2024-512626-28-00暂停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 (ENVISION)
适应症
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 暂停
- 入组人数
- 95
- 试验地点
- 15
- 主要终点
- Part 1: Change From Baseline in the Total Score of Performance of Upper Limb (PUL) (Version 2.0) at Week 72
研究概览
简要总结
Part 1: Change From Baseline in the Total Score of Performance of Upper Limb (PUL) (Version 2.0) at Week 72
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 0 years 至 64 years（18-64 Years, 0-17 Years）
- 性别
- Male
- 接受健康志愿者
- 是
入选标准
- •Definitive diagnosis of DMD based on documented clinical findings and prior genetic testing. A pathogenic frameshift mutation or premature stop codon in the DMD gene, except for any deletion mutations in exon 8 and/or
- •Cohort 1 only: Non-ambulatory per protocol specified criteria.
- •Cohort 2 only: Ambulatory per protocol specified criteria and ≥8 to <18 years of age at the time of Screening.
- •Ability to cooperate with motor assessment testing.
- •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).
- •Recombinant Adeno-Associated Virus Serotype rh74 (rAAVrh74) antibody titers are not elevated as per protocol-specified requirements.
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
Part 1: Change From Baseline in the Total Score of Performance of Upper Limb (PUL) (Version 2.0) at Week 72
Part 1: Change From Baseline in the Total Score of Performance of Upper Limb (PUL) (Version 2.0) at Week 72
次要结局
- Part 1: Change From Baseline in Percent Predicted Forced Vital Capacity (FVC) at Week 72
- Part 1: Change From Baseline in Percent Predicted Peak Expiratory Flow (PEF) at Week 72
- Part 1: Quantity of Delandistrogene Moxeparvovec Dystrophin Expression at Week 12 as Measured by Western Blot
- Part 1: Change From Baseline in Patient-Reported Outcomes Measurement Information (PROMIS) Score in Upper Extremity Function to Week 72
- Number of Participants with a Treatment Emergent Adverse Event (TEAE), Adverse Event of Special Interest (AESI), and Serious Adverse Event (SAE)
- Part 1 (For Cohort 2 Only): Change From Baseline in the North Star Ambulatory Assessment (NSAA) Total Score at Week 72
- Part 1: Change From Baseline in Global Circumferential Strain as Measured by Cardiac MRI at Week 72
- Part 1: Change From Baseline in PUL (Version 2.0) Middle Domain Score at Week 72.
研究者
Sarepta Therapeutics Inc.
Scientific
Sarepta Therapeutics Inc.
研究点 (15)
Loading locations...
相似试验
招募中
1 期
Study to Evaluate the Safety and Efficacy of SRP-9001 in Subjects With Duchenne Muscular DystrophyMedDRA version: 20.0Level: PTClassification code 10013801Term: Duchenne muscular dystrophySystem Organ Class: 10010331 - Congenital, familial and genetic disordersMedDRA version: 20.1Level: PTClassification code 10052655Term: Duchenne muscular dystrophy gene carrierSystem Organ Class: 10010331 - Congenital, familial and genetic disordersDuchenne Muscular DystrophyEUCTR2020-002372-13-ITSAREPTA THERAPEUTICS, INC.116
进行中（未招募）
1 期
A Phase 3, Multinational, Randomized, Double-Blind, Placebo-Controlled Study to Assess the Efficacy and Safety of EN3285 for the Prevention or Delay to Onset of Severe Oral Mucositis in Subjects with Head and Neck Cancer Receiving Chemoradiotherapy - N/ASevere Oral MucositisMedDRA version: 9.1Level: LLTClassification code 10028130Term: Mucositis oralEUCTR2007-006549-42-GBEndo Pharmaceuticals Inc.180
招募中
1 期
Study to evaluate the Safety and Efficacy of SRP-9001 in non ambulatory and ambulatory Subjects with Duchenne Muscular DystrophyDuchenne Muscular DystrophyEUCTR2020-002372-13-SESarepta Therapeutics, Inc.148
尚未招募
3 期
A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of SRP-9001 (Delandistrogene Moxeparvovec) in Non-Ambulatory and Ambulatory Participants With Duchenne Muscular Dystrophy (DMD)Duchenne Muscular DystrophyJPRN-jRCT2033230274Patrick O Malley4
已完成
3 期
A Multinational, Phase 3, Randomized, Double-Blind, Placebo-Controlled, Efficacy and Safety Study of Enzalutamide in Patients With Nonmetastatic Castration-Resistant Prostate Cancer10038597Prostate cancerNL-OMON50210Medivation Inc., a wholly owned subsidiary of Pfizer Inc.42