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

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

More Details

Status
Terminated
Sponsor
Ra Pharmaceuticals

Study Contact