NCT06667453招募中2 期
A Phase 2 Randomized, Double-Blind, Placebo-Controlled, Multiple Ascending Dose Study of PGN-EDODM1 in Adult Participants With Myotonic Dystrophy Type 1 (FREEDOM2-DM1)
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 发起方
- PepGen Inc
- 入组人数
- 24
- 试验地点
- 19
- 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 effects of an investigational medicine, PGN-EDODM1, to see how safe and tolerable multiple administrations of PGN-EDODM1 are for people with myotonic dystrophy type 1 (DM1) compared to placebo.
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
- 16 Years 至 65 Years（Child, Adult, Older 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
- •Presence of myotonia
- •Have sufficient muscle mass in bilateral tibialis anterior (TA) muscles that a needle biopsy can safely be performed
- •Body Mass Index (BMI) of < 35.0 kg/m^2
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 considered clinically significant by the Investigator
- •Medications specific for the treatment of myotonia within 2 weeks prior to screening
- •Percent predicted forced vital capacity (FVC) <40%
- •Use of an investigational drug, device, or product within 30 days of 5 half-lives of the study drug (whichever is longer) prior to Screening
- •Note: Other inclusion and exclusion criteria may apply.
研究组 & 干预措施
Placebo
Placebo Comparator
Participants randomized to the placebo arm will receive doses of saline (0.9% NaCl), once every 4 weeks (Q4W) for 12 weeks
干预措施: Placebo (Other)
PGN-EDODM1
Experimental
Participants will be randomized to receive ascending doses of PGN-EDODM1, once every 4 weeks (Q4W) for 12 weeks
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 Day 112
次要结局
- Maximum Observed Plasma Drug Concentration (Cmax) of PGN-EDODM1(Day 1 through Day 84)
- Time to Maximum Observed Plasma Drug Concentration (Tmax) of PGN-EDODM1(Day 1 through Day 84)
- Apparent Terminal Half-Life (t½) of PGN-EDODM1(Day 1 through Day 84)
- Area Under the Concentration-time Curve of PGN-EDODM1(Day 1 through Day 84)
- Change in splicing index in skeletal muscle tissue(Baseline through Day 91)
- Change in myotonia as measured by video Hand Opening Time (vHOT)(Baseline through Day 112)
- Hand grip strength(Baseline through Day 112)
- Change in mobility as measured by 10 meter walk/run time(Baseline through Day 112)
研究者
研究点 (19)
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