A Long-term Multicenter Prospective Observational Study Evaluating the Comparative Effectiveness and Safety of Sarepta Gene Transfer Therapy vs. Standard of Care in Participants 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
- 500
- 试验地点
- 50
- 主要终点
- Mean Change From Baseline in Time to Walk/Run 10 Meters (10MWR) (Calculated Velocity) at Month 12
研究概览
简要总结
This is a multicenter, prospective, observational Phase 4 study including a post marketing safety requirement, designed to collect both medical history data and prospective data on Duchenne muscular dystrophy (DMD) treatment outcomes in participants receiving delandistrogene moxeparvovec (ELEVIDYS) as part of clinical care, compared to participants with DMD receiving or prescribed to start chronic glucocorticoid treatment at the time of study enrollment in routine clinical practice.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 4 Years 至 —（Child, Adult, Older Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Has an established clinical diagnosis of DMD based on documentation of clinical findings and prior confirmatory genetic testing using a clinical diagnostic genetic test.
- •Is currently receiving or has been prescribed to start chronic glucocorticoid therapy at the time of this observational study enrollment.
- •For ELEVIDYS-treated Participants (Cohorts 1a, 1b, and 1c):
- •Is at least 4 years of age at the time of infusion
- •Will either: a) be initiating or has initiated ELEVIDYS within the last 30 days in routine clinical practice at the time of this observational study enrollment, or b) was administered ELEVIDYS in routine clinical practice and has the required minimum dataset for entry into the observational study per Sponsor approval
- •For Standard of Care Comparators (Cohort 2):
- •Is at least 4 years of age at the time of enrollment
- •Is unexposed to DMD gene therapy at the time of this observational study enrollment
排除标准
- •Has any deletion of exon 8 and/or exon 9 in the DMD gene.
- •Is currently participating in any DMD interventional study at the time of this observational study enrollment.
- •Has any prior exposure to DMD gene therapy other than that described for Cohort 1c (ELEVIDYS Retrospectively Treated Cohort).
- •Has a medical condition or confounding circumstances (for example, prior traumatic limitation for mobility or significant behavioral comorbidity) 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
Cohort 2 (Standard of Care)
Cohort 2: ambulatory DMD participants who are at least 4 years of age at baseline, unexposed to DMD gene therapy, and receiving or prescribed chronic glucocorticoids at study entry.
干预措施: Standard of Care (Drug)
Cohort 1 (Treated)
Cohort 1: participants prescribed ELEVIDYS in a commercial setting. Cohort 1a (Ambulatory ELEVIDYS Prospectively Treated Cohort): participants prescribed ELEVIDYS by commercially treating physicians with consent no later than 30 days from infusion. All participants will be dosed with ELEVIDYS based on United States prescribing information (USPI). The Post Market Requirement (PMR) Cohort (sub-cohort of 1a) consists of participants with laboratory data as specified in the ELEVIDYS USPI.
Cohort 1b (Non-ambulatory ELEVIDYS Prospectively Treated Cohort): non-ambulatory DMD participants prescribed ELEVIDYS commercially and recruited by treating physicians before infusion (enrollment currently closed).
Cohort 1c (ELEVIDYS Retrospectively Treated Cohort): participants dosed with ELEVIDYS with complete baseline data within 6 months prior to dosing and complete prospectively collected annual follow-up data after infusion until the time of cohort entry (sponsor approval required for enrollment).
干预措施: Delandistrogene Moxeparvovec (Genetic)
结局指标
主要结局
Mean Change From Baseline in Time to Walk/Run 10 Meters (10MWR) (Calculated Velocity) at Month 12
时间窗: Baseline, Month 12
Number of Participants Experiencing Acute Liver Injury (ALI)
时间窗: Baseline through Month 12
次要结局
- Time to Rise From Floor (Supine to Stand)(Up to 10 years)
- Loss of Ambulation (LOA)(Up to 10 years)
- Performance of Upper Limb (PUL) Version 2.0 Entry Item A Score(Up to 10 years)
- Patient-reported Outcomes Measurement Information (PROMIS) Domain Scores of Mobility, Upper Extremity and Fatigue(Up to 10 years)
- Pulmonary Function, as Measured by Forced Vital Capacity (FVC) (% Predicted)(Up to 10 years)
- Cardiac Function, Including Left Ventricular Ejection Fraction (LVEF) as Measured by Echocardiogram (ECHO)(Up to 10 years)
- Number of Participants Experiencing Treatment-emergent Adverse Events (TEAEs)(Up to 10 years)
- Time to Walk/Run 10 Meters (Calculated Velocity)(Up to 10 years)
- Performance of Upper Limb (PUL) Version 2.0 Entry Item A Score or Brooke Upper Extremity Scale Score(Up to 10 years)
- Patient-reported Outcomes Measurement Information (PROMIS) Domain Scores of Mobility, Upper Extremity, and Fatigue(Up to 10 years)
- Pulmonary Function as Measured by Forced Vital Capacity (FVC)(Up to 10 years)
- Pulmonary Function as Measured by FVC Percent Predicted (FVC%p)(Up to 10 years)
- Cardiac Function, Including Left Ventricular Ejection Fraction (LVEF), as Measured by Echocardiogram (ECHO) or Cardiac MRI (cMRI)(Up to 10 years)
- Number of Participants Experiencing Serious ALI and Acute Liver Failure (ALF)(Baseline through Month 12)
- Number of Participants Experiencing Complications Associated with ALI and ALF(Up to 10 years)
- Survival Time(Up to 10 years)
- Time to First Vertebral Body (Spine) Fracture(Up to 10 years)