A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD) (EMBARK)
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| ClinicalTrials.gov Identifier: NCT05096221 |
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Recruitment Status :
Active, not recruiting
First Posted : October 27, 2021
Last Update Posted : November 7, 2023
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Sponsor:
Sarepta Therapeutics, Inc.
Collaborator:
Hoffmann-La Roche
Information provided by (Responsible Party):
Sarepta Therapeutics, Inc.
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Brief Summary:
The study will evaluate the safety and efficacy of gene transfer therapy in boys with DMD. It is a randomized, double-blind, placebo-controlled study. The participants who are randomized to the placebo arm will have an opportunity for treatment with gene transfer therapy at the beginning of the second year.
| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Duchenne Muscular Dystrophy | Genetic: delandistrogene moxeparvovec Genetic: placebo | Phase 3 |
| Study Type : | Interventional (Clinical Trial) |
| Actual Enrollment : | 126 participants |
| Allocation: | Randomized |
| Intervention Model: | Parallel Assignment |
| Masking: | Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor) |
| Primary Purpose: | Treatment |
| Official Title: | A Phase 3 Multinational, Randomized, Double-Blind, Placebo-Controlled Systemic Gene Delivery Study to Evaluate the Safety and Efficacy of SRP-9001 in Subjects With Duchenne Muscular Dystrophy (EMBARK) |
| Actual Study Start Date : | October 27, 2021 |
| Actual Primary Completion Date : | October 4, 2023 |
| Estimated Study Completion Date : | November 30, 2024 |
Resource links provided by the National Library of Medicine
| Arm | Intervention/treatment |
|---|---|
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Experimental: Delandistrogene Moxeparvovec followed by Placebo
Participants will receive single intravenous (IV) infusion of delandistrogene moxeparvovec on Day 1. Then, participants will receive a single IV infusion of matching placebo at Year 2.
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Genetic: delandistrogene moxeparvovec
Single IV infusion of delandistrogene moxeparvovec.
Other Names:
Genetic: placebo Single IV infusion of matching placebo. |
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Placebo Comparator: Placebo followed by Delandistrogene Moxeparvovec
Participants will receive matching placebo IV infusion on Day 1. Then, participants will have the opportunity to receive a single IV infusion of delandistrogene moxeparvovec at Year 2.
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Genetic: delandistrogene moxeparvovec
Single IV infusion of delandistrogene moxeparvovec.
Other Names:
Genetic: placebo Single IV infusion of matching placebo. |
Primary Outcome Measures :
- Part 1: Change From Baseline in NSAA Total Score at Week 52 [ Time Frame: Baseline, Week 52 ]
Secondary Outcome Measures :
- Part 1: Quantity of Delandistrogene Moxeparvovec Dystrophin Expression at Week 12 as Measured by Western Blot, in a Subset of Participants [ Time Frame: Week 12 ]
- Part 1: Change From Baseline in Time to Rise From the Floor, Time to Complete 100 and 10 Meter Walk/Run, and the Timed Stair Ascend 4 Steps Test at Week 52 [ Time Frame: Baseline, Week 52 ]
- Part 1: Change From Baseline in Stride Velocity 95th Centile (SV95C) Measured by a Wearable Device [ Time Frame: Baseline, Week 52 ]
- Part 1: Change from Baseline in Patient-Reported Outcomes Measurement Information System (PROMIS) Score in Mobility and Upper Extremity Function to Week 52 [ Time Frame: Baseline, Week 52 ]PROMIS is a family of instruments developed and validated to assess health-related quality of life.
- Part 1: Number of Skills Gained or Improved at Week 52 as Measured by the NSAA [ Time Frame: Baseline, Week 52 ]
- Number of Participants with a Treatment Emergent Adverse Event (TEAE), Serious Adverse Event (SAE), and Adverse Event of Special Interest (AESI) [ Time Frame: Baseline up to Week 104 ]
Information from the National Library of Medicine
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| Ages Eligible for Study: | 4 Years to 7 Years (Child) |
| Sexes Eligible for Study: | Male |
| Accepts Healthy Volunteers: | No |
Criteria
Inclusion Criteria:
- Is ambulatory and from 4 to under 8 years of age at time of randomization.
- Definitive diagnosis of DMD based on documented clinical findings and prior genetic testing.
- Ability to cooperate with motor assessment testing.
- Stable daily dose of oral corticosteroids for at least 12 weeks prior to Screening, and the dose is expected to remain constant throughout the study (except for modifications to accommodate changes in weight).
- rAAVrh74 antibody titers are not elevated as per protocol-specified requirements.
- A pathogenic frameshift mutation or premature stop codon contained between exons 18 and 79 (inclusive), with the exception of mutation fully contained within exon 45.
Exclusion Criteria:
- 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.
- Presence of any other clinically significant illness, medical condition, or requirement for chronic drug treatment that in the opinion of the Investigator creates unnecessary risk for gene transfer.
Other inclusion or exclusion criteria could apply.
Information from the National Library of Medicine
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05096221
Locations
Show 42 study locations
Sponsors and Collaborators
Sarepta Therapeutics, Inc.
Hoffmann-La Roche
Investigators
| Study Director: | Medical Director | Sarepta Therapeutics, Inc. |
| Responsible Party: | Sarepta Therapeutics, Inc. |
| ClinicalTrials.gov Identifier: | NCT05096221 |
| Other Study ID Numbers: |
SRP-9001-301 2019-003374-91 ( EudraCT Number ) |
| First Posted: | October 27, 2021 Key Record Dates |
| Last Update Posted: | November 7, 2023 |
| Last Verified: | November 2023 |
| Studies a U.S. FDA-regulated Drug Product: | Yes |
| Studies a U.S. FDA-regulated Device Product: | No |
Keywords provided by Sarepta Therapeutics, Inc.:
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Muscular Dystrophies Duchenne Muscular Dystrophy DMD North Star Ambulatory Assessment (NSAA) |
Ambulatory Pediatric Gene-Delivery |
Additional relevant MeSH terms:
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Muscular Dystrophies Muscular Dystrophy, Duchenne Muscular Disorders, Atrophic Muscular Diseases Musculoskeletal Diseases |
Neuromuscular Diseases Nervous System Diseases Genetic Diseases, Inborn Genetic Diseases, X-Linked |