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Duchenne Muscular DystrophyPhase 3EMBARKNCT05096221

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

A Phase 3 study testing delandistrogene moxeparvovec in people with Duchenne Muscular Dystrophy, run by Sarepta Therapeutics, Inc. at 42 sites in 9 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
Search all trials

Trial 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. Read more about this study

Who is it for?
Ages 4 to 7 years, men only
Conditions the study is for
Duchenne Muscular Dystrophy
What is being tested?
delandistrogene moxeparvovec
Type of treatment
Injection or infusion
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
126 participants
Where
42 sitesin 9 countries
Study dates
27 Oct 2021to 25 Oct 2024
Phase
Phase 3
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 4 to 7 years
  • Men only
  • No healthy volunteers
  • Child

Who may be able to join

  • 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.

Who cannot join

  • 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.
  • Other inclusion or exclusion criteria could apply.

Trial locations and contacts

42 sites in 9 countries.

42 sites in 9 countries

United States23 sites

  • Children's Hospital Colorado

    Aurora, Colorado, 80045

  • The Johns Hopkins Hospital

    Baltimore, Maryland, 21287

  • Boston Children's Hospital

    Boston, Massachusetts, 02115

  • Lurie Children's Hospital of Chicago

    Chicago, Illinois, 60611

  • Nationwide Children's Hospital

    Columbus, Ohio, 43210

  • University of Texas Southwestern

    Dallas, Texas, 75390

  • Duke University Medical Center, Lenox Baker Children's Hospital

    Durham, North Carolina, 27705

  • University of Florida

    Gainesville, Florida, 32608

  • University of Iowa Stead Family Children's Hospital

    Iowa City, Iowa, 52242

  • UC San Diego Altman Clinical and Translational Research Institute

    La Jolla, California, 92037

  • Arkansas Children's

    Little Rock, Arkansas, 72202

  • UCLA Medical Center

    Los Angeles, California, 90095

  • Children's Wisconsin

    Milwaukee, Wisconsin, 53226

  • Vanderbilt University Medical Center

    Nashville, Tennessee, 37232

  • Columbia University/NYPH

    New York, New York, 10032

  • Children's Hospital of the King's Daughters

    Norfolk, Virginia, 23510

  • Lucile Packard Children's Hospital at Stanford

    Palo Alto, California, 94304

  • The Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104

  • Oregon Health & Science University

    Portland, Oregon, 97239

  • University of Rochester

    Rochester, New York, 14642

  • University of California, Davis

    Sacramento, California, 95817

  • University of Utah Hospital

    Salt Lake City, Utah, 84132

  • Washington University of St. Louis

    St Louis, Missouri, 63110

Other trials in United States

Japan4 sites

  • Kobe University Hospital

    Kobe, 650-0017

  • National Center for Child Health and Development

    Tokyo, 157-8535

  • Tokyo Women's Medical University Hospital - Pediatrics

    Tokyo, 162-8666

  • National Center of Neurology and Psychiatry

    Tokyo, 187-8551

Other trials in Japan

Germany3 sites

  • LMU - Klinikum der Universitaet Muenchen - Kinderklinik und

    Bayern, 80337

  • Universitätsklinikum Essen - Klinik für Kinderheilkunde I

    Essen, 45147

  • University Hospital Hamburg- Eppendorf

    Hamburg, 20251

Other trials in Germany

Italy3 sites

  • IRCCS Istituto G.Gaslini, U.O.

    Genoa, 16147

  • UOC Neurologia, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico

    Milan, 20122

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

    Roma, 00168

Other trials in Italy

United Kingdom3 sites

  • Oxford University Hospitals NHS Foundation Trust

    Headington, Oxford, OX3 9DU

  • Great Ormond Street Hospital for Children NHS Foundation Trust

    London, WC1N3JK

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

    Newcastle upon Tyne, NE1 4LP

Other trials in United Kingdom

Spain2 sites

  • Hospital Sant Joan de Déu

    Barcelona, 08950

  • Hospital Universitari i Politécnico La Fe

    Valencia, Comunidad Valencia

Other trials in Spain

Taiwan2 sites

  • Kaohsiung Medical University Chung-Ho Memorial Hospital

    Kaohsiung City, 807

  • National Taiwan University Hospital

    Taipei, 100225

Other trials in Taiwan

Belgium1 site

  • University Hospital Ghent

    Ghent, 9000

Other trials in Belgium

Hong Kong1 site

  • Hong Kong Children's Hospital

    Kowloon

Other trials in Hong Kong

About this study

Official title: A Phase 3 Multinational, Randomized, Double-Blind, Placebo-Controlled Systemic Gene Delivery Study to Evaluate the Safety and Efficacy of SRP-9001 in Subjects With Duchenne Muscular Dystrophy (EMBARK)

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.

Being tested
delandistrogene moxeparvovec, placebo
Keywords
Muscular Dystrophies, Duchenne Muscular Dystrophy, DMD, North Star Ambulatory Assessment (NSAA), Ambulatory, Pediatric, Gene-Delivery
Medical subject headings
Muscular Dystrophy, Duchenne, Muscular Dystrophies

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • delandistrogene moxeparvovecGenetic

    Also called SRP-9001, delandistrogene moxeparvovec-rokl, ELEVIDYS

    Single IV infusion of delandistrogene moxeparvovec.

  • placeboGenetic

    Single IV infusion of matching placebo.

Study groups in NCT05096221
Study groupRoleWhat they receive
Delandistrogene Moxeparvovec followed by PlaceboExperimentalParticipants 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.
Receives: Genetic: delandistrogene moxeparvovec; Genetic: placebo
Placebo followed by Delandistrogene MoxeparvovecPlacebo comparatorParticipants 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.
Receives: Genetic: delandistrogene moxeparvovec; Genetic: placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

  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.

    Measured: Baseline, Week 52 (Part 1)

Secondary outcomes (11)

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

    Quantity of delandistrogene moxeparvovec dystrophin protein levels (in muscle biopsy samples) were determined by Western blot at Part 1, Week 12. For this endpoint, 2 blocks of tissues were analyzed by Western blot, each with 2 technical replicates to determine the delandistrogene moxeparvovec dystrophin protein level (percent control). The block average value from 2 technical replicates was computed. The overall average was calculated as the mean of the block average values. The overall average values were used for the analysis. Dystrophin protein was measured and then adjusted based on the percentage of muscle content in the biopsy sample. An increase in protein expression indicates production of the delandistrogene moxeparvovec dystrophin protein.

    Measured: Week 12

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

    The time to rise from the floor test quantifies the time required for the participant to stand in an upright position with arms by sides, starting from the supine position with arms by sides. Data are presented for the change from baseline to Week 52 in the time taken (in seconds) to rise from the floor.

    Measured: Baseline, Week 52 (Part 1)

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

    The timed 10MWR quantifies the time required for the participant to run or walk 10 meters (on a straight walkway) from a standing position. Data are presented for change from baseline to Week 52 in the time (in seconds) taken to complete the 10MWR.

    Measured: Baseline, Week 52 (Part 1)

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

    The timed 100MWR quantifies the time required for the participant to run or walk 100 meters (on a straight walkway) from a standing position. Data are presented for change from baseline to Week 52 in the time (in seconds) taken to complete the 100MWR.

    Measured: Baseline, Week 52 (Part 1)

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

    The timed stair ascend 4 steps test quantifies the time required for the participant to ascend 4 standard steps (each step was 6 inches in height). Data presented is the change from baseline to Week 52 in the time (in seconds) to ascend 4 steps.

    Measured: Baseline, Week 52 (Part 1)

  6. Part 1: Change From Baseline in Stride Velocity 95th Centile (SV95C) at Week 52

    Each participant was provided with wearable devices to collect data on stride velocity. Participants wore a device on each ankle. SV95C data was derived based on stride velocity. Data are presented for change from baseline to Week 52 in SV95C.

    Measured: Baseline, Week 52 (Part 1)

  7. Part 1: Change From Baseline in Patient-Reported Outcomes Measurement Information System (PROMIS) Score in Mobility to Week 52

    PROMIS is a family of instruments developed and validated to assess health-related quality of life, including mobility. Mobility function scores ranged from 1 (worst mobility function) to 5 (best mobility function), where higher scores indicate a better clinical outcome.

    Measured: Baseline, Week 52 (Part 1)

  8. Part 1: Change From Baseline in PROMIS Score in Upper Extremity Function to Week 52

    PROMIS is a family of instruments developed and validated to assess health-related quality of life, including upper extremity function. Upper extremity function scores ranged from 1 (not able to do so [worst upper extremity function]) to 5 (no trouble [best upper extremity function]), where higher scores indicate a better clinical outcome.

    Measured: Baseline, Week 52 (Part 1)

  9. Part 1: Number of Skills Gained or Improved at Week 52 as Measured by the NSAA

    The NSAA is a clinician-administered scale that rates performance on various functional activities. As measured by the NSAA, data was collected for the number of skills gained (the average item score was 0 at Baseline and > 0 at Part 1 Week 52) or improved (the average item score at Baseline was > 0 but less than the average item score at Part 1 Week 52). As pre-specified, data are presented for the combined number of skills gained or improved at Week 52.

    Measured: Week 52 (Part 1)

  10. Parts 1 and 2: Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)

    TEAEs were defined as an adverse event (AE) that emerged during treatment, having been absent pre-treatment, or worsened relative to the pretreatment state. SAEs were defined as any adverse event that resulted in death, was life threatening, required or prolonged inpatient hospitalization, persistent or significant disability/incapacity, congenital anomaly/birth defect, or was an important medical event. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section.

    Measured: Up to 104 weeks

  11. Parts 1 and 2: Number of Participants With Adverse Events of Special Interest (AESI)

    AESIs were defined as adverse events (AEs) of special interest, based on pre-specified criteria and pertain to categories labeled hepatotoxicity, immune-mediated myositis, thrombotic microangiopathy (TMA), hypersensitivity, thrombocytopenia, rhabdomyolysis, and troponin I elevations. The data represent the number of participants who experienced an event within the specified AESI category as observed by the principal investigator (PI), after adjudication. Per prespecified analysis, AESI data were only collected based on the specified AESI categories. A Summary of all SAEs and nonserious AEs ("Other"), regardless of causality and regardless of the AESI category, is located in the "Reported Adverse Events" section.

    Measured: Up to 104 weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted27 Oct 2021
  2. Study start27 Oct 2021
  3. Primary completion4 Oct 2023
  4. Study completion25 Oct 2024

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
126 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05096221
Sponsor's protocol ID
SRP-9001-301
Other IDs
2019-003374-91, 2019
Acronym
EMBARK
Status last verified
Jun 2025
First submitted
14 Oct 2021
Last updated
8 Jul 2025
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

Publications

  • 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.

    PubMed 39385046

  • 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 Apr;15(2):545-559. doi: 10.1007/s40120-025-00879-8. Epub 2026 Jan 10.

    PubMed 41518520

  • 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. 2026 Feb;15(1):41-60. doi: 10.1007/s40120-025-00842-7. Epub 2025 Nov 3.

    PubMed 41182527

  • 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.

    PubMed 40354061

  • 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.

    PubMed 39589719

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing delandistrogene moxeparvovec in people with Duchenne Muscular Dystrophy, run by Sarepta Therapeutics, Inc. at 42 sites in 9 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 6 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 8 Jul 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 42 sites in United States, Japan, Germany, Italy, United Kingdom and 4 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 10 Dec 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Part 1: Change From Baseline in North Star Ambulatory Assessment (NSAA) Total Score at Week 52 (time frame: Baseline, Week 52 (Part 1)). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05096221, which also lists 5 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Jul 2025.

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