A Study to Evaluate the Safety, Tolerability, and Efficacy of Zilucoplan in Subjects with Immune-Mediated Necrotizing Myopathy

Phase 1

• Conditions: Immune-Mediated Necrotizing Myopathy; MedDRA version: 21.1Level: LLTClassification code 10072156Term: Immune-mediated necrotizing myopathySystem Organ Class: 100000004859; Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

• Registration Number: EUCTR2019-001497-29-FR
• Lead Sponsor: Ra Pharmaceuticals, Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2019-06-27
• Last Update Posted: 2 March 2022

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 24

Inclusion Criteria

To be eligible for this study, subjects must meet ALL of the following inclusion criteria:
1. Clinical diagnosis of IMNM (Immune-Mediated Necrotizing Myopathy).
2. Positive serology for anti-HMGCR or anti-SRP autoantibodies.
3. Clinical evidence of weakness (= grade 4 out of 5) on manual muscle testing in at least one proximal limb muscle group.
4. CK (creatine kinase) of >1000 U/L at Screening.
5. No change in corticosteroid dose for at least 30 days prior to Baseline or anticipated to occur during the first 8-weeks on study.
6. 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.

Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 18
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 6

Exclusion Criteria

Subjects who meet ANY of the following exclusion criteria must be excluded from the study:
1. History of meningococcal disease.
2. Current or recent systemic infection within 2 weeks prior to Screening or infection requiring intravenous (IV) antibiotics within 4 weeks prior to Screening.
3. Statin use within 30 days prior to Baseline or anticipated to occur during study.
4. Rituximab use within 90 days prior to Baseline or anticipated to occur during study.
5. Recent initiation of intravenous immunoglobulin (IVIG) (i.e., first cycle administered less than 90 days prior to Baseline).
6. Plasma exchange within 4 weeks prior to Baseline or expected to occur during the 8-week Treatment Period.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified