Safety and Efficacy Study of RA101495 in Subjects With Generalized Myasthenia Gravis

Ra Pharmaceuticals

Status and phase: Completed; Phase 2

Conditions: Generalized Myasthenia Gravis

Treatments: Drug: Placebo; Drug: zilucoplan (RA101495)

Study type: Interventional

Funder types: Industry

Identifiers

NCT03315130

RA101495-02.201

Details and patient eligibility

About

The purpose of the study is to evaluate the safety and efficacy of RA101495 in patients with generalized Myasthenia Gravis (gMG). Subjects will be randomized in a 1:1:1 ratio to receive daily SC doses of 0.1 mg/kg RA101495, 0.3 mg/kg RA101495, or matching placebo for 12 weeks.

Enrollment: 45 patients

Sex: All

Ages: 18 to 85 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Diagnosis of gMG [Myasthenia Gravis Foundation of America (MGFA) Class II-IVa] at Screening
• Positive serology for acetylcholine receptor (AChR) autoantibodies
• QMG score ≥ 12 at Screening and Randomization
• No change in corticosteroid dose for at least 30 days prior to Randomization or anticipated to occur during the 12-week Treatment Period
• No change in immunosuppressive therapy, including dose, for at least 30 days prior to Randomization or anticipated to occur during the 12-week Treatment Period

Exclusion criteria

• Thymectomy within 6 months prior to Randomization or scheduled to occur during the 12 week Treatment Period
• History of meningococcal disease
• Current or recent systemic infection within 2 weeks prior to Randomization or infection requiring intravenous (IV) antibiotics within 4 weeks prior to Randomization

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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45 participants in 3 patient groups, including a placebo group

0.1 mg/kg zilucoplan (RA101495)

Experimental group

Treatment:

Drug: zilucoplan (RA101495)

0.3 mg/kg zilucoplan (RA101495)

Experimental group

Treatment:

Drug: zilucoplan (RA101495)

Placebo

Placebo Comparator group

Treatment:

Drug: Placebo