A Multicenter, Open-Label Phase 2 Extension Trial to Characterize the Long-term Safety and Tolerability of Subcutaneous Elamipretide in Subjects With Genetically Confirmed Primary Mitochondrial Myopathy (PMM)
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 入组人数
- 28
- 试验地点
- 4
- 主要终点
- Distance Walked on Six Minute Walk Test
研究概览
简要总结
This is a Phase 2 Open-Label extension study to evaluate the long term safety and tolerability of daily elamipretide injections in patients with genetically confirmed Primary Mitochondrial Disease who previously participated in the SPIMM-202 Clinical Trial
详细描述
This open-label, non-comparative, extension trial will enroll subjects with genetically confirmed PMD who have completed the End-of-Study Visit in the SPIMM-202 trial. Subjects who do not discontinue or withdraw from the trial will receive treatment with 40 mg SC elamipretide for the shortest of the following: 260 weeks; regulatory approval and commercial availability of elamipretide in the subject's respective country; or termination of the clinical development for elamipretide in subjects with PMD.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 16 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Investigator determines the subject can, and subject agrees to, adhere to the trial requirements for the length of the trial including self-administration (by subject or trained caregiver) of the study drug
- •Subject completed the End-of-Study Visit in SPIMM-202
排除标准
- •Subject has any prior or current medical condition that, in the judgment of the Investigator, would prevent the subject from safely participating in and/or completing all trial requirements
- •Subject has received any investigational compound (excluding elamipretide) and/or has participated in another interventional clinical trial within 30 days prior to the SPIMM-203 Baseline Visit (excluding SPIMM-202) or is concurrently enrolled in any non-interventional research of any type judged to be scientifically or medically incompatible with the trial as deemed by the Investigator in consultation with the Sponsor
- •Subject experienced an adverse reaction attributed to study drug resulting in permanent discontinuation of study drug in the SPIMM-202 trial.
- •Female subjects who are pregnant, planning to become pregnant, or lactating
- •Subject has undergone an in-patient hospitalization within the 1 month prior to the SPIMM-203 Baseline Visit
研究组 & 干预措施
elamipretide
Open-label once daily subcutaneous injection of 40mg elamipretide
干预措施: elamipretide (Drug)
结局指标
主要结局
Distance Walked on Six Minute Walk Test
时间窗: Baseline, Month 3, Month 6 and Every 26 weeks for up to 160 weeks
Change From Baseline in Distance Walked (meters) on the Six-Minute Walk Test by Visit
次要结局
- Work Limitations Questionnaire(Baseline, Month 3, Month 6 and Every 26 weeks for up to 160 weeks)
- Physician Global Assessment (PhGA): Categorical(Baseline, Month 3, Month 6 and Every 26 weeks for up to 160 weeks)
- Total Fatigue Score_Primary Mitochondrial Myopathy Symptom Assessment (PMMSA)(Baseline, Month 3, Month 6 and Every 26 weeks for up to 160 weeks)
- EuroQol 5 Dimensions 5 Levels (EQ-5D-5L)(Baseline, Month 3, Month 6 and Every 26 weeks for up to 160 weeks)
- Patient Global Assessment : Categorical(Baseline, Month 3, Month 6 and Every 26 weeks for up to 160 weeks)
- Patient Global Assessment: Continuous Variable(Baseline, Month 3, Month 6 and Every 26 weeks for up to 160 weeks)
- Euroquol Visual Analog Scale(Baseline, Month 3, Month 6 and Every 26 weeks for up to 160 weeks)
- NeuroQOL Fatigue Questionnaire(Baseline, Month 3, Month 6 and Every 26 weeks for up to 160 weeks)
- Physician Global Assessment Score (PhGA) by Visit(Baseline, Month 3, Month 6 and Every 26 weeks for up to 160 weeks)
- PMMSA Individual Items(Baseline, Week 13, 26, 52, 104, 156, End of Study (Week 160))
- Total Fatigue Score During Activities_Primary Mitochondrial Myopathy Symptom Assessment (PMMSA)(Baseline, Month 3, Month 6 and Every 26 weeks for up to 160 weeks)