NCT07608432招募中3 期
A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Investigate the Efficacy, Safety, and Tolerability of DYNE-251 Administered Intravenously in Ambulatory Male Participants 4 to 18 Years of Age With Duchenne Muscular Dystrophy Amenable to Exon-51 Skipping
适应症
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 90
- 试验地点
- 1
研究概览
简要总结
The purpose of the study is to assess the efficacy, safety, and tolerability of zeleciment rostudirsen (DYNE-251) administered intravenously (IV) every 4 weeks to ambulatory Duchenne muscular dystrophy (DMD) participants, 4 to 18 years of age, with dystrophin mutations amenable to exon 51 skipping.
详细描述
The study consists of three periods: a Screening period (up to 6 weeks), a Placebo-Controlled Period (72 weeks) and an open-label Long-Term Extension Period (96 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
- 4 Years 至 18 Years（Child, Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Ambulatory male with confirmed diagnosis of DMD and with a mutation in the dystrophin gene characterized by exon deletion amenable to exon 51 skipping .
- •Rise From Floor (RFF) time must be < 10 seconds for both screening assessments .
- •Receiving a stable daily or weekend dosage of glucocorticoids for at least 24 weeks prior to randomization with the expectation of maintaining a stable dose during the Placebo-Controlled Period of the study (unless dose adjustment is required by weight change)
排除标准
- •Receipt of ongoing immunosuppressive therapy (other than glucocorticoids) within 12 weeks prior to randomization
- •Use of any pharmacologic treatment (other than glucocorticoids) that may have an effect on muscle strength or function within 12 weeks prior to randomization
- •Any change in prophylaxis/treatment for congestive heart failure (CHF) within 12 weeks prior to randomization
- •Receipt of eteplirsen within 1 week prior to randomization
- •Receipt of alternative exon-skipping or dystrophin-modifying therapy or zeleciment rostudirsen within 24 weeks prior to randomization
- •Receipt of givinostat within 12 weeks prior to randomization
- •Receipt of gene therapy at any time
- •Note: Other inclusion or exclusion criteria may apply
研究者
研究点 (1)
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