ENDEAVOR: A Gene Transfer Therapy Study to Evaluate the Safety of and Expression From SRP-9001 (Delandistrogene Moxeparvovec) in Participants With Duchenne Muscular Dystrophy (DMD)
Study Details
Study Description
Brief Summary
This is an open-label gene transfer therapy study evaluating the safety of and expression from SRP-9001 (delandistrogene moxeparvovec) in participants with DMD over 260 weeks.
Condition or Disease | Intervention/Treatment | Phase |
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Phase 1 |
Study Design
Arms and Interventions
Arm | Intervention/Treatment |
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Experimental: SRP-9001 Participants will receive a single intravenous (IV) infusion of SRP-9001 on Day 1. |
Genetic: SRP-9001
Single IV infusion of SRP-9001
Other Names:
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Outcome Measures
Primary Outcome Measures
- Part 1: Change From Baseline in Quantity of Micro-Dystrophin Protein Expression at Week 12, as Measured by Western Blot [Baseline, Week 12]
Secondary Outcome Measures
- Vector Shedding, Measured in Urine, Saliva, and Stool Samples Post-Infusion [Day 1 up to Week 52]
- Level of Antibody Titers to Recombinant Adeno-Associated Virus Serotype rh74 (rAAVrh74) [Day 2 up to Week 260]
- Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs) and Adverse Events (AEs) of Special Interest [Baseline up to Week 260]
- Change From Baseline in Quantity of Micro-Dystrophin Protein Expression at Week 12, as Measured by Immunofluorescence (IF) Fiber Intensity [Baseline, Week 12]
- Change From Baseline in Quantity of Micro-Dystrophin Expression at Week 12, as Measured by IF Percent Dystrophin Positive Fibers (PDPF) [Baseline, Week 12]
Eligibility Criteria
Criteria
Inclusion Criteria:
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Cohort 1 only: Is ambulatory, and ≥4 to <8 years of age at the time of Screening.
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Cohort 2 only: Is ambulatory, and ≥8 to <18 years of age at the time of Screening.
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Cohort 3 only: Non-ambulatory per protocol specified criteria.
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Cohort 4 only: Is ambulatory, and ≥3 to <4 years of age at the time of Screening.
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Definitive diagnosis of DMD based on documented clinical findings and prior genetic testing.
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Ability to cooperate with motor assessment testing.
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Cohorts 1, 2, and 3 only: Stable dose equivalent of oral corticosteroids for at least 12 weeks before screening and the dose is expected to remain constant (except for modifications to accommodate changes in weight) throughout the first year of the study.
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Cohort 4: Do not yet require use of chronic steroids for treatment of their DMD, in the opinion of the Investigator, and are not receiving steroids at the time of Screening.
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rAAVrh74 antibody titers are not elevated as per protocol-specified requirements.
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Genetic mutation inclusion criteria varies by cohort.
Exclusion Criteria:
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Has a concomitant illness, autoimmune disease, chronic drug treatment, and/or cognitive delay/impairment that in the opinion of the Investigator creates unnecessary risks for gene transfer.
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Exposure to gene therapy, investigational medication, or any treatment designed to increase dystrophin expression within protocol specified time limits.
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Abnormality in protocol-specified diagnostic evaluations or laboratory tests.
Other inclusion/exclusion criteria apply.
Contacts and Locations
Locations
Site | City | State | Country | Postal Code | |
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1 | Stanford University | Palo Alto | California | United States | 94304 |
2 | University of California, Davis | Sacramento | California | United States | 95616 |
3 | Washington University in St. Louis | Saint Louis | Missouri | United States | 21205 |
4 | Nationwide Children's Hospital | Columbus | Ohio | United States | 43205 |
5 | Children's Hospital of The King's Daughters | Norfolk | Virginia | United States | 23507 |
Sponsors and Collaborators
- Sarepta Therapeutics, Inc.
- Hoffmann-La Roche
Investigators
- Study Director: Medical Director, Sarepta Therapeutics, Inc.
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- SRP-9001-103