A Gene Transfer Therapy Study to Evaluate the Safety of and Expression From SRP-9001 (Delandistrogene Moxeparvovec) in Participants With Duchenne Muscular Dystrophy (DMD) (ENDEAVOR)
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT04626674|
Recruitment Status : Enrolling by invitation
First Posted : November 12, 2020
Last Update Posted : September 30, 2022
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|Condition or disease||Intervention/treatment||Phase|
|Muscular Dystrophy, Duchenne||Genetic: SRP-9001||Phase 1|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||46 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||An Open-Label, Systemic Gene Delivery Study Using Commercial Process Material to Evaluate the Safety of and Expression From SRP-9001 in Subjects With Duchenne Muscular Dystrophy (ENDEAVOR)|
|Actual Study Start Date :||November 23, 2020|
|Estimated Primary Completion Date :||May 31, 2023|
|Estimated Study Completion Date :||January 31, 2028|
Participants will receive a single intravenous (IV) infusion of SRP-9001 on Day 1.
Single IV infusion of SRP-9001
Other Name: delandistrogene moxeparvovec
- Part 1: Change From Baseline in Quantity of Micro-Dystrophin Protein Expression at Week 12, as Measured by Western Blot [ Time Frame: Baseline, Week 12 ]
- Vector Shedding, Measured in Urine, Saliva, and Stool Samples Post-Infusion [ Time Frame: Day 1 up to Week 52 ]
- Level of Antibody Titers to Recombinant Adeno-Associated Virus Serotype rh74 (rAAVrh74) [ Time Frame: 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 [ Time Frame: 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 [ Time Frame: Baseline, Week 12 ]
- Change From Baseline in Quantity of Micro-Dystrophin Expression at Week 12, as Measured by IF Percent Dystrophin Positive Fibers (PDPF) [ Time Frame: Baseline, Week 12 ]
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|Ages Eligible for Study:||3 Years and older (Child, Adult, Older Adult)|
|Sexes Eligible for Study:||Male|
|Accepts Healthy Volunteers:||No|
- Cohort 1 only: Is ambulatory, and ≥4 to <8 years of age at the time of Screening.
- Cohort 2 only: Is ambulatory, and ≥8 to <18 years of age at the time of Screening.
- Cohort 3 and Cohort 5b: Non-ambulatory per protocol specified criteria at the time of Screening.
- Cohort 4 only: Is ambulatory and ≥3 to <4 years of age at the time of Screening.
- Cohort 5a only: Is ambulatory and ≥4 to <9 years of age at the time of Screening
- For Cohort 1-5: Has a definitive diagnosis of DMD based on documented clinical findings and prior genetic testing.
- Ability to cooperate with motor assessment testing.
- Cohorts 1, 2, 3, and 5 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.
- 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.
- rAAVrh74 antibody titers are not elevated as per protocol-specified requirements.
- Genetic mutation inclusion criteria vary by cohort.
- 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.
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): NCT04626674
|United States, California|
|Palo Alto, California, United States, 94304|
|University of California, Davis|
|Sacramento, California, United States, 95616|
|United States, Missouri|
|Washington University in St. Louis|
|Saint Louis, Missouri, United States, 21205|
|United States, Ohio|
|Nationwide Children's Hospital|
|Columbus, Ohio, United States, 43205|
|United States, Virginia|
|Children's Hospital of The King's Daughters|
|Norfolk, Virginia, United States, 23507|
|Study Director:||Medical Director||Sarepta Therapeutics, Inc.|
|Responsible Party:||Sarepta Therapeutics, Inc.|
|Other Study ID Numbers:||
|First Posted:||November 12, 2020 Key Record Dates|
|Last Update Posted:||September 30, 2022|
|Last Verified:||September 2022|
|Studies a U.S. FDA-regulated Drug Product:||Yes|
|Studies a U.S. FDA-regulated Device Product:||No|
Duchenne Muscular Dystrophy
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked