A Gene Delivery Study to Evaluate the Safety of and Expression From SRP-9001 in Duchenne Muscular Dystrophy (DMD) (ENDEAVOR)
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ClinicalTrials.gov Identifier: NCT04626674 |
Recruitment Status :
Recruiting
First Posted : November 12, 2020
Last Update Posted : December 24, 2020
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Condition or disease | Intervention/treatment | Phase |
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Muscular Dystrophy, Duchenne | Genetic: SRP-9001 | Phase 1 |
Study Type : | Interventional (Clinical Trial) |
Estimated Enrollment : | 10 participants |
Allocation: | N/A |
Intervention Model: | Single Group Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
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, 2021 |
Estimated Study Completion Date : | December 2025 |

Arm | Intervention/treatment |
---|---|
Experimental: SRP-9001
Patients will receive a single intravenous (IV) infusion of SRP-9001 on Day 1.
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Genetic: SRP-9001
Single IV infusion of SRP-9001. |
- Part 1: Change From Baseline in Quantity of Micro-dystrophin Protein Expression Measured by Western Blot [ Time Frame: Baseline up to Week 12 ]
- Vector Shedding as Measured by Polymerase Chain Reaction in Urine, Saliva and Stool Samples Post-infusion [ Time Frame: Baseline up to Week 260 ]
- Immunogenicity of SRP-9001 Determined by Level of Antibody Titers to rAAVrh74 [ Time Frame: Screening up to Week 260 ]
- Incidence of Treatment Emergent Adverse Events (TEAEs), Serious AEs and Adverse Events of Special Interest [ Time Frame: Baseline up to Week 260 ]
- Number of Participants with Clinically Significant Abnormalities in Vital Signs, Physical Examinations, Laboratory Parameters, Electrocardiograms (ECG) and Echocardiograms (ECHO) Findings [ Time Frame: Baseline up to Week 260 ]

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Ages Eligible for Study: | 4 Years to 7 Years (Child) |
Sexes Eligible for Study: | Male |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Is male at birth, ambulatory and from 4 to under 8 years of age at time of randomization.
- Definitive diagnosis of Duchenne Muscular Dystrophy based on documented clinical findings and prior genetic testing
- Ability to cooperate with motor assessment testing.
- Stable dose equivalent of oral corticosteroids for at least 12 weeks.
- rAAVrh74 antibody titers are not elevated as per protocol-specified requirements.
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.

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
Contact: Medical Information | +1-800-690-2003 | clinicaltrials@sarepta.com |
United States, California | |
University of California, Davis | Recruiting |
Sacramento, California, United States, 95616 | |
Contact 916-734-3993 omsarwary@ucdavis.edu | |
Principal Investigator: Craig McDonald, MD | |
United States, Missouri | |
Washington University in St. Louis | Recruiting |
Saint Louis, Missouri, United States, 21205 | |
Contact: Alyssa Sonsoucie 314-362-1566 sonsouciea@wustl.edu | |
Principal Investigator: Craig Zaidman, MD | |
United States, Ohio | |
Nationwide Children's Hospital | Not yet recruiting |
Columbus, Ohio, United States, 43205 | |
Contact: Jerry Mendell | |
Principal Investigator: Jerry Mendell | |
United States, Virginia | |
Children's Hospital of The King's Daughters | Recruiting |
Norfolk, Virginia, United States, 23507 | |
Contact: Cara Headrick 757-668-6417 Cara.Headrick@CHKD.ORG | |
Principal Investigator: Crystal Proud, MD |
Study Director: | Medical Director | Sarepta Therapeutics, Inc. |
Responsible Party: | Sarepta Therapeutics, Inc. |
ClinicalTrials.gov Identifier: | NCT04626674 |
Other Study ID Numbers: |
SRP-9001-103 |
First Posted: | November 12, 2020 Key Record Dates |
Last Update Posted: | December 24, 2020 |
Last Verified: | December 2020 |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
Duchenne Muscular Dystrophy Gene-Delivery DMD |
Ambulatory Pediatric Micro-dystrophin |
Muscular Dystrophies Muscular Dystrophy, Duchenne Muscular Disorders, Atrophic Muscular Diseases Musculoskeletal Diseases |
Neuromuscular Diseases Nervous System Diseases Genetic Diseases, Inborn Genetic Diseases, X-Linked |