Safety and Efficacy Study of Zilucoplan in Subjects With Immune-Mediated Necrotizing Myopathy
Purpose
The purpose of the study is to evaluate the safety and efficacy of zilucoplan in patients with Immune-Mediated Necrotizing Myopathy (IMNM). Subjects will be randomized in a 1:1 ratio to receive daily SC doses of 0.3 mg/kg zilucoplan or matching placebo for 8 weeks.
Condition
- Immune Mediated Necrotizing Myopathy
Eligibility
- Eligible Ages
- Between 18 Years and 75 Years
- Eligible Genders
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- Clinical diagnosis of IMNM (Immune-Mediated Necrotizing Myopathy) - Positive serology for anti-3-hydroxy-3-methyl-glutaryl-coenzyme A reductase (HMGCR) or anti-signal recognition particle (SRP) autoantibodies - Clinical evidence of weakness (≤ grade 4 out of 5) on manual muscle testing in at least one proximal limb muscle group - Creatine kinase (CK) of >1000 U/L at Screening - No change in corticosteroid dose for at least 30 days prior to Baseline or anticipated to occur during the first 8-weeks on study - No changes in immunosuppressive therapy, including dose, for at least 30 days prior to Baseline or anticipated to occur during the first 8-weeks on study
Exclusion Criteria
- History of meningococcal disease - Current or recent systemic infection within 2 weeks prior to Screening or infection requiring intravenous (IV) antibiotics within 4 weeks prior to Screening - Recent initiation of intravenous immunoglobulin (IVIG) (i.e., first cycle administered less than 90 days prior to Baseline) - Rituximab use within 90 days prior to Baseline or anticipated to occur during study - Statin use within 30 days prior to Baseline or anticipated to occur during study - Plasma exchange within 4 weeks prior to Baseline or expected to occur during the 8-week Treatment Period
Study Design
- Phase
- Phase 2
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel Assignment
- Primary Purpose
- Treatment
- Masking
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Arm Groups
Arm | Description | Assigned Intervention |
---|---|---|
Experimental 0.3 mg/kg zilucoplan |
Daily subcutaneous (SC) injection |
|
Placebo Comparator Placebo |
Daily subcutaneous (SC) injection |
|
More Details
- Status
- Terminated
- Sponsor
- Ra Pharmaceuticals