NCT05573698已完成1 期
A Phase 1b, Randomized, Double-blind, Placebo-controlled, Multiple Ascending Dose and Multi-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
- 32
- 试验地点
- 1
- 主要终点
- Safety Measure
研究概览
简要总结
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetic, and pharmacodynamic effects of multiple doses of intravenous DT-216 in adult patients with Friedrich Ataxia.
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.
- •Able and willing to sign informed consent form prior to study enrollment.
- •Stage 5.5 or less on the Functional Staging for Ataxia (FSA) at screening.
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.
研究组 & 干预措施
Multiple Dose: DT-216
Experimental
Participants will be administered multiple doses of DT-216
干预措施: DT-216 (Drug)
Multiple Dose: DT-216 matching placebo
Placebo Comparator
Participants will be administered multiple doses of placebo
干预措施: Placebo (Drug)
结局指标
主要结局
Safety Measure
时间窗: Up to approximately 60 days
Frequency of treatment emergent adverse events (TEAEs)
次要结局
- Pharmacokinetic parameters(Up to approximately 60 days)
研究者
研究点 (1)
Loading locations...
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