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

Ra Pharmaceuticals

Status and phase: Terminated; Phase 2

Conditions: Immune Mediated Necrotizing Myopathy

Treatments: Other: Placebo; Drug: zilucoplan

Study type: Interventional

Funder types: Industry

Identifiers

NCT04025632

RA101495-02.202

Details and patient eligibility

About

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.

Enrollment: 27 patients

Sex: All

Ages: 18 to 75 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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27 participants in 2 patient groups, including a placebo group

0.3 mg/kg zilucoplan

Experimental group

Description:

Daily subcutaneous (SC) injection

Treatment:

Drug: zilucoplan

Placebo

Placebo Comparator group

Description:

Daily subcutaneous (SC) injection

Treatment:

Other: Placebo