A Phase 2/3, Double-Blind, Randomized, Placebo-Controlled, Parallel Group, Multicenter Study to Evaluate the Efficacy and Safety of Ravulizumab in Adult Participants With Dermatomyositis
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 入组人数
- 38
- 试验地点
- 1
- 主要终点
- Number of Participants With International Myositis Assessment and Clinical Studies Total Improvement Score (IMACS-TIS) (TIS40) Response at Week 26 of the Randomized Controlled Period
研究概览
简要总结
This is a Phase 2/3, double-blind, randomized, placebo-controlled, parallel group, multicenter study to evaluate the efficacy, safety, pharmacokinetics, pharmacodynamics, and immunogenicity of ravulizumab in adult participants with dermatomyositis (DM).
详细描述
The study will be conducted in 2 parts: Part A (Phase 2) and Part B (Phase 3). There will be 3 periods in both Part A and Part B of this study: Screening Period, Randomized Controlled Period, and Open-Label Extension Period.
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
- Double (Participant, Investigator)
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •18 years of age or older at the time of signing the informed consent.
- •Body weight ≥ 30 kilograms at the time of Screening.
- •Male or female.
- •Diagnosis: Meet 2017 American College of Rheumatology/European League Against Rheumatism classification criteria for definite or probable DM.
- •Participants who have an inadequate response or are intolerant to 1 or more DM treatments, including systemic corticosteroids or immunosuppressive/immunomodulatory therapies (for example, azathioprine, methotrexate, rituximab, intravenous immunoglobulin), either in combination or as monotherapy.
- •Vaccinated against Neisseria meningitidis within 3 years prior to initiating ravulizumab as per national and local guidelines. Participants must receive the vaccination at least 2 weeks before first study intervention. The sponsor recommends that national and local guidelines for prophylactic antibiotics should also be followed.
- •Female participants of childbearing potential and male participants must follow specified contraception guidance as described in the protocol.
排除标准
- •Participants who have been diagnosed with cancer within the last 3 years need to have appropriate negative cancer screening as per local standard of care within 6 months before Screening (basal or squamous cell skin cancer or carcinoma in situ of the cervix needs to have been excised and without evidence of residual disease for at least 3 months before Screening).
- •Evidence of active malignant disease or malignancies diagnosed within the previous 3 years including hematological malignancies and solid tumors.
- •Participants with other forms of myositis.
- •As per investigator discretion, participants with significant muscle damage (for example, severe muscle atrophy, end stage muscle disease, MRI with severe atrophy or fibrofatty replacement)
- •History of Neisseria meningitidis infection.
- •Human immunodeficiency virus (HIV) infection (evidenced by HIV Type 1 or Type 2 antibody titer).
- •Active systemic bacterial, viral, or fungal infection within 14 days prior to ravulizumab administration.
- •Presence of fever ≥ 38°Celsius (100.4°Fahrenheit) within 7 days prior to study drug administration on Day
- •History of hypersensitivity to murine proteins or to 1 of the excipients of ravulizumab.
- •Pregnant, breastfeeding, or intending to conceive during the course of the study.
- •Inability or unwillingness to adhere to the protocol requirements.
研究组 & 干预措施
Ravulizumab
Participants will receive ravulizumab in both Parts A and B.
干预措施: Ravulizumab (Drug)
Placebo
Participants will receive placebo in both Parts A and B.
干预措施: Placebo (Drug)
结局指标
主要结局
Number of Participants With International Myositis Assessment and Clinical Studies Total Improvement Score (IMACS-TIS) (TIS40) Response at Week 26 of the Randomized Controlled Period
时间窗: Week 26
Data are presented for the number of participants with a TIS40 response, defined as an IMACS-TIS score ≥ 40 at Week 26. IMACS-TIS is a clinical instrument that encompasses 6 core set measure (CSMs) (physician, patient, extra-muscular global activity, muscle strength, Health Assessment Questionnaire \[HAQ\], and muscle enzyme levels). A Total Improvement Score (TIS: 0-100), was determined by summing scores in each CSM, and was based on the improvement and relative weight of each CSM. A higher score indicated greater improvement. TIS40 was considered a moderate improvement score.
次要结局
- TIS at Week 26(Week 26)
- Change From Baseline In Cutaneous Dermatomyositis Disease Area And Severity Index (CDASI) Activity Score at Week 26(Baseline, Week 26)
- Number of Participants With Response Related to Muscle Enzymes: Normalization of Most Abnormal Baseline Enzyme at Week 26(Baseline, Week 26)
- Change From Baseline In IMACS CSMs: Extra-Muscular Disease Activity Based on Myositis Disease Activity Assessment Tool (MDAAT) at Week 26(Baseline, Week 26)
- Change From Baseline In IMACS CSMs: Physician Global Activity Assessment at Week 26(Baseline, Week 26)
- Change From Baseline In IMACS CSMs: Patient Global Activity Assessment at Week 26(Baseline, Week 26)
- Change From Baseline In IMACS CSMs: Manual Muscle Testing Subset 8 Muscles (MMT-8) at Week 26(Baseline, Week 26)
- Change From Baseline In IMACS CSMs: Health Assessment Questionnaire (HAQ) at Week 26(Baseline, Week 26)
- Number of Participants With CDASI Response (>=7-point Improvement) at Week 26(Week 26)
- Number of Participants With Cutaneous Dermatomyositis Activity Physician's Global Assessment (CDA-IGA) Response at Week 26(Week 26)
- Number of Participants With ≥ 20-Point Improvement Response on IMACS-TIS (TIS20) Response at Week 26(Week 26)
- Number of Participants With ≥ 60-Point Improvement Response on IMACS-TIS (TIS60) Response at Week 26(Week 26)
- Time to First Response of TIS20, TIS40, or TIS60(Baseline through Week 26)
- Number of Participants With Clinical Worsening (CW) During the RCP At 2 Consecutive Visits(Baseline through Week 26)
- Number of Participants Who Received Acute Rescue Therapy With Standard DM Treatment(Baseline through Week 26)