A Gene Transfer Therapy Study to Evaluate the Safety of and Expression From Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)
Phase 1
Active, not recruiting
- Conditions
- Muscular Dystrophy, Duchenne
- Interventions
- Genetic: delandistrogene moxeparvovec
- Registration Number
- NCT04626674
- Lead Sponsor
- Sarepta Therapeutics, Inc.
- Brief Summary
This is an open-label gene transfer therapy study evaluating the safety of and expression from delandistrogene moxeparvovec in participants with DMD. The maximum participant duration for this study is 156 weeks.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- Male
- Target Recruitment
- 55
Inclusion Criteria
- For Cohorts 1-7: Has a definitive diagnosis of DMD based on documented clinical findings and prior genetic testing.
- Cohort 1: Is ambulatory, and ≥4 to <8 years of age at the time of Screening.
- Cohort 2: Is ambulatory, and ≥8 to <18 years of age at the time of Screening.
- Cohort 3: Non-ambulatory per protocol specified criteria at the time of Screening.
- Cohort 4: Is ambulatory and ≥3 to <4 years of age at the time of Screening.
- Cohort 5a: Is ambulatory and ≥4 to <9 years of age.
- Cohort 5b: Non-ambulatory per protocol specified criteria at the time of Screening.
- Cohort 6: Is ambulatory, and ≥2 to <3 years of age at the time of Screening.
- Cohort 7: Non-ambulatory per protocol-specified criteria at the time of Screening.
- Ability to cooperate with motor assessment testing.
- Cohorts 1, 2, 3, 5, and 7 only: Stable dose equivalent of oral glucocorticoids 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.
- Cohorts 4 and 6: 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.
- rAAVrh74 antibody titers are not elevated as per protocol-specified requirements.
- Genetic mutation inclusion criteria vary by cohort.
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Exclusion Criteria
- 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.
- Exposure to gene therapy, investigational medication, or any treatment designed to increase dystrophin expression within protocol-specified time limits.
- Abnormality in protocol-specified diagnostic evaluations or laboratory tests.
Other inclusion/exclusion criteria apply.
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Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Delandistrogene Moxeparvovec delandistrogene moxeparvovec Participants will receive a single intravenous (IV) infusion of delandistrogene moxeparvovec on Day 1.
- Primary Outcome Measures
Name Time Method Part 1: Change from Baseline in Quantity of Delandistrogene Moxeparvovec Dystrophin Expression at Week 12, as Measured by Western Blot Baseline, Week 12 Part 1: Quantity of Delandistrogene Moxeparvovec Dystrophin Expression at Week 12 as Measured by Western Blot Week 12
- Secondary Outcome Measures
Name Time Method Level of Antibody Titers to Recombinant Adeno-Associated Virus Serotype rh74 (rAAVrh74) Day 2 up to Week 156 Change from Baseline in Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression at Week 12, as Measured by Immunofluorescence (IF) Fiber Intensity 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) Baseline, Week 12 Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression at Week 12 as Measured by IF Fiber Intensity: Week 12 Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression at Week 12 as Measured by IF PDPF Week 12 Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs) and Adverse Events (AEs) of Special Interest Baseline up to Week 156 Vector Shedding, Measured in Urine, Saliva, and Stool Samples Post-Infusion Day 1 up to Week 104
Trial Locations
- Locations (5)
Stanford University
🇺🇸Palo Alto, California, United States
Washington University in St. Louis
🇺🇸Saint Louis, Missouri, United States
Nationwide Children's Hospital
🇺🇸Columbus, Ohio, United States
Children's Hospital of The King's Daughters
🇺🇸Norfolk, Virginia, United States
University of California, Davis
🇺🇸Sacramento, California, United States
Related News
FDA Grants Traditional Approval to Elevidys Gene Therapy for Ambulatory DMD, Accelerated Approval for Nonambulatory Patients
FDA granted traditional approval for Sarepta Therapeutics’ SRP-9001 (Elevidys) for ambulatory Duchenne muscular dystrophy (DMD) patients aged 4+, expanding its use. Accelerated approval for nonambulatory patients was also announced, pending confirmatory trial results. SRP-9001, a gene therapy, showed functional improvements in trials, despite not meeting the primary endpoint in the phase 3 EMBARK study.
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