Immune-Mediated Necrotizing Myopathy MedDRA version: 21.1 Level: LLT Classification code 10072156 Term: Immune-mediated necrotizing myopathy System Organ Class: 100000004859
Conditions
Sponsors
Ra Pharmaceuticals, Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
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
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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: • To evaluate the safety and tolerability of zilucoplan in subjects with IMNM • To evaluate the efficacy of zilucoplan in subjects with IMNM;Secondary Objective: Not Applicable;Primary end point(s): Primary Efficacy Endpoint: • Percent change from Baseline to Week 8 in CK levels.;Timepoint(s) of evaluation of this end point: week 8 | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Secondary Efficacy Endpoints: • Change from Baseline to Week 8 in Triple Timed Up and Go (3TUG) Test (in ambulatory subjects only) • Change from Baseline to Week 8 in Proximal Manual Muscle Testing (MMT) • Change from Baseline to Week 8 in Physician Global Activity Visual Analogue Scale (VAS) • Change from Baseline to Week 8 in Patient Global Activity VAS • Change from Baseline to Week 8 in Health Assessment Questionnaire (HAQ) • Change from Baseline to Week 8 in Myositis Disease Activity Assessment Tool (MDAAT) Score • Change from Baseline to Week 8 in American College of Rheumatology/European League Against Rheumatism (ACR/EULAR) Response Criteria • Change from Baseline to Week 8 in FACIT-Fatigue Scale ;Timepoint(s) of evaluation of this end point: week 8 | — |
Countries
France, Netherlands, Sweden, United Kingdom, United States
Contacts
Public ContactClinical Trial Information
Ra Pharmaceuticals, Inc.
Outcome results
None listed