Duchenne Muscular DystrophyPhase 3ENVISIONNCT05881408
A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Non-Ambulatory and Ambulatory 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 46 sites in 14 countries.
Trial summary
The study will evaluate the safety and efficacy of delandistrogene moxeparvovec gene transfer therapy in non-ambulatory and ambulatory males with DMD. This is a randomized, double-blind, placebo-controlled 2-part study. Participants will be in the study for approximately 128 weeks. Read more about this study
- Who is it for?
- Any age, 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
- 148 participants
- Where
- 46 sitesin 14 countries
- Study dates
- 31 May 2023to 30 Jun 2028
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
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 Any age
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Definitive diagnosis of DMD based on documented clinical findings and prior genetic testing.
- Cohort 1 only: Non-ambulatory per protocol-specified criteria.
- Cohort 2 only: Ambulatory per protocol-specified criteria and ≥8 to <18 years of age at the time of Screening.
- 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).
- Recombinant Adeno-Associated Virus Serotype rh74 (rAAVrh74) antibody titers are not elevated as per protocol-specified requirements.
- A pathogenic frameshift mutation or premature stop codon in the DMD gene, except for any deletion mutations in exon 8 and/or 9.
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
46 sites in 14 countries.
46 sites in 14 countries
United States14 sites
The Johns Hopkins Hospital, Charlotte R. Bloomberg Children's Center, Pediatric Clinical Research Unit
Baltimore, Maryland, 21287
Boston Children's Hospital
Boston, Massachusetts, 02115
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
Nationwide Children's Hospital
Columbus, Ohio, 43205
Lenox Baker Children's Hospital (Duke University)
Durham, North Carolina, 27705
University of Florida, UF Health Center for Pediatric Neuromuscular and Rare Diseases
Gainesville, Florida, 32608
Arkansas Children's Hospital
Little Rock, Arkansas, 72202
Children's Hospital of the King's Daughters
Norfolk, Virginia, 23510
Lucile Packard Children's Hospital Stanford
Palo Alto, California, 94304
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
University of Rochester, Department of Neurology
Rochester, New York, 14642
University of California at Davis Medical Center
Sacramento, California, 95817
Rady Children's Hospital-San Diego
San Diego, California, 92123
Washington University of St. Louis, St. Louis Children's Hospital
St Louis, Missouri, 63110
Italy4 sites
U.O.S.D Centro Traslazionale di Miologia e Patologie Neurodegenerative, Istituto G. Gaslini, Istituto Pediatrico di Ricovero e Cura a Carattere Scientifico
Genova, 16147
UOC Neurologia, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
Milan, 20122
IRCCS Istituto Neurologico Carlo Besta Neurepsichiatria Infantile 2 - Epilettologia e Neurologia dello Sviluppo
Milan, 20133
UOC Neuropsichiatria Infantile, Area Salute del Bambino, Fondazione Policlinico Universitario A. Gemelli IRCCS
Roma, 00168
South Korea4 sites
Kyungpook National University Hospital
Daegu, 41944
Seoul National University Hospital
Seoul, NAP, 03080
Gangnam Severance Hospital, Yonsei University Health System
Seoul, 06273
Pusan National University Yangsan Hospital
Yangsan, Gyeongsangnam-do, 50612
Canada3 sites
Research Institute McGill University Health Centre
Montreal, Quebec, H4A3J1
The Children's Hospital of Eastern Ontario
Ottawa, Ontario, K1H8L1
Centre Hospitalier Universitaire de Québec - Université Laval (pavillon Centre Hospitalier Universitaire Laval)
Québec, G1V 4G2
Germany3 sites
Universitatsklinikum Essen, Klinik fur Kinderheilkunde I, Abteilung Neuropadiatrie Essen
Essen, North Rhine-Westphalia
Universitatsklinikum Hamburg Eppendorf
Hamburg
LMU- Klinikum der Universitat Munchen, Kinderklinik und Kinderpoliklinik im Dr. von Haunerschen Kinderspital, Abeteilung Neuropadiatrie, Campus Innenstadt
München, Bavaria
Japan3 sites
National Center of Neurology and Psychiatry
Kodaira, Tokyo, 187-8551
Tokyo Women's Medical University Hospital
Shinjuku-ku, Tokyo, 162-866
National Hospital Organization Osaka Toneyama Medical Center
Toyonaka-shi, Osaka, 560-8552
United Kingdom3 sites
Great Ormond Street Hospital for Children Foundation Trust
London, Greater London, WC1N 3JH
Institute of Translational and Clinical Research
Newcastle upon Tyne, NE13BZ
Oxford University Hospitals NHS Foundation Trust
Oxford, Oxfordshire
Australia2 sites
The Royal Children's Hospital
Parkville, Victoria, 3052
The Children's Hospital at Westmead
Westmead, New South Wales, 2145
Israel2 sites
Institute of Neruology, Schneider Children's Medical Center of Israel
Petah Tikva
Tel Aviv Sourasky Medical Center
Tel Aviv, 6423906
Spain2 sites
Hospital Sant Joan de Deu
Esplugues de Llobregat, Barcelona, 08950
Hospital Universitari Politecnic La Fe
Valencia, 46026
Sweden2 sites
Sahlgrenska Universitetssjukhuset
Gothenburg, 416 85
Karolinska Universitetssjukhuset/Astrid Lindgrens Barnsjukhus, Barnneurologen
Solna, 171 76
Taiwan2 sites
Kaohsiung Medical University Chung-Ho Memorial Hospital
Kaohsiung City
National Taiwan University Hospital
Taipei
About this study
Official title: A Phase 3, Multinational, Randomized, Double-Blind, Placebo-Controlled Systemic Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of SRP- 9001 in Non-Ambulatory and Ambulatory Subjects With Duchenne Muscular Dystrophy (ENVISION)
The study will evaluate the safety and efficacy of delandistrogene moxeparvovec gene transfer therapy in non-ambulatory and ambulatory males with DMD. This is a randomized, double-blind, placebo-controlled 2-part study. Participants will be in the study for approximately 128 weeks. All participants will have the opportunity to receive intravenous (IV) delandistrogene moxeparvovec in either Part 1 or Part 2.
- Conditions
- Duchenne Muscular Dystrophy
- Being tested
- delandistrogene moxeparvovec, placebo
- Keywords
- DMD, Gene-Delivery, Pediatric, North Star Ambulatory Assessment (NSAA), Performance of Upper Limb (PUL), Duchenne
- Medical subject headings
- Muscular Dystrophy, Duchenne
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 group | Role | What they receive |
|---|---|---|
| Delandistrogene Moxeparvovec followed by Placebo | Experimental | Participants will receive single IV infusion of delandistrogene moxeparvovec on Day 1. Then, participants will receive a single IV infusion of matching placebo at approximately 72 weeks. Receives: 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 approximately 72 weeks. Receives: Genetic: delandistrogene moxeparvovec; Genetic: placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Part 1: Change From Baseline in the Total Score of Performance of Upper Limb (PUL) (Version 2.0) at Week 72
Measured: Baseline, Week 72
Secondary outcomes (8)
Part 1: Change From Baseline in Percent Predicted Forced Vital Capacity (FVC) at Week 72
Measured: Baseline, Week 72
Part 1: Change From Baseline in Percent Predicted Peak Expiratory Flow (PEF) at Week 72
Measured: Baseline, Week 72
Part 1: Quantity of Delandistrogene Moxeparvovec Dystrophin Expression at Week 12 as Measured by Western Blot
Measured: Week 12
Part 1: Change From Baseline in Patient-Reported Outcomes Measurement Information (PROMIS) Score in Upper Extremity Function to Week 72
Measured: Baseline, Week 72
Number of Participants with a Treatment Emergent Adverse Event (TEAE), Adverse Event of Special Interest (AESI), and Serious Adverse Event (SAE)
Measured: Baseline up to Week 124
Part 1 (For Cohort 2 Only): Change From Baseline in the North Star Ambulatory Assessment (NSAA) Total Score at Week 72
Measured: Baseline, Week 72
Part 1: Change From Baseline in Global Circumferential Strain as Measured by Cardiac MRI at Week 72
Measured: Baseline, Week 72
Part 1: Change From Baseline in the Middle Domain Score of PUL (Version 2.0) at Week 72
Measured: Baseline, Week 72
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 May 2023
- Study start31 May 2023
- Primary completion31 May 2027 (estimated)
- Study completion30 Jun 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Duchenne Muscular DystrophyStudies of the same condition, open and under way.
- More trials run by Sarepta Therapeutics, Inc.The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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
- 148 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Sarepta Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Hoffmann-La Roche
- Responsible party
- Sponsor
- Investigators
- Medical Director, Study director, Sarepta Therapeutics, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05881408
- Sponsor's protocol ID
- SRP-9001-303
- Other IDs
- 2020-002372-13, 2024-512626-28-00
- Acronym
- ENVISION
- Status last verified
- May 2026
- First submitted
- 19 May 2023
- Last updated
- 22 May 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 22 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 3, Multinational, Randomized, Double-Blind, Placebo-Controlled Systemic Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of SRP- 9001 in Non-Ambulatory and Ambulatory Subjects With Duchenne Muscular Dystrophy (ENVISION)
- ClinicalTrials.gov ID
- NCT05881408
- Acronym / sponsor ID
- ENVISION / SRP-9001-303
- Conditions
- Duchenne Muscular Dystrophy
- Keywords
- DMD, Gene-Delivery, Pediatric, North Star Ambulatory Assessment (NSAA), Performance of Upper Limb (PUL), Duchenne
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 148 participants (estimated)
- Lead sponsor
- Sarepta Therapeutics, Inc.Industry (pharma, biotech, devices)
- Collaborators
- Hoffmann-La Roche
- Study start
- 31 May 2023 (actual)
- Primary completion
- 31 May 2027 (estimated)
- Study completion
- 30 Jun 2028 (estimated)
- First posted
- 31 May 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The study will evaluate the safety and efficacy of delandistrogene moxeparvovec gene transfer therapy in non-ambulatory and ambulatory males with DMD. This is a randomized, double-blind, placebo-controlled 2-part study. Participants will be in the study for approximately 128 weeks. All participants will have the opportunity to receive intravenous (IV) delandistrogene moxeparvovec in either Part 1 or Part 2.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Delandistrogene Moxeparvovec followed by Placebo | Experimental | Genetic: delandistrogene moxeparvovec, Genetic: placeboParticipants will receive single IV infusion of delandistrogene moxeparvovec on Day 1. Then, participants will receive a single IV infusion of matching placebo at approximately 72 weeks. |
| Placebo followed by Delandistrogene Moxeparvovec | Placebo comparator | Genetic: delandistrogene moxeparvovec, Genetic: placeboParticipants 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 approximately 72 weeks. |
Who may qualify
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (7 listed)
- Definitive diagnosis of DMD based on documented clinical findings and prior genetic testing.
- Cohort 1 only: Non-ambulatory per protocol-specified criteria.
- Cohort 2 only: Ambulatory per protocol-specified criteria and ≥8 to <18 years of age at the time of Screening.
- 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).
Key exclusion criteria (4 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| delandistrogene moxeparvovecAlso: SRP-9001, delandistrogene moxeparvovec-rokl, ELEVIDYSSingle IV infusion of delandistrogene moxeparvovec | Genetic | Delandistrogene Moxeparvovec followed by Placebo, Placebo followed by Delandistrogene Moxeparvovec |
| placeboSingle IV infusion of matching placebo | Genetic | Delandistrogene Moxeparvovec followed by Placebo, Placebo followed by Delandistrogene Moxeparvovec |
Primary outcome
Part 1: Change From Baseline in the Total Score of Performance of Upper Limb (PUL) (Version 2.0) at Week 72
Time frame: Baseline, Week 72
8 secondary outcomes
Part 1: Change From Baseline in Percent Predicted Forced Vital Capacity (FVC) at Week 72
Time frame: Baseline, Week 72
Part 1: Change From Baseline in Percent Predicted Peak Expiratory Flow (PEF) at Week 72
Time frame: Baseline, Week 72
Part 1: Quantity of Delandistrogene Moxeparvovec Dystrophin Expression at Week 12 as Measured by Western Blot
Time frame: Week 12
Part 1: Change From Baseline in Patient-Reported Outcomes Measurement Information (PROMIS) Score in Upper Extremity Function to Week 72
Time frame: Baseline, Week 72
Number of Participants with a Treatment Emergent Adverse Event (TEAE), Adverse Event of Special Interest (AESI), and Serious Adverse Event (SAE)
Time frame: Baseline up to Week 124
Part 1 (For Cohort 2 Only): Change From Baseline in the North Star Ambulatory Assessment (NSAA) Total Score at Week 72
Time frame: Baseline, Week 72
Part 1: Change From Baseline in Global Circumferential Strain as Measured by Cardiac MRI at Week 72
Time frame: Baseline, Week 72
Part 1: Change From Baseline in the Middle Domain Score of PUL (Version 2.0) at Week 72
Time frame: Baseline, Week 72
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 May 2026.
- Click here to access the website, clinicaltrials.sarepta.com/ENVISION, for additional information for the study.Link provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Medical Director study director
Sarepta Therapeutics, Inc.
46 sites in 14 countries: see every site and its status.
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 46 sites in 14 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 7 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: under way, not looking for participants, as of the sponsor's last update on 22 May 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 46 sites in United States, Italy, South Korea, Canada, Germany and 9 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?
This study is still under way. Results are not available in the source record yet.
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 the Total Score of Performance of Upper Limb (PUL) (Version 2.0) at Week 72 (time frame: Baseline, Week 72). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 22 May 2026.