A Phase 1 interventional study of delandistrogene moxeparvovec in Duchenne Muscular Dystrophy, sponsored by Sarepta Therapeutics, Inc.. Recruiting at 12 sites in United States. Open to male participants aged 2 Years and older. Per ClinicalTrials.gov, last updated 2026-09-11.
Sponsored by Sarepta Therapeutics, Inc. · Phase 1, Interventional, and Treatment
Cohort 8 (non-ambulatory participants) is currently enrolling new participants. Enrollment for Cohorts 1 through 7 has been completed.
This is an open-label gene transfer therapy study evaluating the safety of and expression from delandistrogene moxeparvovec in participants with Duchenne Muscular Dystrophy (DMD). The maximum participant duration for this study is 156 weeks.
481 studies on the registry are indexed under Muscular Dystrophy, Duchenne; 108 are open to participants now.
This study's planned enrollment of 83 is above the median of 26 across 332 interventional 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.
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.
All Cohorts:
Exclusion Criteria:
Note: Other inclusion/exclusion criteria 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.
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.
A condition in which the immune system attacks the body’s own tissues, such as rheumatoid arthritis, lupus, psoriasis, type 1 diabetes or inflammatory bowel disease.
Immunotherapy studies often leave out active autoimmune disease, and some name exceptions, such as thyroid disease on replacement hormone. Check the exceptions this study lists.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
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.
Hypersensitivity
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Excipients
The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Autoimmune disease
A condition in which the immune system attacks the body’s own tissues, such as rheumatoid arthritis, lupus, psoriasis, type 1 diabetes or inflammatory bowel disease.
Immunotherapy studies often leave out active autoimmune disease, and some name exceptions, such as thyroid disease on replacement hormone. Check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Participants will receive a single intravenous (IV) infusion of delandistrogene moxeparvovec on Day 1.
Genetic: delandistrogene moxeparvovec
Single IV infusion of delandistrogene moxeparvovec
Also known as: SRP-9001, delandistrogene moxeparvovec-rokl, ELEVIDYS
Part 1 (Cohorts 1 to 5): Change from Baseline in Quantity of Delandistrogene Moxeparvovec Dystrophin Expression at Week 12, as Measured by Western Blot
Time frame: Baseline, Week 12
Part 1 (Cohorts 6 to 8): Quantity of Delandistrogene Moxeparvovec Dystrophin Expression at Week 12 as Measured by Western Blot
Time frame: Week 12
Cohort 8: Number of Participants with Acute Liver Injury (ALI)
Time frame: Baseline up to Week 72
Vector Shedding, Measured in Urine, Saliva, and Stool Samples Post-Infusion
Time frame: Day 1 up to Week 104
Level of Antibody Titers to Recombinant Adeno-Associated Virus Serotype rh74 (rAAVrh74)
Time frame: Day 2 up to Week 156
Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs) and Adverse Events of Special Interest (AESIs)
Time frame: Baseline up to Week 156
Cohort 8: Number of Participants With Infections, Edema, Wound-healing Complications, Hyperlipidemia, Angioedema, and Intestinal Lung Disease/ Non-infectious Pneumonitis
Time frame: Baseline up to Week 72
Change from Baseline in Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression at Week 12, as Measured by Immunofluorescence (IF) Fiber Intensity
Time frame: Baseline, Week 12
Change from Baseline in Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression at Week 12, as Measured by IF Percent Dystrophin Positive Fibers (PDPF)
Time frame: Baseline, Week 12
Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression at Week 12 as Measured by IF Fiber Intensity
Time frame: Week 12
Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression at Week 12 as Measured by IF PDPF
Time frame: Week 12
Cohort 8: Number of Participants With Hepatic AEs, Hepatic Biomarkers, and Laboratory Assessments Indicative of Either Acute Hepatocellular Injury or Acute Liver Dysfunction
Time frame: Baseline up to Week 72
Cohort 8: Number of Participants with Severe ALI
Time frame: Baseline up to Week 72
Cohort 8: Duration of Steroids Administered
Time frame: Baseline up to Week 72
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Muscular Dystrophy, Duchenne →