An observational study in Duchenne Muscular Dystrophy, sponsored by Sarepta Therapeutics, Inc.. Enrolling by invitation at 29 sites in United States. Open to male participants aged 4 Years and older. Per ClinicalTrials.gov, last updated 2026-10-23.
Sponsored by Sarepta Therapeutics, Inc. · Observational
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.
481 studies on the registry are indexed under Muscular Dystrophy, Duchenne; 108 are open to participants now.
This study's planned enrollment of 500 is above the median of 60 across 132 observational studies indexed under Muscular Dystrophy, Duchenne.
Browse Muscular Dystrophy, Duchenne studies →Sarepta Therapeutics, Inc. is the lead sponsor of 52 studies on the registry; 8 are open to participants now.
Of its 21 completed or terminated interventional studies of FDA-regulated products, 12 (57%) have results posted.
Counted across the registry records on this site, refreshed daily.
DMD participants within each prescriber's practice who either (1) have been prescribed delandistrogene moxeparvovec or (2) are receiving or have been prescribed to begin chronic glucocorticoid therapy in routine clinical practice, and who meet the study eligibility criteria, will be invited to enroll into the study and will be followed according to the protocol.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
For ELEVIDYS-treated Participants (Cohorts 1a, 1b, and 1c):
For Standard of Care Comparators (Cohort 2):
Exclusion Criteria:
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:
Other inclusion/exclusion criteria may apply.
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Corticosteroids (steroids)
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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).
Genetic: Delandistrogene Moxeparvovec
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.
Drug: Standard of Care
No study medication will be provided by the sponsor during this study.
Also known as: SRP-9001, delandistrogene moxeparvovec-rokl, ELEVIDYS
No study medication will be provided by the sponsor during this study.
Also known as: Chronic glucocorticoid treatment
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
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
Started Feb 2024 (actual). Primary completion is estimated for Dec 2028, 58 months after the start.
Of 63 observational studies under Muscular Dystrophy, Duchenne, half reached primary completion within 40 months of starting (the median), counting those still running; a quarter within 12 and three quarters within 120. This study's planned 58 months is above that.
Primary completion moved once since we started tracking on Sep 25, 2026: Dec 2029 → Dec 2028, on Oct 23, 2026.
Earlier changes are on the record history on ClinicalTrials.gov ↗
How these figures are worked out, and which studies are left out of them.
From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗
Eligibility is decided by the study team. Share this record with your doctor or contact the team directly.
No contact was published for this record. The registry link below has the sponsor’s details.
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Muscular Dystrophy, Duchenne →