Skip to main content
OpenTrials
Recruiting

NCT Number: NCT04626674

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

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.

Recruiting

Interested in participating?

Request Info

Key information

Conditions

Duchenne Muscular Dystrophy Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Genetic Diseases, X-Linked Muscular Diseases Muscular Disorders, Atrophic Muscular Dystrophies Muscular Dystrophy, Duchenne Musculoskeletal Diseases Nervous System Diseases Neuromuscular Diseases

Age range

2 year and older

Sex eligibility

Male

Study type

Interventional

Phase

Phase 1

Primary location

Arkansas Children's Hospital, Little Rock, Arkansas, United States

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

delandistrogene moxeparvovec

Genetic

Single IV infusion of delandistrogene moxeparvovec

Other names: SRP-9001, delandistrogene moxeparvovec-rokl, ELEVIDYS

Primary outcomes

  1. 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

  2. Part 1 (Cohorts 6 to 8): Quantity of Delandistrogene Moxeparvovec Dystrophin Expression at Week 12 as Measured by Western Blot

    Time frame: Week 12

  3. Cohort 8: Number of Participants with Acute Liver Injury (ALI)

    Time frame: Baseline up to Week 72

Secondary outcomes

  1. Vector Shedding, Measured in Urine, Saliva, and Stool Samples Post-Infusion

    Time frame: Day 1 up to Week 104

  2. Level of Antibody Titers to Recombinant Adeno-Associated Virus Serotype rh74 (rAAVrh74)

    Time frame: Day 2 up to Week 156

  3. 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

  4. 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

  5. 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

  6. 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

  7. Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression at Week 12 as Measured by IF Fiber Intensity

    Time frame: Week 12

  8. Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression at Week 12 as Measured by IF PDPF

    Time frame: Week 12

  9. 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

  10. Cohort 8: Number of Participants with Severe ALI

    Time frame: Baseline up to Week 72

  11. Cohort 8: Duration of Steroids Administered

    Time frame: Baseline up to Week 72

Study contacts

Contact information is provided by the study sponsor or research team.

Sarepta Therapeutics Inc., For Clinical Trial Information, Select Option 4

CONTACT

[email protected]

1-888-SAREPTA (1-888-727-3782)

Sponsors and collaborators

Lead sponsor

Sarepta Therapeutics, Inc.

Industry

Collaborators

  • Hoffmann-La Roche

Registry information

Official study 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)

Acronym: ENDEAVOR

Important dates

Study start
2020
Primary completion
2027
Study completion
2028
First posted
Nov 12, 2020
Registry last updated
Jun 24, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.