An Observational Study Comparing Delandistrogene Moxeparvovec (ELEVIDYS) With Standard of Care in Participants With Duchenne Muscular Dystrophy
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Delandistrogene Moxeparvovec, Standard of Care.
- Who it may be relevant to
- Registry conditions: Duchenne Muscular Dystrophy. Basic parameters: from 4 years · Male.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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Official title
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
Overview
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.
Interventions
- Genetic Delandistrogene Moxeparvovec
No study medication will be provided by the sponsor during this study. - Drug Standard of Care
No study medication will be provided by the sponsor during this study.
Primary outcome measures
- Mean Change From Baseline in Time to Walk/Run 10 Meters (10MWR) (Calculated Velocity) at Month 12 [Time frame: Baseline, Month 12]
- Number of Participants Experiencing Acute Liver Injury (ALI) [Time frame: Baseline through Month 12]
Secondary outcome measures (11)
- Time to Rise From Floor (Supine to Stand) [Time frame: Up to 10 years]
- Loss of Ambulation (LOA) [Time frame: Up to 10 years]
- Performance of Upper Limb (PUL) Version 2.0 Entry Item A Score or Brooke Upper Extremity Scale Score [Time frame: Up to 10 years]
- Patient-reported Outcomes Measurement Information (PROMIS) Domain Scores of Mobility, Upper Extremity, and Fatigue [Time frame: Up to 10 years]
- Pulmonary Function as Measured by Forced Vital Capacity (FVC) [Time frame: Up to 10 years]
- Pulmonary Function as Measured by FVC Percent Predicted (FVC%p) [Time frame: Up to 10 years]
- Cardiac Function, Including Left Ventricular Ejection Fraction (LVEF), as Measured by Echocardiogram (ECHO) or Cardiac MRI (cMRI) [Time frame: Up to 10 years]
- Number of Participants Experiencing Serious ALI and Acute Liver Failure (ALF) [Time frame: Baseline through Month 12]
- Number of Participants Experiencing Complications Associated with ALI and ALF [Time frame: Up to 10 years]
- Survival Time [Time frame: Up to 10 years]
- Time to First Vertebral Body (Spine) Fracture [Time frame: Up to 10 years]
Eligibility criteria
Inclusion criteria
- 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
Exclusion criteria
- 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.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Cohort
Study locations
United States · 25 centers
- Arkansas Children's Hospital — Little Rock
- Children's Hospital Los Angeles - PIN — Los Angeles
- University of Colorado - PPDS — Aurora
- Connecticut Children's Medical Center - Hartford — Hartford
- Children's National Medical Center — Washington D.C.
- Nicklaus Children's Hospital — Miami
- Nemours Children's Hospital - Orlando — Orlando
- All Children's Research Institute, Inc — St. Petersburg
- … and 17 more centers
Identifiers
NCT: NCT06270719 · SRP-9001-401