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
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 入组人数
- 27
- 试验地点
- 18
- 主要终点
- Percentage Change From Baseline to Week 8 in Serum Creatine Kinase (CK) Levels
研究概览
简要总结
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 18 Years 至 75 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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
排除标准
- •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
研究组 & 干预措施
0.3 mg/kg zilucoplan
Daily subcutaneous (SC) injection
干预措施: zilucoplan (Drug)
Placebo
Daily subcutaneous (SC) injection
干预措施: Placebo (Other)
结局指标
主要结局
Percentage Change From Baseline to Week 8 in Serum Creatine Kinase (CK) Levels
时间窗: Baseline (Day 1) and end of Main Portion (Week 8)
All laboratory samples were obtained prior to administration of study drug at applicable visits. CK levels were measured by a central laboratory.
Number of Participants Who Experienced a Treatment-Emergent Adverse Event (TEAE)
时间窗: Baseline (Day 1) to end of Main Portion (Week 8)
A TEAE was defined as: * An adverse event (AE) that occurred after study treatment start that was not present at the time of treatment start. * An AE that increased in severity after treatment start if the event was present at the time of treatment start.
次要结局
- Change From Baseline to Week 8 in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale Score(Baseline (Day 1) and end of Main Portion (Week 8))
- Change From Baseline to Week 8 in Proximal Manual Muscle Testing (MMT) Score(Baseline (Day 1) and end of Main Portion (Week 8))
- Change From Baseline to Week 8 in Triple Timed Up and Go Test (3TUG) Time(Baseline (Day 1) and end of Main Portion (Week 8))
- Change From Baseline to Week 8 in Physician Global Activity Visual Analogue Scale (VAS) Score(Baseline (Day 1) and end of Main Portion (Week 8))
- Change From Baseline to Week 8 in Patient Global Activity VAS Score(Baseline (Day 1) and end of Main Portion (Week 8))
- Change From Baseline to Week 8 in HAQ Score(Baseline (Day 1) and end of Main Portion (Week 8))
- Number of Participants Who Achieve at Least Minimal Response Based on the American College of Rheumatology/European League Against Rheumatism (ACR/EULAR) Response Criteria Scale(Baseline (Day 1) and end of Main Portion (Week 8))
- Change From Baseline to Week 8 in MDAAT Extramuscular Disease Activity VAS Score(Baseline (Day 1) and end of Main Portion (Week 8))