NCT06204809已完成1 期
A Phase 1 Placebo-Controlled Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Single-Ascending Doses of PGN-EDODM1 in Adult Participants With Myotonic Dystrophy Type 1 (FREEDOM-DM1)
适应症
干预措施
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 发起方
- PepGen Inc
- 入组人数
- 24
- 试验地点
- 12
- 主要终点
- Number of participants with Adverse Events, Serious Adverse Events, with abnormal Clinical Laboratory tests, abnormal ECGs, and abnormal Vital Signs
研究概览
简要总结
The primary purpose of the study is to evaluate the safety and tolerability of single intravenous (IV) doses of PGN-EDODM1 administered to participants with Myotonic Dystrophy Type 1 (DM1).
The study consists of 2 periods: A Screening Period (up to 30 days) and a Treatment and Observation Period (16 weeks).
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 60 Years（Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Confirmed diagnosis of DM1, as defined as having a repeat sequence in the DMPK gene with at least 100 CTG repeats
- •Medical Research Council (MRC) score of ≥ Grade 4- in bilateral tibialis anterior (TA) muscles (the ability to move through full range of motion and hold against at least moderate pressure from the examiner)
- •Presence of myotonia
排除标准
- •Congenital DM1
- •Known history or presence of any clinically significant conditions that may interfere with study safety assessments
- •Abnormal laboratory tests at screening
- •Medications specific for the treatment of myotonia within 2 weeks prior to screening
- •Percent predicted forced vital capacity (FVC) <40%
- •Note: Other inclusion and exclusion criteria may apply.
研究组 & 干预措施
PGN-EDODM1
Experimental
PGN-EDODM1 for infusion
干预措施: PGN-EDODM1 for infusion (Drug)
Placebo
Placebo Comparator
0.9% NaCl
干预措施: Placebo (Other)
结局指标
主要结局
Number of participants with Adverse Events, Serious Adverse Events, with abnormal Clinical Laboratory tests, abnormal ECGs, and abnormal Vital Signs
时间窗: Baseline to Week 16
次要结局
- Area Under the Concentration-time Curve of PGN-EDODM1(Baseline up to Day 3)
- Apparent Terminal Half-Life (t½) of PGN-EDODM1(Baseline up to Day 3)
- Maximum Observed Plasma Drug Concentration (Cmax) of PGN-EDODM1(Baseline up to Day 3)
- Time to Maximum Observed Plasma Drug Concentration (Tmax) of PGN-EDODM1(Baseline up to Day 3)
研究者
研究点 (12)
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