NCT06874010招募中1 期
A Phase 1/2, Open-Label, Multiple Ascending Dose Study to Assess the Safety, Tolerability, and Pharmacokinetics of Subcutaneous and Intravenous DT-216P2 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
- 招募中
- 入组人数
- 20
- 试验地点
- 3
- 主要终点
- Frequency of treatment-emergent adverse events (TEAE)
研究概览
简要总结
The purpose of this study is to evaluate the safety and tolerability, and pharmacokinetics of DT-216P2 administered multiple ascending doses in patients with FA.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 65 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participants must be 18-65 years of age inclusive, at the time of signing the informed consent.
- •Genetically confirmed diagnosis of FA, with homozygous GAA repeat expansions in the frataxin gene.
- •Stage 5.5 or less on the FSA at screening.
- •BMI between 16 and 32 kg/m2 at screening; weight should be <= 100 kg at screening.
- •Male and/or female using protocol defined and regulatory approved contraception.
- •Capable of giving signed informed consent.
排除标准
- •Any concomitant medical condition that in the opinion of the investigator, puts the participant at risk or precludes participant from completing the study protocol.
- •Any clinically significant nonmedical conditions and psychiatric disorders that could put the participant at higher risk for participation in the study, influence the participant's ability to participate in the study, or interfere with interpretation of the participant's study results, in the opinion of the investigator.
- •Received an investigational agent within the last 30 days or 5 half-lives, whichever longer, prior to the first dose of study drug, or are in follow-up of another clinical study prior to study enrollment. Exception: Potential participants who are currently on Omaveloxolone must be on stable doses for at least 3 months.
- •Is not willing to comply with the contraceptive requirements during the study period, as per protocol.
研究组 & 干预措施
DT-216P2
Experimental
干预措施: DT-216P2 (Drug)
结局指标
主要结局
Frequency of treatment-emergent adverse events (TEAE)
时间窗: Up to Week 12
次要结局
- Area under the curve (AUClast, AUCinf)(Up to Week 12)
- Maximum concentration (Cmax)(Up to Week 12)
- Time to Cmax (Tmax)(Up to Week 12)
- Half-life (t1/2)(Up to Week 12)
- Frataxin expression at baseline and after treatment will be measured(Up to Week 12)
研究者
研究点 (3)
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