A Long-term Observational Study Evaluating Sarepta Therapeutics, Inc.'s Exon-Skipping Therapies in Patients With Duchenne Muscular Dystrophy Under Conditions of Routine Clinical Practice
试验速览
- 阶段
- 不适用
- 状态
- Enrolling By Invitation
- 入组人数
- 300
- 试验地点
- 20
- 主要终点
- Loss of Ambulation (LOA)
研究概览
简要总结
This is a phase 4, multicenter, prospective, observational study designed to collect both medical history data and prospective data on Duchenne Muscular Dystrophy (DMD) treatment outcomes in participants receiving eteplirsen, golodirsen, and casimersen in routine clinical practice. Participants in this study will have been prescribed eteplirsen, golodirsen, or casimersen commercially prior to entry into the study.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Is willing to provide informed assent or consent (if applicable) and has a parent(s) or legal guardian(s) or is a participant ≥18 years of age who is (are) willing to provide informed consent for the participant to participate in the study and comply with study data collection procedures.
- •Has an established clinical diagnosis of DMD, as documented prior to screening by a genetic report.
- •Receiving, or initiating treatment with, eteplirsen, golodirsen, or casimersen at the time of observational study enrollment. Note: Participants with a prescription for eteplirsen, golodirsen, or casimersen at enrollment must initiate the exon-skipping therapy within 6 months of the date of enrollment or will no longer be eligible for this study. Note: Enrollment of eteplirsen participants has been completed, no additional participants will be enrolled.
排除标准
- •Is currently participating in any DMD interventional study at the time of this study enrollment.
- •Has declined to provide consent for collection of their genetic data.
- •Has a medical condition or confounding circumstances that, in the opinion of the Investigator, might compromise:
- •The participant's ability to comply with the protocol-required procedures
- •The participant's wellbeing or safety, and/or
- •The clinical interpretability of the data collected from the participant.
- •Other inclusion/exclusion criteria may apply.
研究组 & 干预措施
Eteplirsen
干预措施: Eteplirsen (Drug)
Golodirsen
干预措施: Golodirsen (Drug)
Casimersen
干预措施: Casimersen (Drug)
结局指标
主要结局
Loss of Ambulation (LOA)
时间窗: Up to 5 years
Time to Rise From the Floor (Supine to Stand)
时间窗: Up to 5 years
Time to Walk/Run 10 Meters
时间窗: Up to 5 years
Performance of Upper Limb Module for DMD 2.0 (PUL 2.0) Entry Item A
时间窗: Up to 5 years
Pulmonary Function, as Measured by Forced Vital Capacity (FVC) (% Predicted)
时间窗: Up to 5 years
Cardiac Function, Including Left Ventricular Ejection Fraction (LVEF) as Measured by Echocardiogram (ECHO)
时间窗: Up to 5 years
次要结局
未报告次要终点