NCT05285540已完成1 期
A Phase 1a, Randomized, Double-blind, Placebo-controlled, Single Ascending Dose Study of DT-216 in Adult Patients With Friedreich 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
- 39
- 试验地点
- 1
- 主要终点
- Frequency of treatment adverse events (TEAEs)
研究概览
简要总结
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetic and pharmacodynamic effects of intravenous DT-216 in adult patients with Friedreich Ataxia. This single ascending dose study is randomized, double-blind, placebo-controlled.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Sequential
- 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 55 Years（Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Genetically confirmed diagnosis of Friedreich Ataxia with homozygous GAA repeat expansions
- •Body mass index (BMI) between 17 and 32 kg/m2
- •Stage 5.5 or less on the Functional Staging for Ataxia (FSA)
排除标准
- •Has any concomitant medical condition that in the opinion of the investigator, puts the participant at risk or precludes participant from completing the study
- •Has clinically significant abnormal laboratory results
- •Has significant cardiac disease
- •Received an investigational drug within 3 months of screening
- •Note: Other protocol defined Inclusion/Exclusion criteria may apply.
研究组 & 干预措施
Single Dose: DT-216
Experimental
DT-216 will be administered once
干预措施: DT-216 (Drug)
Single Dose: DT-216 matching placebo
Experimental
Placebo will be administered once
干预措施: DT-216 matching Placebo (Drug)
结局指标
主要结局
Frequency of treatment adverse events (TEAEs)
时间窗: Up to approximately 30 days
Frequency of treatment emergent adverse events (TEAEs)
次要结局
- Maximum Plasma Concentration (Cmax) of DT-216(Up to approximately 30 days)
- Time to Maximum Plasma Concentration (Tmax) of DT-216(Up to approximately 30 days)
- Area Under the Concentration-time Curve (AUC) of DT-216(Up to approximately 30 days)
研究者
研究点 (1)
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