NCT05967351Enrolling By Invitation3 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
干预措施
试验速览
- 阶段
- 3 期
- 状态
- Enrolling By Invitation
- 入组人数
- 400
- 试验地点
- 38
- 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
The purpose of this study is to provide a single clinical study with a uniform approach to monitoring long-term safety and efficacy in participants who received delandistrogene moxeparvovec in a previous clinical study. No study drug will be administered as part of this study.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Other
- 盲法
- None
入排标准
- 性别
- Male
- 接受健康志愿者
- 否
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
- •Not applicable
研究组 & 干预措施
Delandistrogene Moxeparvovec
Experimental
Participants received delandistrogene moxeparvovec in a previous clinical study.
干预措施: delandistrogene moxeparvovec (Genetic)
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)
时间窗: Up to 10 years
次要结局
- Change in the North Star Ambulatory Assessment (NSAA) Total Score From Pre-infusion Baseline of Delandistrogene Moxeparvovec to the End of the Study Participation(Baseline, up to 10 years)
- Change in Time to Rise From Floor From Pre-infusion Baseline of Delandistrogene Moxeparvovec to the End of the Study Participation(Baseline, up to 10 years)
- Change in the Time of 10-meter Walk/Run (10MWR) From Pre-infusion Baseline of Delandistrogene Moxeparvovec to the End of the Study Participation(Baseline, up to 10 years)
- Change in Performance of Upper Limb (PUL) (Version 2.0) Total Scores From Pre-infusion Baseline of Delandistrogene Moxeparvovec to the End of the Study Participation(Baseline, up to 10 years)
- Change in PUL (Version 2.0) Domain Specific Scores From Pre-infusion Baseline of Delandistrogene Moxeparvovec to the End of the Study Participation(Baseline, up to 10 years)
- Change in Forced Vital Capacity Percent (FVC%) Predicted From Pre-infusion Baseline of Delandistrogene Moxeparvovec to the End of the Study Participation(Baseline, up to 10 years)
- Change in Peak Expiratory Flow Percent (PEF%) Predicted From Pre-infusion Baseline to the End of the Study Participation of Delandistrogene Moxeparvovec(Baseline, up to 10 years)
- Change in Cardiac Magnetic Resonance Imaging (MRI) Findings From Pre-infusion Baseline of Delandistrogene Moxeparvovec to the End of the Study Participation(Baseline, up to 10 years)
- Change in Musculoskeletal MRI Findings From Pre-infusion Baseline of Delandistrogene Moxeparvovec to the End of the Study Participation(Baseline, up to 10 years)
研究者
研究点 (38)
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