Systemic Gene Delivery Phase I/IIa Clinical Trial for Duchenne Muscular Dystrophy Using rAAVrh74.MHCK7.Micro-dystrophin (microDys-IV-001)
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 4
- 试验地点
- 2
- 主要终点
- Number of Participants With Adverse Events (AEs)
研究概览
简要总结
This study was an open-label single-dose gene transfer therapy study evaluating the safety of delandistrogene moxeparvovec intravenous (IV) administration in boys with DMD. This study was originally designed to consist of 12 patients across 2 Cohorts. Cohort A would have included participants ages 3 months to 3 years, and Cohort B included participants ages 4 to 7 years old. No participants were enrolled in Cohort A.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 3 Months 至 7 Years（Child）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Cohort A participants: 3 months to 3 years of age, inclusive
- •Cohort B participants: 4 to 7 years of age, inclusive
- •Definitive diagnosis of DMD based on documented clinical findings and prior genetic testing.
- •Ability to cooperate with motor assessment testing.
- •Cohort A participants: No previous treatment with corticosteroids.
- •Cohort B participants: Stable dose equivalent of oral corticosteroids for at least 12 weeks prior to screening and the dose is expected to remain constant (except for potential modifications to accommodate changes in weight) throughout the first year of the study.
- •Cohorts A & B: A frameshift mutation contained between exons 18 and 58 (inclusive).
排除标准
- •Exposure to gene therapy, investigational medication, or any treatment designed to increase dystrophin expression within protocol specified time limits.
- •Abnormality in protocol-specified diagnostic evaluations or laboratory tests.
- •Presence of any other clinically significant illness, medical condition, or requirement for chronic drug treatment that in the opinion of the Investigator creates unnecessary risk for gene transfer.
- •Other inclusion or exclusion criteria could apply.
结局指标
主要结局
Number of Participants With Adverse Events (AEs)
时间窗: Up to 5 years
An AE is any untoward medical occurrence in a clinical study participant that does not necessarily have a causal relationship with the study drug. An AE can, therefore, be any unfavorable and unintended symptom, sign, disease, condition, or test abnormality that occurs during or after administration of a study drug, whether or not considered related to the study drug. A summary of serious and all other non-serious adverse events regardless of causality is located in the Reported Adverse Events module.
次要结局
- Change From Baseline at Year 5 in the 100 Meter Timed Test(Baseline, Year 5)
- Change From Baseline at Day 90 in Delandistrogene Moxeparvovec Dystrophin Expression as Measured by IF Percent Dystrophin Positive Fibers (PDPF)(Baseline, Day 90)
- Change From Baseline at Day 90 in Delandistrogene Moxeparvovec Dystrophin Expression as Measured by Immunofluorescence (IF) Fiber Intensity(Baseline, Day 90)
- Change From Baseline at Day 90 in Delandistrogene Moxeparvovec Dystrophin Expression as Measured by Western Blot(Baseline, Day 90)