JPRN-jRCT2033230274尚未招募3 期
A Phase 3, Multinational, Randomized, Double-Blind, Placebo-Controlled Systemic Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of SRP-9001 in Non-Ambulatory and Ambulatory Subjects With Duchenne Muscular Dystrophy - ENVISIO
Patrick O Malley0 个研究点目标入组 4 人开始时间: 2023年8月2日最近更新:
适应症
试验速览
- 阶段
- 3 期
- 状态
- 尚未招募
- 发起方
- 入组人数
- 4
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- >= 8age old 至 < 18age old（—）
- 性别
- Male
入选标准
- •1.Definitive diagnosis of DMD based on documented clinical findings and prior genetic testing.
- •2.Cohort 1 only: Non-ambulatory per protocol specified criteria.
- •3.Cohort 2 only: Ambulatory per protocol specified criteria and >=8 to <18 years of age at the time of Screening.
- •4.Ability to cooperate with motor assessment testing.
- •5.Stable daily dose of oral corticosteroids for at least 12 weeks prior to Screening, and the dose is expected to remain constant throughout the study (except for modifications to accommodate changes in weight).
- •6.Recombinant Adeno-Associated Virus Serotype rh74 (rAAVrh74) antibody titers are not elevated as per protocol-specified requirements.
- •7.A pathogenic frameshift mutation or premature stop codon contained between exons 18 and 79 (inclusive).
- •Other inclusion criteria could apply.
排除标准
- •1.Exposure to gene therapy, investigational medication, or any treatment designed to increase dystrophin expression within protocol specified time limits.
- •2.Abnormality in protocol-specified diagnostic evaluations or laboratory tests.
- •3.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 exclusion criteria could apply.
研究者
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