NCT02753530已完成2 期
Phase II Study of Arimoclomol for the Treatment of Sporadic Inclusion Body Myositis (IBM)
ZevraDenmark12 个研究点 分布在 2 个国家目标入组 152 人开始时间: 2017年8月16日最近更新:
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 152
- 试验地点
- 12
- 主要终点
- Change in Inclusion Body Myositis Functional Rating Scale (IBMFRS) Total Score
研究概览
简要总结
Funding Source - FDA Office of Orphan Products Development (OOPD). The purpose of this study is to evaluate the safety and efficacy of the study drug, arimoclomol in IBM patients.
详细描述
A Phase 2/3, randomized, double-blind, placebo-controlled, international, 20-month intervention study in patients with IBM.
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
- 45 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Meet any of the European Neuromuscular Centre Inclusion Body Myositis research diagnostic criteria 2011 categories for IBM.
- •Demonstrate being able to arise from a chair without support from another person or device.
- •Able to ambulate at least 20 ft/6 meters with or without assistive device. Once arisen from the chair, participant may use any walking device, i.e. walker/frame, can, crutches, or braces. They cannot be supported by another person and cannot use furniture or wall for support.
- •Age at onset of weakness >45 years.
- •Body weight of >= 40 kg.
- •Able to give informed consent.
排除标准
- •History of any of the following excludes subject participation in the study: chronic infection particularly HIV or Hepatitis B or C; cancer other than basal cell cancer less than five years prior; or other chronic serious medical illnesses.
- •Presence of any of the following on routine blood screening: White blood cells (WBC) <3000; platelets < 100,000; hematocrit <30%; blood urea nitrogen (BUN) >30 mg/dL; creatinine >1.5 x upper limit of normal; symptomatic liver disease with serum albumin <3 g/dL.
- •History of most recent creatine kinase >15x the upper limit of normal without any other explanation besides IBM.
- •History of non-compliance with other therapies.
- •Use of testosterone except for physiologic replacement doses in case of androgen deficiency. Participants must have documented proof of the androgen deficiency.
- •Coexistence of other disease that would be likely to affect outcome measures
- •Drug or alcohol abuse within past three months.
- •Participation in a recent drug study in the last 30 days prior to the screening visit or use of a biologic agent less than 6 months prior to the screening visit.
- •Women who are lactating or unwilling to use adequate method of birth control who are not surgically sterile. Adequate birth control includes use of intrauterine device, abstinence, or oral contraceptives or a double barrier method, e.g condom plus diaphragm will be necessary for both male and female participants.
- •Participants taking >7.5 mg prednisolone or equivalent or participants on Intravenous Immunoglobulin Therapy (IVIg) or other immunosuppressants within the last 3 months.
- •Clinically significant renal or hepatic disease, as indicated by clinical laboratory assessment.
研究组 & 干预措施
Arimoclomol
Experimental
248 mg arimoclomol base (equivalent to 400 mg arimoclomol citrate) 3 times daily
干预措施: Arimoclomol (Drug)
Placebo
Placebo Comparator
248 mg matching placebo 3 times daily
干预措施: Placebo (Other)
结局指标
主要结局
Change in Inclusion Body Myositis Functional Rating Scale (IBMFRS) Total Score
时间窗: Change from Baseline to Month 20
Measured by rate of decline in the IBMFRS between experimental and placebo groups. The IBMFRS is a 10-item questionnaire. Scores for each item range from 0 to 4. There is a total maximum score of 40 and minimum score of 0. The higher the score the better functional status of the person.
次要结局
- Health Assessment Questionnaire - Disability Index (HAQ-DI)(Change from Baseline to Month 12 and Month 20)
- Falls and Near Falls(Accumulated number from Baseline to Month 20)
- Modified Timed up and go (mTUG)(Change from Baseline to Month 12 and Month 20)
- Short Form-36 (SF-36) Mental Component Score(Change from Baseline to Month 12 and Month 20)
- Change in Inclusion Body Myositis Functional Rating Scale (IBMFRS) Total Score(Change from Baseline to Month 12)
- Grip Strength(Change from Baseline to Month 12 and 20)
- 6 Minute Walk Test (6MWT); Distance After 6 Minutes (6MWD)(Change from Baseline to Month 12 and Month 20)
- Short Form-36 (SF-36) Physical Component Score(Change from Baseline to Month 12 and Month 20)
- Patient Global Impression of Severity (PGIS)(Change from Baseline to Month 12 and Month 20)
- Clinician Global Impression of Severity (CGIS)(Change from Baseline to Month 12 and Month 20)
- Maximum Voluntary Isometric Contraction (MVICT) of Quadriceps(Change from Baseline to Month 12 and Month 20)
- Patient Global Impression of Change (PGIC)(Change from Baseline to Month 12 and Month 20)
- Clinician Global Impression of Change (CGIC)(Change from Baseline to Month 12 and Month 20)
- Manual Muscle Testing (MMT), Total Score(Change from Baseline to Month 12 and Month 20)
- 2 Minute Walk Test (2MWT)(Change from Baseline to Month 12 and Month 20)
研究者
研究点 (12)
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