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

Purpose

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.

Condition

  • Myasthenia Gravis, Generalized

Eligibility

Eligible Ages
Between 18 Years and 74 Years
Eligible Genders
All
Accepts Healthy Volunteers
No

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

Study Design

Phase
Phase 3
Study Type
Interventional
Allocation
Randomized
Intervention Model
Parallel Assignment
Primary Purpose
Treatment
Masking
Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Arm Groups

ArmDescriptionAssigned Intervention
Experimental
0.3 mg/kg zilucoplan (RA101495)
  • Drug: zilucoplan (RA101495)
    Daily subcutaneous (SC) injection
Placebo Comparator
Placebo
  • Drug: Placebo
    Daily subcutaneous (SC) injection

More Details

Status
Completed
Sponsor
Ra Pharmaceuticals, Inc.

Study Contact