A Phase 2, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety, Tolerability, and Efficacy of Zilucoplan in Subjects with Immune-Mediated Necrotizing Myopathy

Ra Pharmaceuticals, Inc.0 个研究点目标入组 24 人开始时间: 2020年1月21日最近更新: 2022年3月14日

适应症

试验速览

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•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

•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.

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