NCT02445794
已完成
1 期
A Randomized, Double-blind, Controlled Study to Assess the Safety, Tolerability, and Pharmacokinetics of RT001 in Patients With Friedreich's Ataxia
概览
- 阶段
- 1 期
- 干预措施
- Low dose cohort
- 疾病 / 适应症
- Friedreich's Ataxia
- 发起方
- Retrotope, Inc.
- 入组人数
- 19
- 试验地点
- 2
- 主要终点
- Number of Patients With Adverse Events
- 状态
- 已完成
- 最后更新
- 5年前
概览
简要总结
The purpose of this study is to evaluate the safety, tolerability and pharmacokinetics of RT001 in patients with Friedreich's ataxia.
详细描述
Study RT001-002 is a randomized, double-blind, controlled, ascending dose study to evaluate the safety, tolerability, pharmacokinetic, disease state, and exploratory endpoints in patients with Friedreich's ataxia after oral administration. The study includes 2 dose levels of RT001.
研究者
入排标准
入选标准
- •Male or female 18 to 50 years of age
- •Medical history consistent with the symptoms of FRDA at ≤ 25 years of age
- •Homozygous for GAA repeat expansions in the Frataxin gene in the affected range for FRDA
- •FARS-Neurological score of 20-90 points
- •Ambulatory (with or without assistive device) and capable of performing assessments/evaluations
- •Body Mass Index ≤ 29.9 kg/m2
- •Agrees to dietary restrictions and agrees to receive calls from a dietary coach
- •Signed the informed consent form prior to entry into the study
- •Agrees to spend the required number of overnight clinic days
- •Able to provide the necessary repeated blood samples
排除标准
- •Received treatment with other experimental therapies within the last 30 days prior to the first dose
- •Known point mutation in the FXN gene
- •History of malignancies (other than basal cell carcinomas)
- •Impaired renal function at screening
- •Alanine transaminase (ALT) or aspartate transaminase (AST) laboratory values \> 2 x upper limit of normal (ULN) at screening
- •Known hepatitis B surface antigen (HBsAg)-positive, or known or suspected active hepatitis C infection, or is known to be human immunodeficiency virus (HIV) positive
- •Female who is breastfeeding or has a positive pregnancy test
- •Male participant or female participant of child bearing potential, who is sexually active and unwilling/unable to use a medically acceptable and effective double barrier birth control method throughout the study
- •Unwilling or unable to comply with the requirements of the protocol
- •Clinically significant cardiac abnormalities at screening that, in the opinion of the Investigator, would make the patient unsuitable for enrollment
研究组 & 干预措施
RT001, oral, 1.8 g/day
RT001, oral, 1.8 g QD for 28 days or matching comparator
干预措施: Low dose cohort
RT001, oral, 1.8 g/day
RT001, oral, 1.8 g QD for 28 days or matching comparator
干预措施: High dose cohort
RT001, oral, 9 g/day
RT001, oral, 4.5 g BID for 28 days or matching comparator
干预措施: Low dose cohort
RT001, oral, 9 g/day
RT001, oral, 4.5 g BID for 28 days or matching comparator
干预措施: High dose cohort
结局指标
主要结局
Number of Patients With Adverse Events
时间窗: 28 days
次要结局
- Pharmacokinetics - Area Under the Concentration-time Curve After a Single Dose(24 hours)
- Pharmacokinetics - Maximum Observed Plasma Concentration After a Single Dose(24 hours)
- Pharmacokinetics - Time to Reach Maximum Plasma Concentration After a Single Dose(24 hours)
- Pharmacokinetics - Maximum Observed Plasma Concentration After Final Dose on Day 28(Day 28-Day 31 (3 days))
- Pharmacokinetics - Terminal Half-life Estimation After Final Dose on Day 28(Day 28-Day 31 (3 days))
- Change From Baseline at 28 Days in the Timed 25 Foot Walk (T25FW)(28 days)
- Change From Baseline at 28 Days in the Friedreich Ataxia Rating Scale (FARS) - Neurological Score (Minimum Score 0, Maximum Score 125, Lower is Better)(28 days)
- Change From Baseline at 28 Days in Peak Workload for the Treated Population vs. the Comparator Population(28 days)
研究点 (2)
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