A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Non-Ambulatory and Ambulatory Participants With Duchenne Muscular Dystrophy (DMD) (ENVISION)
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| ClinicalTrials.gov Identifier: NCT05881408 |
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Recruitment Status :
Recruiting
First Posted : May 31, 2023
Last Update Posted : November 24, 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 delandistrogene moxeparvovec gene transfer therapy in non-ambulatory and ambulatory males with DMD. This is a randomized, double-blind, placebo-controlled 2-part study. Participants will be in the study for approximately 128 weeks. All participants will have the opportunity to receive intravenous (IV) delandistrogene moxeparvovec in either Part 1 or Part 2.
| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Duchenne Muscular Dystrophy | Genetic: delandistrogene moxeparvovec Genetic: placebo | Phase 3 |
| Study Type : | Interventional (Clinical Trial) |
| Estimated Enrollment : | 148 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 Transfer Therapy Study to Evaluate the Safety and Efficacy of SRP- 9001 in Non-Ambulatory and Ambulatory Subjects With Duchenne Muscular Dystrophy (ENVISION) |
| Actual Study Start Date : | May 31, 2023 |
| Estimated Primary Completion Date : | January 31, 2026 |
| Estimated Study Completion Date : | January 31, 2027 |
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 IV infusion of delandistrogene moxeparvovec on Day 1. Then, participants will receive a single IV infusion of matching placebo at approximately 72 weeks.
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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 approximately 72 weeks.
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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 the Total Score of Performance of Upper Limb (PUL) (Version 2.0) at Week 72 [ Time Frame: Baseline, Week 72 ]
Secondary Outcome Measures :
- Part 1: Change From Baseline in Percent Predicted Forced Vital Capacity (FVC) at Week 72 [ Time Frame: Baseline, Week 72 ]
- Part 1: Change From Baseline in Percent Predicted Peak Expiratory Flow (PEF) at Week 72 [ Time Frame: Baseline, Week 72 ]
- Part 1: Quantity of Delandistrogene Moxeparvovec Dystrophin Expression at Week 12 as Measured by Western Blot [ Time Frame: Week 12 ]
- Part 1: Change From Baseline in Patient-Reported Outcomes Measurement Information (PROMIS) Score in Upper Extremity Function to Week 72 [ Time Frame: Baseline, Week 72 ]
- Number of Participants with a Treatment Emergent Adverse Event (TEAE), Adverse Event of Special Interest (AESI), and Serious Adverse Event (SAE) [ Time Frame: Baseline up to Week 124 ]
- Part 1 (For Cohort 2 Only): Change From Baseline in the North Star Ambulatory Assessment (NSAA) Total Score at Week 72 [ Time Frame: Baseline, Week 72 ]
- Part 1: Change From Baseline in Global Circumferential Strain as Measured by Cardiac MRI at Week 72 [ Time Frame: Baseline, Week 72 ]
Information from the National Library of Medicine
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| Ages Eligible for Study: | Child, Adult, Older Adult |
| Sexes Eligible for Study: | Male |
| Accepts Healthy Volunteers: | No |
Criteria
Inclusion Criteria:
- Definitive diagnosis of DMD based on documented clinical findings and prior genetic testing.
- Cohort 1 only: Non-ambulatory per protocol specified criteria.
- Cohort 2 only: Ambulatory per protocol specified criteria and ≥8 to <18 years of age at the time of Screening.
- 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).
- Recombinant Adeno-Associated Virus Serotype rh74 (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).
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): NCT05881408
Contacts
| Contact: Sarepta Therapeutics Inc., For Clinical Trial Information, Select Option 4 | 1-888-SAREPTA (1-888-727-3782) | SareptAlly@sarepta.com |
Locations
| United States, Arkansas | |
| Arkansas Children's Hospital | Active, not recruiting |
| Little Rock, Arkansas, United States, 72202 | |
| United States, California | |
| Lucile Packard Children's Hospital Stanford | Active, not recruiting |
| Palo Alto, California, United States, 94304 | |
| University of California at Davis Medical Center | Recruiting |
| Sacramento, California, United States, 95817 | |
| Contact SareptAlly@sarepta.com | |
| Principal Investigator: Craig McDonald, MD | |
| Rady Children's Hospital-San Diego | Active, not recruiting |
| San Diego, California, United States, 92123 | |
| United States, Florida | |
| University of Florida, UF Health Center for Pediatric Neuromuscular and Rare Diseases | Active, not recruiting |
| Gainesville, Florida, United States, 32608 | |
| United States, Illinois | |
| Ann & Robert H. Lurie Children's Hospital of Chicago | Active, not recruiting |
| Chicago, Illinois, United States, 60611 | |
| United States, Iowa | |
| University of Iowa Hospitals and Clinics, Dept of Pediatrics | Active, not recruiting |
| Iowa City, Iowa, United States, 52242 | |
| United States, Maryland | |
| The Johns Hopkins Hospital, Charlotte R. Bloomberg Children's Center, Pediatric Clinical Research Unit | Active, not recruiting |
| Baltimore, Maryland, United States, 21287 | |
| United States, Massachusetts | |
| Boston Children's Hospital | Active, not recruiting |
| Boston, Massachusetts, United States, 02115 | |
| United States, Missouri | |
| Washington University of St. Louis, St. Louis Children's Hospital | Active, not recruiting |
| Saint Louis, Missouri, United States, 63110 | |
| United States, New York | |
| University of Rochester, Department of Neurology | Active, not recruiting |
| Rochester, New York, United States, 14642 | |
| United States, North Carolina | |
| Lenox Baker Children's Hospital (Duke University) | Active, not recruiting |
| Durham, North Carolina, United States, 27705 | |
| United States, Pennsylvania | |
| Children's Hospital of Philadelphia | Active, not recruiting |
| Philadelphia, Pennsylvania, United States, 19104 | |
| United States, Virginia | |
| Children's Hospital of the King's Daughters | Active, not recruiting |
| Norfolk, Virginia, United States, 23510 | |
Sponsors and Collaborators
Sarepta Therapeutics, Inc.
Hoffmann-La Roche
Investigators
| Study Director: | Medical Director | Sarepta Therapeutics, Inc. |
Additional Information:
| Responsible Party: | Sarepta Therapeutics, Inc. |
| ClinicalTrials.gov Identifier: | NCT05881408 |
| Other Study ID Numbers: |
SRP-9001-303 2020-002372-13 ( EudraCT Number ) |
| First Posted: | May 31, 2023 Key Record Dates |
| Last Update Posted: | November 24, 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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Ambulatory Non-ambulatory DMD Gene-Delivery |
Pediatric North Star Ambulatory Assessment (NSAA) Performance of Upper Limb (PUL) Duchenne |
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 |