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
适应症
干预措施
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 19
- 试验地点
- 2
- 主要终点
- Number of Patients With Adverse Events
研究概览
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
- 18 Years 至 50 Years（Adult）
- 性别
- All
- 接受健康志愿者
- 否
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
- •Diabetes mellitus (Type 1 or 2)
- •Suicidal ideation as determined by the Columbia-Suicide Severity Rating Scale
- •History, within the last 2 years, of alcohol abuse, significant mental illness, or physical opioid dependence
- •Cannot adhere to the dietary guidance required to be followed by the protocol
- •Cannot take the medication due to impairment in swallowing capsules
研究组 & 干预措施
RT001, oral, 1.8 g/day
Experimental
RT001, oral, 1.8 g QD for 28 days or matching comparator
干预措施: Low dose cohort (Drug)
RT001, oral, 1.8 g/day
Experimental
RT001, oral, 1.8 g QD for 28 days or matching comparator
干预措施: High dose cohort (Drug)
RT001, oral, 9 g/day
Experimental
RT001, oral, 4.5 g BID for 28 days or matching comparator
干预措施: Low dose cohort (Drug)
RT001, oral, 9 g/day
Experimental
RT001, oral, 4.5 g BID for 28 days or matching comparator
干预措施: High dose cohort (Drug)
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)
研究者
研究点 (2)
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