Safety and Efficacy Study of Zilucoplan in Subjects With Immune-Mediated Necrotizing Myopathy

• Status: Terminated
• Trial end date: 2021-06-15
• Target enrollment: 0
• Participant gender: All

Summary

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.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Ra Pharmaceuticals

Criteria

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