A Phase 2, Two-Part, Multiple-Ascending-Dose Study of SRP-5051 for Dose Determination, Then Dose Expansion, in Patients With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 入组人数
- 62
- 试验地点
- 25
- 主要终点
- Part B: Change From Baseline in Dystrophin Protein Level at Week 28
研究概览
简要总结
This study will be comprised of 2 parts: 1) Part A (Multiple Ascending Dose [MAD]) will be conducted to evaluate the safety and tolerability of vesleteplirsen at MAD levels to determine the maximum tolerated dose (MTD), and 2) Part B will be conducted to further evaluate the vesleteplirsen doses selected in Part A. Participants enrolling in Part B will be those who completed Part A or Study 5051-102 (NCT03675126) and meet applicable eligibility criteria for Part B, as well as additional participants who meet applicable eligibility criteria for enrollment at the beginning of Part B.
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
- None
入排标准
- 年龄范围
- 7 Years 至 21 Years（Child, Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- 未提供
研究组 & 干预措施
Part A: Vesleteplirsen
Participants received escalating dose levels of vesleteplirsen, every 4 weeks, via intravenous (IV) infusion for up to 75 weeks during Part A. Once the doses have been selected for Part B, all participants who have completed Part A will transition to Part B.
干预措施: Vesleteplirsen (Drug)
Part B: Vesleteplirsen
Participants received vesleteplirsen at the doses selected based on data from Part A every 4 weeks, via IV infusion, for up to 5 years. This included the participants who rolled over from Part A, as well as the additional participants who enrolled at the beginning of Part B.
干预措施: Vesleteplirsen (Drug)
结局指标
主要结局
Part B: Change From Baseline in Dystrophin Protein Level at Week 28
时间窗: Part B: Baseline, Week 28
Part A: Incidence of Adverse Events (AEs)
时间窗: Part A: Baseline up to 75 weeks
次要结局
- Part B: Change from Baseline in Percent Dystrophin-Positive Fibers (PDPF) and Mean Intensity, as Measured by Immunofluorescence Assay at Week 28(Part B: Baseline, Week 28)
- Part A: Pharmacokinetics (PK): Plasma Concentration of Vesleteplirsen(Pre-dose and at multiple time points (up to 32 hours) after end of infusion)
- Part A: PK: Urine Concentration of Vesleteplirsen(Pre-dose and at multiple time periods (up to 48 hours) after end of infusion)
- Part B: Change From Baseline in Exon-Skipping Levels at Week 28(Part B: Baseline, Week 28)
- Part B: Incidence of Adverse Events (AEs)(Part B: Baseline up to Week 304)
- Part B: PK: Plasma Concentration of Vesleteplirsen(Part B predose and at multiple timepoints (up to 48 hours) after end of infusion)
- Part B: PK: Urine Concentration of Vesleteplirsen(Part B predose and at multiple timepoints (up to 48 hours) after end of infusion)