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 (EXPEDITION)
试验速览
- 阶段
- 不适用
- 状态
- 尚未招募
- 入组人数
- 10
- 主要终点
- Number of Participants with a Treatment-emergent Adverse Event (TEAE)
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Observational
入排标准
- 年龄范围
- No limit 至 No limit（—）
- 性别
- Male
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
- •Participant or family does not want to disclose participation with general practitioner/primary care physician and other medical providers.
结局指标
主要结局
Number of Participants with a Treatment-emergent Adverse Event (TEAE)
时间窗: up to 5 years after dosing
Number of participants experiencing any adverse event that emerges during treatment, having been absent pre-treatment, or worsens relative to the pre-treatment state.
Number of Participants with a Serious Adverse Event (SAE)
时间窗: up to 5 years after dosing
Number of participants experiencing any adverse event that results in death, is life-threatening, requires hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, or is a congenital anomaly/birth defect.
Number of Participants with an Adverse Event of Special Interest (AESI)
时间窗: up to 5 years after dosing
Number of participants experiencing any adverse event of scientific and medical concern specific to the drug's safety profile, which may require close monitoring.
次要结局
- Change in the North Star Ambulatory Assessment (NSAA) Total Score(From Pre-infusion Baseline to 5 Years Post-infusion)
- Change in Time to Rise From Floor(From Pre-infusion Baseline to 5 Years Post-infusion)
- Change in the Time of 10-meter Walk/Run (10MWR)(From Pre-infusion Baseline to 5 Years Post-infusion)
- Change in Performance of Upper Limb (PUL) (Version 2.0) Total Scores(From Pre-infusion Baseline to 5 Years Post-infusion)
- Change in PUL (Version 2.0) Domain Specific Scores(From Pre-infusion Baseline to 5 Years Post-infusion)
- Change in Forced Vital Capacity Percent (FVC%) Predicted(From Pre-infusion Baseline to 5 Years Post-infusion)
- Change in Peak Expiratory Flow Percent (PEF%) Predicted(From Pre-infusion Baseline to 5 Years Post-infusion)
- Change in Cardiac Magnetic Resonance Imaging (MRI) Findings(From Pre-infusion Baseline to 5 Years Post-infusion)
- Change in Musculoskeletal MRI Findings(From Pre-infusion Baseline to 5 Years Post-infusion)