Duchenne Muscular Dystrophy
Conditions
Keywords
Duchenne Muscular Dystrophy, DMD, Observational, Standard of Care, Phase 4, ELEVIDYS
Brief summary
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
GENETICDelandistrogene Moxeparvovec
No study medication will be provided by the sponsor during this study.
DRUGStandard of Care
No study medication will be provided by the sponsor during this study.
Sponsors
Sarepta Therapeutics, Inc.
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
MALE
Age
4 Years to No maximum
Healthy volunteers
No
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/
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Mean Change From Baseline in Time to Walk/Run 10 Meters (10MWR) (Calculated Velocity) at Month 12 | Baseline, Month 12 |
| Number of Participants Experiencing Acute Liver Injury (ALI) | Baseline through Month 12 |
Secondary
| Measure | Time frame |
|---|---|
| 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 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 |
Countries
United States
Outcome results
None listed