A Phase 3, Multicenter, Randomized, Double Blind, Placebo-Controlled Study to Confirm the Safety, Tolerability, and Efficacy of Zilucoplan in Subjects With Generalized Myasthenia Gravis

RA101495-02.301

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.

2019-09-17

2021-12-30

2021-12-30

Completed

Early phase I

174

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

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE3'], 'designInfo': {'allocation': 'RANDOMIZED', 'interventionModel': 'PARALLEL', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'QUADRUPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR', 'OUTCOMES_ASSESSOR']}}, 'enrollmentInfo': {'count': 174, 'type': 'ACTUAL'}}

2024-05-02