A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Multiple Ascending Dose Study of PGN-EDO51 With a Long-Term Extension in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment (CONNECT2-EDO51)
试验速览
- 阶段
- 2 期
- 状态
- 撤回
- 发起方
- PepGen Inc
- 入组人数
- 16
- 主要终点
- Adverse events and serious adverse events (safety and tolerability of PGN-EDO51 during the MAD period)
研究概览
简要总结
The study consists of 3 periods: A Screening Period (up to 45 days), a double-blind, placebo-controlled Multiple Ascending Dose (MAD) Period (28 weeks), and a Long-Term Extension (LTE) Period (108 weeks).
The primary purpose of the MAD period is to evaluate the safety and tolerability and levels of dystrophin after multiple ascending intravenous (IV) doses of PGN-EDO51 administered to participants with Duchenne muscular dystrophy (DMD). During the MAD period, participants will be randomized to either receive PGN-EDO51 or placebo in a 3:1 fashion, meaning that participants have a 75% chance of receiving PGN-EDO51 and a 25% chance of receiving placebo during this period. The primary purpose of the open-label LTE period is to evaluate the long-term safety and tolerability of PGN-EDO51 in participants who have completed the MAD period. All participants who roll-over into the LTE will receive PGN-EDO51 (no placebo in the LTE).
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
- 6 Years 至 16 Years（Child）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Confirmed diagnosis of DMD with a genetic alteration that can be treated by skipping exon 51
- •Body weight at least 25kg (55lbs)
- •Performance of Upper Limb (PUL) 2.0 entry score of at least 4
排除标准
- •Known history or presence of any clinically significant conditions that may interfere with study safety assessments
- •Treatment with any gene replacement therapy for the treatment of DMD at any time
- •Current or recent systemic infection within 2 weeks prior to Screening or infection requiring IV antibiotics within 4 weeks prior to Screening
- •Recent surgery requiring anesthesia within 3 months prior to Screening or expected surgery requiring general anesthesia during the study
研究组 & 干预措施
PGN-EDO51 at Dose Level 1 or Placebo every 4 weeks
干预措施: IV infusion (Drug)
PGN-EDO51 at Dose Level 1 or Placebo every 4 weeks
干预措施: Placebo (Other)
PGN-EDO51 at Dose Level 2 or Placebo every 4 weeks
干预措施: IV infusion (Drug)
PGN-EDO51 at Dose Level 2 or Placebo every 4 weeks
干预措施: Placebo (Other)
结局指标
主要结局
Adverse events and serious adverse events (safety and tolerability of PGN-EDO51 during the MAD period)
时间窗: Signing of informed consent to Week 28
Dystrophin levels (MAD period)
时间窗: Baseline to Week 28
Adverse events and serious adverse events (safety and tolerability of PGN-EDO51 during the LTE period)
时间窗: Signing of informed consent to Week 108
次要结局
- Plasma pharmacokinetic (PK) parameters (MAD period)(Baseline to Week 28)
- Skeletal muscle concentration of PGN-EDO51 (MAD period)(Baseline to Week 28)
- Plasma pharmacokinetic (PK) parameters (LTE period)(Baseline to Week 104)