NCT07220603招募中2 期
An Open-Label Extension Study Evaluating Safety and Pharmacokinetics in Participants With Myotonic Dystrophy Type 1 (FREEDOM-OLE)
干预措施
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 发起方
- PepGen Inc
- 入组人数
- 48
- 试验地点
- 3
- 主要终点
- Safety and tolerability as assessed by number of participants with Adverse Events (AEs)
研究概览
简要总结
The purpose of this study is to learn about the long-term safety and tolerability of PGN-EDODM1 in participants with myotonic dystrophy type 1 (DM1) who have completed a prior study with PGN-EDODM1.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participant has completed a prior study with PGN-EDODM1
排除标准
- •Abnormal laboratory tests at screening considered clinically significant by the Investigator
- •Use of an investigational drug (other than PGN-EDODM1), device, or product, within 30 days or 5 half-lives of the study drug (whichever is longer) prior to study entry
研究组 & 干预措施
PGN-EDODM1
Experimental
Participants will receive doses of PGN-EDODM1 once every 4 weeks (Q4W)
干预措施: PGN-EDODM1 (Drug)
结局指标
主要结局
Safety and tolerability as assessed by number of participants with Adverse Events (AEs)
时间窗: Baseline through Week 108
次要结局
未报告次要终点
研究者
研究点 (3)
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