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

Stopped early · Phase 2 · Has a placebo group

Conditions studied: Immune Mediated Necrotizing Myopathy

In brief

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.

Key facts

Study ID

NCT04025632

Run by

Ra Pharmaceuticals

People needed

27

Starts

2019-11-07

Expected to finish

2021-06-14

Last updated by the study team

2022-07-27

Who can join

Age: 18 and older, up to 75. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• 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

Where it is running

• University of California Los Angeles — Los Angeles, California, United States
• University of California Irvine — Orange, California, United States
• University of Florida Health Jacksonville — Jacksonville, Florida, United States
• University of South Florida — Tampa, Florida, United States
• University of Kansas Medical Center — Kansas City, Kansas, United States
• National Institute of Health — Bethesda, Maryland, United States
• Brigham and Women's Hospital — Boston, Massachusetts, United States
• Washington University School of Medicine in Saint Louis — St Louis, Missouri, United States
• Northwell Health Neuroscience Institute — Great Neck, New York, United States
• The Ohio State University — Columbus, Ohio, United States
• University of Pennsylvania — Philadelphia, Pennsylvania, United States
• Wesley Neurology Clinic — Memphis, Tennessee, United States
• Austin Neuromuscular Center — Austin, Texas, United States
• The University of Texas Health Science Center at Houston — Houston, Texas, United States
• Hôpital Universitaire Pitié Salpêtrière — Paris, France
• Amsterdam UMC — Amsterdam, Netherlands
• University College London Hospitals NHS Foundation Trust — London, United Kingdom
• Salford Royal NHS Barnes Clinical Research Facility — Manchester, United Kingdom

Full record on ClinicalTrials.gov

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