the date of the first administration of IMP were not considered TEAEs.	3.5% 3/86 • Number of events 3 • From Baseline (Day 1) to Safety follow-up visit (19 Weeks [12 weeks Treatment Period plus up to 7 weeks follow-up]) A TEAE is defined as an AE starting on or after the time of first administration of IMP and up to and including 40 days after the final dose (or last contact depending on which occurs first). Adverse events starting before the date of the first administration of IMP were not considered TEAEs.
General disorders Injection site bruising	9.1% 8/88 • Number of events 11 • From Baseline (Day 1) to Safety follow-up visit (19 Weeks [12 weeks Treatment Period plus up to 7 weeks follow-up]) A TEAE is defined as an AE starting on or after the time of first administration of IMP and up to and including 40 days after the final dose (or last contact depending on which occurs first). Adverse events starting before the date of the first administration of IMP were not considered TEAEs.	16.3% 14/86 • Number of events 18 • From Baseline (Day 1) to Safety follow-up visit (19 Weeks [12 weeks Treatment Period plus up to 7 weeks follow-up]) A TEAE is defined as an AE starting on or after the time of first administration of IMP and up to and including 40 days after the final dose (or last contact depending on which occurs first). Adverse events starting before the date of the first administration of IMP were not considered TEAEs.
General disorders Injection site pain	3.4% 3/88 • Number of events 3 • From Baseline (Day 1) to Safety follow-up visit (19 Weeks [12 weeks Treatment Period plus up to 7 weeks follow-up]) A TEAE is defined as an AE starting on or after the time of first administration of IMP and up to and including 40 days after the final dose (or last contact depending on which occurs first). Adverse events starting before the date of the first administration of IMP were not considered TEAEs.	9.3% 8/86 • Number of events 9 • From Baseline (Day 1) to Safety follow-up visit (19 Weeks [12 weeks Treatment Period plus up to 7 weeks follow-up]) A TEAE is defined as an AE starting on or after the time of first administration of IMP and up to and including 40 days after the final dose (or last contact depending on which occurs first). Adverse events starting before the date of the first administration of IMP were not considered TEAEs.
Infections and infestations Urinary tract infection	4.5% 4/88 • Number of events 4 • From Baseline (Day 1) to Safety follow-up visit (19 Weeks [12 weeks Treatment Period plus up to 7 weeks follow-up]) A TEAE is defined as an AE starting on or after the time of first administration of IMP and up to and including 40 days after the final dose (or last contact depending on which occurs first). Adverse events starting before the date of the first administration of IMP were not considered TEAEs.	8.1% 7/86 • Number of events 7 • From Baseline (Day 1) to Safety follow-up visit (19 Weeks [12 weeks Treatment Period plus up to 7 weeks follow-up]) A TEAE is defined as an AE starting on or after the time of first administration of IMP and up to and including 40 days after the final dose (or last contact depending on which occurs first). Adverse events starting before the date of the first administration of IMP were not considered TEAEs.
Infections and infestations Nasopharyngitis	3.4% 3/88 • Number of events 3 • From Baseline (Day 1) to Safety follow-up visit (19 Weeks [12 weeks Treatment Period plus up to 7 weeks follow-up]) A TEAE is defined as an AE starting on or after the time of first administration of IMP and up to and including 40 days after the final dose (or last contact depending on which occurs first). Adverse events starting before the date of the first administration of IMP were not considered TEAEs.	5.8% 5/86 • Number of events 5 • From Baseline (Day 1) to Safety follow-up visit (19 Weeks [12 weeks Treatment Period plus up to 7 weeks follow-up]) A TEAE is defined as an AE starting on or after the time of first administration of IMP and up to and including 40 days after the final dose (or last contact depending on which occurs first). Adverse events starting before the date of the first administration of IMP were not considered TEAEs.
Injury, poisoning and procedural complications Contusion	3.4% 3/88 • Number of events 3 • From Baseline (Day 1) to Safety follow-up visit (19 Weeks [12 weeks Treatment Period plus up to 7 weeks follow-up]) A TEAE is defined as an AE starting on or after the time of first administration of IMP and up to and including 40 days after the final dose (or last contact depending on which occurs first). Adverse events starting before the date of the first administration of IMP were not considered TEAEs.	8.1% 7/86 • Number of events 8 • From Baseline (Day 1) to Safety follow-up visit (19 Weeks [12 weeks Treatment Period plus up to 7 weeks follow-up]) A TEAE is defined as an AE starting on or after the time of first administration of IMP and up to and including 40 days after the final dose (or last contact depending on which occurs first). Adverse events starting before the date of the first administration of IMP were not considered TEAEs.
Investigations Lipase increased	1.1% 1/88 • Number of events 1 • From Baseline (Day 1) to Safety follow-up visit (19 Weeks [12 weeks Treatment Period plus up to 7 weeks follow-up]) A TEAE is defined as an AE starting on or after the time of first administration of IMP and up to and including 40 days after the final dose (or last contact depending on which occurs first). Adverse events starting before the date of the first administration of IMP were not considered TEAEs.	7.0% 6/86 • Number of events 6 • From Baseline (Day 1) to Safety follow-up visit (19 Weeks [12 weeks Treatment Period plus up to 7 weeks follow-up]) A TEAE is defined as an AE starting on or after the time of first administration of IMP and up to and including 40 days after the final dose (or last contact depending on which occurs first). Adverse events starting before the date of the first administration of IMP were not considered TEAEs.
Investigations Amylase increased	2.3% 2/88 • Number of events 2 • From Baseline (Day 1) to Safety follow-up visit (19 Weeks [12 weeks Treatment Period plus up to 7 weeks follow-up]) A TEAE is defined as an AE starting on or after the time of first administration of IMP and up to and including 40 days after the final dose (or last contact depending on which occurs first). Adverse events starting before the date of the first administration of IMP were not considered TEAEs.	5.8% 5/86 • Number of events 5 • From Baseline (Day 1) to Safety follow-up visit (19 Weeks [12 weeks Treatment Period plus up to 7 weeks follow-up]) A TEAE is defined as an AE starting on or after the time of first administration of IMP and up to and including 40 days after the final dose (or last contact depending on which occurs first). Adverse events starting before the date of the first administration of IMP were not considered TEAEs.
Nervous system disorders Headache	15.9% 14/88 • Number of events 19 • From Baseline (Day 1) to Safety follow-up visit (19 Weeks [12 weeks Treatment Period plus up to 7 weeks follow-up]) A TEAE is defined as an AE starting on or after the time of first administration of IMP and up to and including 40 days after the final dose (or last contact depending on which occurs first). Adverse events starting before the date of the first administration of IMP were not considered TEAEs.	15.1% 13/86 • Number of events 16 • From Baseline (Day 1) to Safety follow-up visit (19 Weeks [12 weeks Treatment Period plus up to 7 weeks follow-up]) A TEAE is defined as an AE starting on or after the time of first administration of IMP and up to and including 40 days after the final dose (or last contact depending on which occurs first). Adverse events starting before the date of the first administration of IMP were not considered TEAEs.
Nervous system disorders Myasthenia gravis	5.7% 5/88 • Number of events 6 • From Baseline (Day 1) to Safety follow-up visit (19 Weeks [12 weeks Treatment Period plus up to 7 weeks follow-up]) A TEAE is defined as an AE starting on or after the time of first administration of IMP and up to and including 40 days after the final dose (or last contact depending on which occurs first). Adverse events starting before the date of the first administration of IMP were not considered TEAEs.	9.3% 8/86 • Number of events 10 • From Baseline (Day 1) to Safety follow-up visit (19 Weeks [12 weeks Treatment Period plus up to 7 weeks follow-up]) A TEAE is defined as an AE starting on or after the time of first administration of IMP and up to and including 40 days after the final dose (or last contact depending on which occurs first). Adverse events starting before the date of the first administration of IMP were not considered TEAEs.
Skin and subcutaneous tissue disorders Rash	5.7% 5/88 • Number of events 9 • From Baseline (Day 1) to Safety follow-up visit (19 Weeks [12 weeks Treatment Period plus up to 7 weeks follow-up]) A TEAE is defined as an AE starting on or after the time of first administration of IMP and up to and including 40 days after the final dose (or last contact depending on which occurs first). Adverse events starting before the date of the first administration of IMP were not considered TEAEs.	3.5% 3/86 • Number of events 3 • From Baseline (Day 1) to Safety follow-up visit (19 Weeks [12 weeks Treatment Period plus up to 7 weeks follow-up]) A TEAE is defined as an AE starting on or after the time of first administration of IMP and up to and including 40 days after the final dose (or last contact depending on which occurs first). Adverse events starting before the date of the first administration of IMP were not considered TEAEs.

Additional Information

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Restriction type: GT60