A Phase 2, Open-Label, Multiple Ascending Dose Study of PGN-EDO51 With a Long-Term Extension in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment (CONNECT1-EDO51)
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 发起方
- PepGen Inc
- 入组人数
- 7
- 试验地点
- 5
- 主要终点
- Adverse events and serious adverse events (safety and tolerability of PGN-EDO51 in MAD period)
研究概览
简要总结
The study consists of 3 periods: A Screening Period (up to 45 days), a Multiple Ascending Dose (MAD) Period (16 weeks), and a Long-Term Extension (LTE) Period (108 weeks).
The primary purpose of the MAD period is to evaluate the safety and tolerability of multiple ascending intravenous (IV) doses of PGN-EDO51 administered to participants with Duchenne Muscular Dystrophy (DMD). The primary purpose of the LTE period is to evaluate the long-term safety and tolerability of PGN-EDO51 in participants who have completed the MAD period.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- None
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 able to be corrected by skipping Exon 51
- •Body weight at least 18kg at Screening
- •Performance of Upper Limb (PUL) 2.0 entry score of at least 4 at Screening (assessing upper limb function in ambulant and non-ambulant individuals with DMD)
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 every 4 weeks
干预措施: PGN-EDO51 (Drug)
PGN-EDO51 at Dose Level 2 every 4 weeks
干预措施: PGN-EDO51 (Drug)
结局指标
主要结局
Adverse events and serious adverse events (safety and tolerability of PGN-EDO51 in MAD period)
时间窗: Baseline to Week 16
Adverse events and serious adverse events
Adverse events and serious adverse events (long-term safety and tolerability of PGN-EDO51 in LTE period)
时间窗: Baseline to Week 108
Adverse events and serious adverse events
次要结局
- Plasma pharmacokinetic (PK) parameters (MAD period)(Baseline to Week 12)
- PK Plasma levels (LTE period)(Baseline to Week 104)
- Skeletal muscle concentration of PGN-EDO51 (MAD period)(Baseline to Week 16)
- Dystrophin Levels (MAD period)(Baseline to Week 16)