2023-505043-39-00招募中3 期
A Phase 3, Multinational, Long-Term Follow-Up Study to Evaluate Safety and Efficacy in Subjects Who Have Previously Received SRP-9001 in a Clinical Study
适应症
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 75
- 试验地点
- 7
- 主要终点
- Number of Participants with a Treatment-emergent Adverse Event (TEAE), Serious Adverse Event (SAE), and Adverse Event of Special Interest (AESI)
研究概览
简要总结
To evaluate the long-term safety of delandistrogene moxeparvovec
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
- •Received delandistrogene moxeparvovec for Duchenne muscular dystrophy in a previous clinical study
- •Has (a) parent(s) or legal caregiver(s) or is ≥18 years of age and able to understand and comply with the study visit schedule and all other protocol requirements
排除标准
- 未提供
结局指标
主要结局
Number of Participants with a Treatment-emergent Adverse Event (TEAE), Serious Adverse Event (SAE), and Adverse Event of Special Interest (AESI)
Number of Participants with a Treatment-emergent Adverse Event (TEAE), Serious Adverse Event (SAE), and Adverse Event of Special Interest (AESI)
次要结局
- Change in the North Star Ambulatory Assessment (NSAA) Total Score From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec
- Change in Time to Rise From Floor From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec
- Change in Time of 10-meter Walk/Run (10MWR) from Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec
- Change in Performance of Upper Limb (Version 2.0) Total Scores from Pre-infusion Baseline to 5 Years Post-Infusion of Delandistrogene Moxeparvovec
- Change in PUL (Version 2.0) Domain Specific Scores from Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec
- Change in Forced Vital Capacity Percent (FVC%) Predicted from Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec)
- Change in Peak Expiratory Flow Percent (PEF%) Predicted from Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec
- Change in Cardiac Magnetic Resonance Imaging (MRI) findings from Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec
- Change in Musculoskeletal MRI Findings from Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec
研究者
Sarepta Therapeutics Inc.
Scientific
Sarepta Therapeutics Inc.
研究点 (7)
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