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

Ra Pharmaceuticals

Status and phase

Completed

Phase 3

Conditions

Myasthenia Gravis, Generalized

Treatments

Drug: zilucoplan (RA101495)

Drug: Placebo

Study type

Interventional

Funder types

Industry

Identifiers

NCT04115293

RA101495-02.301

Details and patient eligibility

About

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.

Enrollment

174 patients

Sex

All

Ages

18 to 74 years old

Volunteers

No Healthy Volunteers

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