Phase 3
Completed N=174
Safety, Tolerability, and Efficacy of Zilucoplan in Subjects With Generalized Myasthenia Gravis
Myasthenia Gravis, Generalized
Source: ClinicalTrials.gov NCT04115293 ↗
Enrolled (actual)
174
Serious AEs
13.8%
Results posted
Jan 2023
Primary outcomePrimary: Change From Baseline (CFB) to Week 12 in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Total Score — -2.30; -4.39 score on a scale — p=<0.001
◆ Published Evidence
Highly cited
254citations · ~85 / year
Safety and efficacy of zilucoplan in patients with generalised myasthenia gravis (RAISE): a randomised, double-blind, placebo-controlled, phase 3 study.
Summary
The RAISE study is a multicenter, randomized, double-blind, placebo controlled study to confirm the efficacy, safety, and tolerability of zilucoplan in subjects with generalized Myasthenia Gravis. Subjects will be randomized in a 1:1 ratio to receive daily SC doses of 0.3 mg/kg zilucoplan or placebo for 12 weeks.
Linked Publications (5)
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Safety and efficacy of zilucoplan in patients with generalised myasthenia gravis (RAISE): a randomised, double-blind, placebo-controlled, phase 3 study.
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Improvement of fatigue in generalised myasthenia gravis with zilucoplan.
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Changes in corticosteroid and non-steroidal immunosuppressive therapy with long-term zilucoplan treatment in generalized myasthenia gravis.
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Maintenance of zilucoplan efficacy in patients with generalised myasthenia gravis up to 24 weeks: a model-informed analysis.
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Efficacy of zilucoplan in patients with generalised myasthenia gravis who have not previously received immunoglobulin or plasma exchange: A subgroup analysis from the Phase 3 RAISE study.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change From Baseline (CFB) to Week 12 in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Total Score |
-2.30; -4.39 | <0.001 sig |
| SECONDARY Change From Baseline to Week 12 in the Quantitative Myasthenia Gravis (QMG) Total Score |
-3.25; -6.19 | <0.001 sig |
| SECONDARY Change From Baseline to Week 12 in the Myasthenia Gravis Composite (MGC) Scale Total Score |
-5.42; -8.62 | 0.0023 sig |
| SECONDARY Change From Baseline to Week 12 in the Myasthenia Gravis - Quality of Life Revised (MG-QoL15r) Scale Total Score |
-3.16; -5.65 | 0.0128 sig |
| SECONDARY Time to First Receipt of Rescue Therapy Over the 12-week Treatment Period |
NA; NA | — |
| SECONDARY Percentage of Participants Achieving Minimal Symptom Expression (MSE) at Week 12 Without Rescue Therapy |
5.8; 14.0 | 0.0885 |
| SECONDARY Percentage of Participants Achieving a ≥ 3-point Reduction in MG-ADL Score at Week 12 Without Rescue Therapy |
46.1; 73.1 | <0.001 sig |
| SECONDARY Percentage of Participants Achieving a ≥5-point Reduction in QMG Score Without Rescue Therapy at Week 12 |
33.0; 58.0 | 0.0012 sig |
| SECONDARY Percentage of Participants With Treatment-emergent Adverse Events (TEAEs) |
70.5; 76.7 | — |
Eligibility Criteria
Inclusion Criteria
- Diagnosis of gMG [Myasthenia Gravis Foundation of America (MGFA) Class II-IV] at Screening
- Positive serology for acetylcholine receptor (AChR) autoantibodies
- MG-ADL Score of ≥ 6 at Screening and Baseline
- QMG score ≥ 12 at Screening and Baseline
- No change in corticosteroid dose for at least 30 days prior to Baseline or anticipated to occur during the 12-week Treatment Period
- No change in immunosuppressive therapy, including dose, for at least 30 days prior to Baseline or anticipated to occur during the 12-week Treatment Period
Exclusion Criteria
- Thymectomy within 12 months prior to Baseline or scheduled to occur during the 12 week Treatment Period
- History of meningococcal disease
- Current or recent systemic infection within 2 weeks prior to Baseline or injection requiring intravenous (IV) antibiotics within 4 weeks prior to Baseline
Data sourced from ClinicalTrials.gov (NCT04115293) and the linked publication. Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.