Duchenne Muscular Dystrophy
Conditions
Keywords
Duchenne Muscular Dystrophy, DMD, Observational, Standard of Care, Phase 4
Brief summary
This is a multicenter, prospective, observational Phase 4 study in the United States. The study is designed to collect both medical history and prospective data on Duchenne muscular dystrophy (DMD) treatment outcomes in participants receiving delandistrogene moxeparvovec as part of clinical care, compared to participants with DMD receiving or prescribed to start chronic glucocorticoid treatment at baseline 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 a definitive diagnosis of DMD prior to Screening based on documentation of clinical findings and confirmatory genetic testing. * Is currently receiving or has been prescribed to start chronic glucocorticoid therapy at the time of this observational study enrollment. A participant recruited to Cohorts 1a or 2: * Is at least 4 years of age at the time of enrollment. * Is ambulatory per protocol specified criteria. A participant recruited to Cohort 1b: \- Is non-ambulatory per protocol-specified criteria. For Delandistrogene Moxeparvovec-treated Participants: \- Will be initiating usual care treatment with delandistrogene moxeparvovec at the time of study enrollment. For Comparators: \- Is unexposed to DMD gene therapy at the time of 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 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 |
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 | Up to 10 years |
| Time to Walk/Run 10 Meters (Calculated Velocity) | 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 |
| Patient-reported Outcomes Measurement Information (PROMIS) Domain Scores of Mobility, Upper Extremity and Fatigue | Up to 10 years |
Countries
United States
Outcome results
None listed