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Study is being terminated due to a business decision.
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This is a gene transfer therapy study evaluating the safety of and delandistrogene moxeparvovec dystrophin protein expression from delandistrogene moxeparvovec following therapeutic plasma exchange (plasmapheresis) in ambulatory male participants with DMD and pre-existing antibodies to AAVrh74 over a period of 59 weeks.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Delandistrogene Moxeparvovec After Plasmapheresis Procedure | Experimental | Participants will receive a single intravenous (IV) infusion of delandistrogene moxeparvovec on Day 1 after plasmapheresis procedure if AAVrh74 antibodies are sufficiently low. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| delandistrogene moxeparvovec | Genetic | Single IV infusion of delandistrogene moxeparvovec |
|
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in Quantity of Delandistrogene Moxeparvovec Dystrophin Expression Adjusted by Muscle Content Biopsied Muscle as Measured by Western Blot | Baseline, Week 12 | |
| Change From Baseline in Quantity of Delandistrogene Moxeparvovec Dystrophin Expression in Biopsied Muscle as Measured by Immunofluorescence (IF) Fiber Intensity | Baseline, Week 12 | |
| Change From Baseline in Quantity of Delandistrogene Moxeparvovec Dystrophin Expression in Biopsied Muscle as Measured by IF Percent Dystrophin-positive Fibers (PDPF) | Baseline, Week 12 | |
| Mean Concentration of Vector Genome Copies Using Polymerase Chain Reaction in Muscle Tissue Biopsy, After Delandistrogene Moxeparvovec Administration | Week 12 |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants with a Treatment Emergent Adverse Event (TEAE), Adverse Event of Special Interest (AESI), and Serious Adverse Event (SAE) | Baseline up to End of Study (Up to Week 59) | |
| Change from Baseline in rAAVrh74 Antibody Titers | Baseline, Week 1 |
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Inclusion Criteria:
Exclusion Criteria:
Note: Other inclusion or exclusion criteria could apply.
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| Name | Affiliation | Role |
|---|---|---|
| Medical Director | Sarepta Therapeutics, Inc. | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Florida, College of Medicine | Gainesville | Florida | 32610 | United States | ||
| Washington University School of Medicine in St. Louis |
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| Plasmapheresis | Procedure | Therapeutic plasma exchange procedure |
|
| St Louis |
| Missouri |
| 63110 |
| United States |
| Nationwide Children's Hospital | Columbus | Ohio | 43205 | United States |
| ID | Term |
|---|---|
| D020388 | Muscular Dystrophy, Duchenne |
| ID | Term |
|---|---|
| D009136 | Muscular Dystrophies |
| D020966 | Muscular Disorders, Atrophic |
| D009135 | Muscular Diseases |
| D009140 | Musculoskeletal Diseases |
| D009468 | Neuromuscular Diseases |
| D009422 | Nervous System Diseases |
| D040181 | Genetic Diseases, X-Linked |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
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| ID | Term |
|---|---|
| D010956 | Plasmapheresis |
| ID | Term |
|---|---|
| D001781 | Blood Component Removal |
| D013812 | Therapeutics |
| D016060 | Sorption Detoxification |
| D005112 | Extracorporeal Circulation |
| D013514 | Surgical Procedures, Operative |
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