A Gene Transfer Therapy Study to Evaluate the Safety of and Expression From Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD) - Non-Ambulatory Cohort (ENDEAVOR)

June 19, 2026 updated by: Sarepta Therapeutics, Inc.

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)

Cohort 8 (non-ambulatory participants) is currently enrolling new participants. Enrollment for Cohorts 1 through 7 has been completed.

This is an open-label gene transfer therapy study evaluating the safety of and expression from delandistrogene moxeparvovec in participants with Duchenne Muscular Dystrophy (DMD). The maximum participant duration for this study is 156 weeks.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Detailed Description

Enrollment for Cohorts 1 through 7 has been completed. Cohort 8 is currently enrolling new participants.

Study Type

Interventional

Enrollment (Estimated)

83

Phase

  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

  • Name: Sarepta Therapeutics Inc., For Clinical Trial Information, Select Option 4
  • Phone Number: 1-888-SAREPTA (1-888-727-3782)
  • Email: SareptAlly@Sarepta.com

Study Locations

  • United States
    • Arkansas
      • Little Rock, Arkansas, United States, 72202
        • Recruiting
        • Arkansas Children's Hospital
        • Principal Investigator:
          • Aravindhan Veerapandiyan, MD
        • Contact:
    • California
      • Palo Alto, California, United States, 94304
      • Sacramento, California, United States, 95616
        • Recruiting
        • University of California, Davis
        • Principal Investigator:
          • Craig McDonald, MD
    • Missouri
      • St Louis, Missouri, United States, 63110
    • Ohio
      • Columbus, Ohio, United States, 43205
        • Active, not recruiting
        • Nationwide Children's Hospital
    • Texas
      • Flower Mound, Texas, United States, 75028
        • Recruiting
        • Neurology Rare Disease Center
        • Principal Investigator:
          • Diana Castro, MD
        • Contact:
    • Virginia
      • Norfolk, Virginia, United States, 23507
        • Recruiting
        • Children's Hospital of The King's Daughters
        • Principal Investigator:
          • Crystal Proud, MD
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

7 months and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • For Cohorts 1-8: Has a definitive diagnosis of DMD based on documented clinical findings and prior genetic testing.
  • Cohort 8: Non-ambulatory per protocol-specified criteria at the time of Screening, has a performance upper limb (PUL) entry item score ≥3 at the Screening visit and has a total PUL score of ≥20 and ≤40 at the time of Screening.
  • Cohorts 1, 2, 3, 5, 7 and 8 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.
  • 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 with time to rise from the floor ≤7 seconds at the screening visit.
  • 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.
  • 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.
  • Genetic mutation inclusion criteria vary by cohort.

All Cohorts:

  • Ability to cooperate with motor assessment testing.
  • rAAVrh74 antibody titers are not elevated as per protocol-specified requirements.

Exclusion Criteria:

  • Cohort 8: Any confounding factors that would prevent the use of oral sirolimus including a known hypersensitivity to sirolimus or any of its excipients.
  • 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.

Note: Other inclusion/exclusion criteria apply.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Delandistrogene Moxeparvovec
Participants will receive a single intravenous (IV) infusion of delandistrogene moxeparvovec on Day 1.
Genetic: delandistrogene moxeparvovec
Single IV infusion of delandistrogene moxeparvovec
Other Names:
  • SRP-9001
  • delandistrogene moxeparvovec-rokl
  • ELEVIDYS

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Part 1 (Cohorts 1 to 5): Change from Baseline in Quantity of Delandistrogene Moxeparvovec Dystrophin Expression at Week 12, as Measured by Western Blot
Time Frame: Baseline, Week 12
Baseline, Week 12
Part 1 (Cohorts 6 to 8): Quantity of Delandistrogene Moxeparvovec Dystrophin Expression at Week 12 as Measured by Western Blot
Time Frame: Week 12
Week 12
Cohort 8: Number of Participants with Acute Liver Injury (ALI)
Time Frame: Baseline up to Week 72
Baseline up to Week 72

Secondary Outcome Measures

Outcome Measure
Time Frame
Vector Shedding, Measured in Urine, Saliva, and Stool Samples Post-Infusion
Time Frame: Day 1 up to Week 104
Day 1 up to Week 104
Level of Antibody Titers to Recombinant Adeno-Associated Virus Serotype rh74 (rAAVrh74)
Time Frame: Day 2 up to Week 156
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
Time Frame: Baseline, Week 12
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)
Time Frame: Baseline, Week 12
Baseline, Week 12
Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression at Week 12 as Measured by IF PDPF
Time Frame: Week 12
Week 12
Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs) and Adverse Events of Special Interest (AESIs)
Time Frame: Baseline up to Week 156
Baseline up to Week 156
Cohort 8: Number of Participants With Infections, Edema, Wound-healing Complications, Hyperlipidemia, Angioedema, and Intestinal Lung Disease/ Non-infectious Pneumonitis
Time Frame: Baseline up to Week 72
Baseline up to Week 72
Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression at Week 12 as Measured by IF Fiber Intensity
Time Frame: Week 12
Week 12
Cohort 8: Number of Participants With Hepatic AEs, Hepatic Biomarkers, and Laboratory Assessments Indicative of Either Acute Hepatocellular Injury or Acute Liver Dysfunction
Time Frame: Baseline up to Week 72
Baseline up to Week 72
Cohort 8: Number of Participants with Severe ALI
Time Frame: Baseline up to Week 72
Baseline up to Week 72
Cohort 8: Duration of Steroids Administered
Time Frame: Baseline up to Week 72
Baseline up to Week 72

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sarepta Therapeutics, Inc.

Collaborators

Hoffmann-La Roche

Investigators

  • Study Director: Medical Director, Sarepta Therapeutics, Inc.

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

November 23, 2020

Primary Completion (Estimated)

December 31, 2027

Study Completion (Estimated)

February 29, 2028

Study Registration Dates

First Submitted

November 6, 2020

First Submitted That Met QC Criteria

November 6, 2020

First Posted (Actual)

November 12, 2020

Study Record Updates

Last Update Posted (Actual)

June 24, 2026

Last Update Submitted That Met QC Criteria

June 19, 2026

Last Verified

June 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • SRP-9001-103

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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