A Randomized, Double-Blind, Controlled, Phase 2/3 Study to Assess Efficacy, Long Term Safety and Tolerability of RT001 in Subjects With Friedreich's Ataxia
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 65
- 试验地点
- 6
- 主要终点
- Change From Baseline to 11 Months in Maximum Consumption of Oxygen (mlO2/kg/Min) Using Cardiopulmonary Exercise Testing (CPET)
研究概览
简要总结
The purpose of this study is to assess the Efficacy, Long Term Safety and Tolerability of RT001 in subjects with Friedreich's Ataxia
详细描述
This is a randomized, double-blind, placebo-controlled study to evaluate the efficacy, safety, tolerability, in subjects with FRDA following the oral administration of study drug (active or placebo capsules). Sixty eligible patients will undergo various assessments at different time points during the the study. The study duration is 13 months which includes screening, treatment and safety follow up phone call.
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
- 12 Years 至 50 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female 12 to 50 years of age
- •Medical history consistent with the symptoms of FRDA at ≤ 25 years of age
- •Detection of biallelic pathogenic variants in frataxin gene (FXN)
- •Ambulatory (with or without assistive device) and capable of performing other assessments/evaluations.
- •Must be able to walk 25 feet during the timed 1-minute walk
排除标准
- •Received treatment with other experimental therapies within the last 30 days prior to the first dose
- •Previously participated in the RT001 trial
- •Refusal to discontinue fish oils or other oil-based supplements for the duration of the study (Screening till last study procedure completed)
- •History of malignancies (other than basal cell carcinomas)
- •Inability to complete CPET protocol
- •Female who is breastfeeding or has a positive pregnancy test
- •History of uncontrolled diabetes mellitus (Type 1 or 2)
研究组 & 干预措施
RT001
- 9 capsules daily (8.64 g total dose) given as 3 capsules three times a day (TID) with meals for the first month of treatment.
- Six capsules daily (5.76 g total dose) given as 3 capsules (BID) with breakfast, and 3 capsules with dinner after the first month of treatment
干预措施: RT001 (Drug)
Placebo
- 9 capsules daily (8.64 g total dose) given as 3 capsules three times a day (TID) with meals for the first month of treatment.
- Six capsules daily (5.76 g total dose) given as 3 capsules (BID) with breakfast, and 3 capsules with dinner after the first month of treatment
干预措施: Placebo (Drug)
结局指标
主要结局
Change From Baseline to 11 Months in Maximum Consumption of Oxygen (mlO2/kg/Min) Using Cardiopulmonary Exercise Testing (CPET)
时间窗: 11 months
To measure the change in MVO2 from baseline to Month 11 using CPET
次要结局
- Change From Baseline in the Timed 1 Minute Walk Distance(11 months)