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

NCT04626674 | Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: SRP-9001-103
• Study Type: interventional
• Target: 83
• Locations: 1 country
• Sites: 5

Brief Summary

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

Conditions

Muscular Dystrophy, Duchenne

Keywords

Duchenne Muscular Dystrophy; Gene-Delivery; DMD; Ambulatory Non-ambulatory; Pediatric; Dystrophin

Outcome Measures

Primary Outcomes (3)

• Part 1 (Cohorts 1 to 5): Change from Baseline in Quantity of Delandistrogene Moxeparvovec Dystrophin Expression at Week 12, as Measured by Western Blot

  Baseline, Week 12

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

  Week 12

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

  Baseline up to Week 72

Secondary Outcomes (11)

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

  Day 1 up to Week 104

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

  Day 2 up to Week 156

• Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs) and Adverse Events of Special Interest (AESIs)

  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

  Baseline up to Week 72

• Change from Baseline in Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression at Week 12, as Measured by Immunofluorescence (IF) Fiber Intensity

  Baseline, Week 12

Study Arms (1)

Delandistrogene Moxeparvovec

EXPERIMENTAL

Participants will receive a single intravenous (IV) infusion of delandistrogene moxeparvovec on Day 1.

Genetic: delandistrogene moxeparvovec

Interventions

delandistrogene moxeparvovec GENETIC

Single IV infusion of delandistrogene moxeparvovec

Also known as: SRP-9001, delandistrogene moxeparvovec-rokl, ELEVIDYS

Delandistrogene Moxeparvovec

Eligibility Criteria

• Age: 2 Years+
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• For Cohorts 1-8: Has a definitive diagnosis of DMD based on documented clinical findings and prior genetic testing.
• 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.
• 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.
• Ability to cooperate with motor assessment testing.
• 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.
• 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.
• rAAVrh74 antibody titers are not elevated as per protocol-specified requirements.

You may not qualify if:

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

Sponsors & Collaborators

• Sarepta Therapeutics, Inc.: lead
• Hoffmann-La Roche: collaborator

Study Sites (5)

Stanford University

Palo Alto, California, 94304, United States

RECRUITING

University of California, Davis

Sacramento, California, 95616, United States

RECRUITING

Washington University in St. Louis

St Louis, Missouri, 21205, United States

RECRUITING

Nationwide Children's Hospital

Columbus, Ohio, 43205, United States

RECRUITING

Children's Hospital of The King's Daughters

Norfolk, Virginia, 23507, United States

RECRUITING

Related Publications (2)

• Potter RA, Moeller IH, Khan S, Haegel H, Hollenstein A, Steiner G, Wandel C, Murphy AP, Asher DR, Palatinsky E, Griffin DA, Mason S, Iannaccone ST, Zaidman CM, Rodino-Klapac LR. Immunologic investigations into transgene directed immune-mediated myositis following delandistrogene moxeparvovec gene therapy. Sci Rep. 2025 Jan 2;15(1):4. doi: 10.1038/s41598-024-84077-w.

  PMID: 39747998 DERIVED

• Zaidman CM, Proud CM, McDonald CM, Lehman KJ, Goedeker NL, Mason S, Murphy AP, Guridi M, Wang S, Reid C, Darton E, Wandel C, Lewis S, Malhotra J, Griffin DA, Potter RA, Rodino-Klapac LR, Mendell JR. Delandistrogene Moxeparvovec Gene Therapy in Ambulatory Patients (Aged >/=4 to <8 Years) with Duchenne Muscular Dystrophy: 1-Year Interim Results from Study SRP-9001-103 (ENDEAVOR). Ann Neurol. 2023 Nov;94(5):955-968. doi: 10.1002/ana.26755. Epub 2023 Sep 7.

  PMID: 37539981 DERIVED

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Condition Hierarchy (Ancestors)

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

Study Officials

• Medical Director

  Sarepta Therapeutics, Inc.

  STUDY DIRECTOR

Central Study Contacts

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

CONTACT

1-888-SAREPTA (1-888-727-3782); SareptAlly@Sarepta.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 6, 2020
• First Posted: November 12, 2020
• Study Start: November 23, 2020
• Primary Completion (Estimated): December 31, 2027
• Study Completion (Estimated): February 29, 2028
• Last Updated: March 4, 2026

Record last verified: 2026-02

Locations

US (5)