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

NCT05096221 | Completed Phase 3 Results DMC FDA Drug

• 2 other identifiers: SRP-9001-3012019-003374-91
• Study Type: interventional
• Target: 126
• Locations: 9 countries
• Sites: 42

Brief Summary

The study will evaluate the safety and efficacy of gene transfer therapy in boys with DMD. It is a randomized, double-blind, placebo-controlled study. The participants who are randomized to the placebo arm will have an opportunity for treatment with gene transfer therapy at the beginning of the second year.

Conditions

Duchenne Muscular Dystrophy

Keywords

Muscular Dystrophies; Duchenne Muscular Dystrophy; DMD; North Star Ambulatory Assessment (NSAA); Ambulatory; Pediatric; Gene-Delivery

Outcome Measures

Primary Outcomes (1)

• Part 1: Change From Baseline in North Star Ambulatory Assessment (NSAA) Total Score at Week 52

  The NSAA is a healthcare provider administered scale that rates performance of various motor abilities in ambulant children with Duchenne Muscular Dystrophy and is used to monitor disease progression and treatment effects. During assessment, participants are asked to perform 17 different functional activities that are graded as: 2 - "Normal" - no obvious modification of activity; 1 - Modified method but achieves goal independent of assistance; 0 - Unable to achieve independently. The NSAA total score is defined as the sum of all 17 items, ranging from 0 (worst) to 34 (best). The response vector consists of the change from baseline in NSAA total score at the post-baseline visit. The model includes the covariates of treatment group, visit, treatment group by visit interaction, age group, baseline NSAA total score, and baseline NSAA total score by visit interaction. All covariates are fixed effects in this analysis. An increase in score indicates an improvement in motor function.

  Baseline, Week 52 (Part 1)

Secondary Outcomes (11)

• Part 1: Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression at Week 12 as Measured by Western Blot Adjusted by Muscle Content

  Week 12

• Part 1: Change From Baseline in Time to Rise From the Floor at Week 52

  Baseline, Week 52 (Part 1)

• Part 1: Change From Baseline in Time to Complete 10 Meter Walk/Run (10MWR) at Week 52

  Baseline, Week 52 (Part 1)

• Part 1: Change From Baseline in Time to Complete 100 Meter Walk/Run (100MWR) at Week 52

  Baseline, Week 52 (Part 1)

• Part 1: Change From Baseline in the Timed Stair Ascend 4 Steps Test at Week 52

  Baseline, Week 52 (Part 1)

Study Arms (2)

Delandistrogene Moxeparvovec followed by Placebo

EXPERIMENTAL

Participants will receive single intravenous (IV) infusion of delandistrogene moxeparvovec on Day 1. Then, participants will receive a single IV infusion of matching placebo at Year 2.

Genetic: delandistrogene moxeparvovec; Genetic: placebo

Placebo followed by Delandistrogene Moxeparvovec

PLACEBO COMPARATOR

Participants will receive matching placebo IV infusion on Day 1. Then, participants will have the opportunity to receive a single IV infusion of delandistrogene moxeparvovec at Year 2.

Genetic: delandistrogene moxeparvovec; Genetic: placebo

Interventions

delandistrogene moxeparvovec GENETIC

Single IV infusion of delandistrogene moxeparvovec.

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

Delandistrogene Moxeparvovec followed by Placebo; Placebo followed by Delandistrogene Moxeparvovec

placebo GENETIC

Single IV infusion of matching placebo.

Delandistrogene Moxeparvovec followed by Placebo; Placebo followed by Delandistrogene Moxeparvovec

Eligibility Criteria

• Age: 4 Years - 7 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Is ambulatory and from 4 to under 8 years of age at time of randomization.
• Definitive diagnosis of DMD based on documented clinical findings and prior genetic testing.
• Ability to cooperate with motor assessment testing.
• Stable daily dose of oral corticosteroids for at least 12 weeks prior to Screening, and the dose is expected to remain constant throughout the study (except for modifications to accommodate changes in weight).
• rAAVrh74 antibody titers are not elevated as per protocol-specified requirements.
• A pathogenic frameshift mutation or premature stop codon contained between exons 18 and 79 (inclusive), with the exception of mutation fully contained within exon 45.

You may not qualify if:

• 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.
• Presence of any other clinically significant illness, medical condition, or requirement for chronic drug treatment that in the opinion of the Investigator creates unnecessary risk for gene transfer.

Sponsors & Collaborators

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

Study Sites (42)

Arkansas Children's

Little Rock, Arkansas, 72202, United States

Location

UC San Diego Altman Clinical and Translational Research Institute

La Jolla, California, 92037, United States

Location

UCLA Medical Center

Los Angeles, California, 90095, United States

Location

Lucile Packard Children's Hospital at Stanford

Palo Alto, California, 94304, United States

Location

University of California, Davis

Sacramento, California, 95817, United States

Location

Children's Hospital Colorado

Aurora, Colorado, 80045, United States

Location

University of Florida

Gainesville, Florida, 32608, United States

Location

Lurie Children's Hospital of Chicago

Chicago, Illinois, 60611, United States

Location

University of Iowa Stead Family Children's Hospital

Iowa City, Iowa, 52242, United States

Location

The Johns Hopkins Hospital

Baltimore, Maryland, 21287, United States

Location

Boston Children's Hospital

Boston, Massachusetts, 02115, United States

Location

Washington University of St. Louis

St Louis, Missouri, 63110, United States

Location

Columbia University/NYPH

New York, New York, 10032, United States

Location

University of Rochester

Rochester, New York, 14642, United States

Location

Duke University Medical Center, Lenox Baker Children's Hospital

Durham, North Carolina, 27705, United States

Location

Nationwide Children's Hospital

Columbus, Ohio, 43210, United States

Location

Oregon Health & Science University

Portland, Oregon, 97239, United States

Location

The Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

Vanderbilt University Medical Center

Nashville, Tennessee, 37232, United States

Location

University of Texas Southwestern

Dallas, Texas, 75390, United States

Location

University of Utah Hospital

Salt Lake City, Utah, 84132, United States

Location

Children's Hospital of the King's Daughters

Norfolk, Virginia, 23510, United States

Location

Children's Wisconsin

Milwaukee, Wisconsin, 53226, United States

Location

University Hospital Ghent

Ghent, 9000, Belgium

Location

LMU - Klinikum der Universitaet Muenchen - Kinderklinik und

Bayern, 80337, Germany

Location

Universitätsklinikum Essen - Klinik für Kinderheilkunde I

Essen, 45147, Germany

Location

University Hospital Hamburg- Eppendorf

Hamburg, 20251, Germany

Location

Hong Kong Children's Hospital

Kowloon, Hong Kong

Location

IRCCS Istituto G.Gaslini, U.O.

Genoa, 16147, Italy

Location

UOC Neurologia, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico

Milan, 20122, Italy

Location

UOC Neuropsichiatria Infantile, Area Salute del Bambino, Fondazione Policlinico Universitario A. Gamelli IRCCS

Roma, 00168, Italy

Location

Kobe University Hospital

Kobe, 650-0017, Japan

Location

National Center for Child Health and Development

Tokyo, 157-8535, Japan

Location

Tokyo Women's Medical University Hospital - Pediatrics

Tokyo, 162-8666, Japan

Location

National Center of Neurology and Psychiatry

Tokyo, 187-8551, Japan

Location

Hospital Universitari i Politécnico La Fe

Valencia, Comunidad Valencia, Spain

Location

Hospital Sant Joan de Déu

Barcelona, 08950, Spain

Location

Kaohsiung Medical University Chung-Ho Memorial Hospital

Kaohsiung City, 807, Taiwan

Location

National Taiwan University Hospital

Taipei, 100225, Taiwan

Location

Oxford University Hospitals NHS Foundation Trust

Headington, Oxford, OX3 9DU, United Kingdom

Location

Great Ormond Street Hospital for Children NHS Foundation Trust

London, WC1N3JK, United Kingdom

Location

The Newcastle Upon Tyne NHS Hospital NHS Foundation Trust, Royal Victoria Infirmary

Newcastle upon Tyne, NE1 4LP, United Kingdom

Location

Related Publications (5)

• Mendell JR, Muntoni F, McDonald CM, Mercuri EM, Ciafaloni E, Komaki H, Leon-Astudillo C, Nascimento A, Proud C, Schara-Schmidt U, Veerapandiyan A, Zaidman CM, Guridi M, Murphy AP, Reid C, Wandel C, Asher DR, Darton E, Mason S, Potter RA, Singh T, Zhang W, Fontoura P, Elkins JS, Rodino-Klapac LR. AAV gene therapy for Duchenne muscular dystrophy: the EMBARK phase 3 randomized trial. Nat Med. 2025 Jan;31(1):332-341. doi: 10.1038/s41591-024-03304-z. Epub 2024 Oct 9.

  PMID: 39385046 RESULT

• Mendell JR, Muntoni F, McDonald CM, Mercuri EM, Ciafaloni E, Komaki H, Leon-Astudillo C, Nascimento A, Proud C, Schara-Schmidt U, Veerapandiyan A, Zaidman CM, Furgerson M, Ding K, Singh P, Potter R, Asher DR, Murphy AP, Reid C, Hooper G, Torre CO, Manfrini M, Rodino-Klapac LR. Two-Year Outcomes Following Delandistrogene Moxeparvovec Treatment in Ambulatory Patients with Duchenne Muscular Dystrophy: Phase 3 EMBARK Trial. Neurol Ther. 2026 Jan 10. doi: 10.1007/s40120-025-00879-8. Online ahead of print.

  PMID: 41518520 DERIVED

• Audhya I, Nacson AB, Gooch K, Basnyat B, Slota C, Martin S, Murphy A, Lansdall CJ, Ciobanu T, Nascimento A, Veerapandiyan A. Caregiver-reported Patient Experiences with Duchenne Muscular Dystrophy: Qualitative In-trial Interviews 1 Year After Delandistrogene Moxeparvovec in the Pivotal EMBARK Trial. Neurol Ther. 2025 Nov 3. doi: 10.1007/s40120-025-00842-7. Online ahead of print.

  PMID: 41182527 DERIVED

• Vandenborne K, Walter GA, Straub V, Willcocks RJ, Forbes SC, Mercuri EM, Muntoni F, Ding K, Ennamuri S, Reid C, Murphy AP, Manfrini M, Mendell JR, Elkins JS, Rodino-Klapac LR. Quantitative Muscle Magnetic Resonance Outcomes in Patients With Duchenne Muscular Dystrophy: An Exploratory Analysis From the EMBARK Randomized Clinical Trial. JAMA Neurol. 2025 Jul 1;82(7):734-744. doi: 10.1001/jamaneurol.2025.0992.

  PMID: 40354061 DERIVED

• McDonald CM, Elkins JS, Dharmarajan S, Gooch K, Ciobanu T, Lansdall CJ, Murphy AP, McDougall F, Mercuri EM, Audhya I; EMBARK Study Group. Caregiver Global Impression Observations from EMBARK: A Phase 3 Study Evaluating Delandistrogene Moxeparvovec in Ambulatory Patients with Duchenne Muscular Dystrophy. Neurol Ther. 2025 Feb;14(1):211-225. doi: 10.1007/s40120-024-00685-8. Epub 2024 Nov 26.

  PMID: 39589719 DERIVED

Related Links

• SRP-9001-301 Plain Language Study Summary

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne; Muscular Dystrophies

Condition Hierarchy (Ancestors)

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

Results Point of Contact

• Title: Sarepta Therapeutics Inc.
• Organization: Sarepta Therapeutics Inc.

SareptAlly@sarepta.com

1-888-727-3782

Study Officials

• Medical Director

  Sarepta Therapeutics, Inc.

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 14, 2021
• First Posted: October 27, 2021
• Study Start: October 27, 2021
• Primary Completion: October 4, 2023
• Study Completion: October 25, 2024
• Last Updated: July 8, 2025
• Results First Posted: December 10, 2024

Record last verified: 2025-06

Locations

TW (2); GB (3); BE (1); HK (1); DE (3); US (23); JP (4); IT (3); ES (2)