Duchenne Muscular DystrophyPhase 2ENVOLNCT06128564
A Gene Delivery Study to Evaluate the Safety and Expression of Delandistrogene Moxeparvovec in Participants Under the Age of Four With Duchenne Muscular Dystrophy (DMD)
A Phase 2 study testing delandistrogene moxeparvovec in people with Duchenne Muscular Dystrophy, run by Hoffmann-La Roche at 7 sites in 6 countries.
Trial summary
This open-label, single-arm study will evaluate the safety and expression of delandistrogene moxeparvovec in participants with DMD. Participants will be in the study for approximately 264 weeks. Read more about this study
- Who is it for?
- Ages 2 to 3 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?
- No, everyone receives an active treatment
- How many people
- 13 participants
- Where
- 7 sitesin 6 countries
- Study dates
- 29 Nov 2023to 12 Feb 2030
- Phase
- Phase 2
- 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 2 to 3 years
- Men only
- No healthy volunteers
- Child
Who may be able to join
- Cohort A: >=3 years of age to <4 years of age
- Cohort B: >=2 years of age to <3 years of age
- Has a definitive diagnosis of DMD prior to screening based on documentation of clinical findings and prior confirmatory genetic testing using a clinical diagnostic genetic test
- Able to cooperate with age-appropriate motor assessment testing
- A pathogenic frameshift mutation or premature stop codon contained between exons 18 and 79 (inclusive)
Who cannot join
- Exposure to gene therapy, investigational medication, or any treatment designed to increase dystrophin expression, within protocol-specified time limits
- Recombinant Adeno-Associated Virus Serotype rh74 (rAArh74) antibody titers are elevated, as per protocol-specified criteria
- Receiving regular oral corticosteroids as a treatment for DMD or planning to receive oral corticosteroids as a treatment for DMD within 1 year of baseline
- 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
- Medical condition or extenuating circumstance that, in the opinion of the investigator, might compromise the participant's ability to comply with the protocol required testing or procedures, or compromise the participant's well-being or safety, or clinical interpretability
- Other inclusion or exclusion criteria could apply
Trial locations and contacts
7 sites in 6 countries.
7 sites in 6 countries
United Kingdom2 sites
Great Ormond Street Hospital for Children
London, WC1N 3JH
John Radcliffe Hospital
Oxford, OX3 9DU
Italy1 site
PU A. Gemelli, Università Cattolica del Sacro Cuore
Rome, Lazio, 00168
About this study
Official title: A Two-Part, Open-Label Systemic Gene Delivery Study to Evaluate the Safety and Expression of RO7494222 (SRP-9001) in Subjects Under the Age of Four With Duchenne Muscular Dystrophy
This open-label, single-arm study will evaluate the safety and expression of delandistrogene moxeparvovec in participants with DMD. Participants will be in the study for approximately 264 weeks.
- Conditions
- Duchenne Muscular Dystrophy
- Being tested
- delandistrogene moxeparvovec
- Keywords
- DMD, Duchenne, Dystrophin, Gene-Delivery, Pediatric
- 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, ELEVIDYS, RO7494222, delandistrogene moxeparvovec-rokl
Single IV infusion of delandistrogene moxeparvovec
| Study group | Role | What they receive |
|---|---|---|
| Delandistrogene Moxeparvovec | Experimental | Participants will receive a single intravenous (IV) infusion of delandistrogene moxeparvovec on Day 1. Receives: Genetic: delandistrogene moxeparvovec |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage of Participants with a Treatment-emergent Adverse Event (TEAE), Serious Adverse Event (SAE), and Adverse Event of Special Interest (AESI)
Measured: Baseline up to Week 260
Secondary outcomes (1)
Change in Quantity of Delandistrogene Moxeparvovec Dystrophin as Measured by Western Blot
Measured: Baseline, Week 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Nov 2023
- Study start29 Nov 2023
- Primary completion4 Mar 2026
- Study completion12 Feb 2030 (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 Hoffmann-La RocheThe 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 13 participants (actual)
Sponsor and investigators
- Lead sponsor
- Hoffmann-La Roche
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Sarepta Therapeutics, Inc.
- Responsible party
- Sponsor
- Investigators
- Clinical Trials, Study director, Hoffmann-La Roche
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06128564
- Sponsor's protocol ID
- BN43881
- Other IDs
- 2022-000691-19, 2023-509901-57-00, 2023
- Acronym
- ENVOL
- Status last verified
- Sep 2026
- First submitted
- 8 Nov 2023
- Last updated
- 9 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: For eligible studies, qualified researchers may request access to individual patient level clinical data. See Roche's commitment to transparency of clinical study information here: https://go.roche.com/data\_sharing
Study at a glance
From the registry record, last updated by the sponsor on 9 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Two-Part, Open-Label Systemic Gene Delivery Study to Evaluate the Safety and Expression of RO7494222 (SRP-9001) in Subjects Under the Age of Four With Duchenne Muscular Dystrophy
- ClinicalTrials.gov ID
- NCT06128564
- Acronym / sponsor ID
- ENVOL / BN43881
- Conditions
- Duchenne Muscular Dystrophy
- Keywords
- DMD, Duchenne, Dystrophin, Gene-Delivery, Pediatric
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 13 participants (actual)
- Lead sponsor
- Hoffmann-La RocheIndustry (pharma, biotech, devices)
- Collaborators
- Sarepta Therapeutics, Inc.
- Study start
- 29 Nov 2023 (actual)
- Primary completion
- 4 Mar 2026 (actual)
- Study completion
- 12 Feb 2030 (estimated)
- First posted
- 13 Nov 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.
This open-label, single-arm study will evaluate the safety and expression of delandistrogene moxeparvovec in participants with DMD. Participants will be in the study for approximately 264 weeks.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Delandistrogene Moxeparvovec | Experimental | Genetic: delandistrogene moxeparvovecParticipants will receive a single intravenous (IV) infusion of delandistrogene moxeparvovec on Day 1. |
Who may qualify
- Ages
- 2 to 3 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (5 listed)
- Cohort A: >=3 years of age to <4 years of age
- Cohort B: >=2 years of age to <3 years of age
- Has a definitive diagnosis of DMD prior to screening based on documentation of clinical findings and prior confirmatory genetic testing using a clinical diagnostic genetic test
- Able to cooperate with age-appropriate motor assessment testing
- A pathogenic frameshift mutation or premature stop codon contained between exons 18 and 79 (inclusive)
Key exclusion criteria (6 listed)
- Exposure to gene therapy, investigational medication, or any treatment designed to increase dystrophin expression, within protocol-specified time limits
- Recombinant Adeno-Associated Virus Serotype rh74 (rAArh74) antibody titers are elevated, as per protocol-specified criteria
- Receiving regular oral corticosteroids as a treatment for DMD or planning to receive oral corticosteroids as a treatment for DMD within 1 year of baseline
- 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
- Medical condition or extenuating circumstance that, in the opinion of the investigator, might compromise the participant's ability to comply with the protocol required testing or procedures, or compromise the participant's well-being or safety, or clinical interpretability
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, ELEVIDYS, RO7494222, delandistrogene moxeparvovec-roklSingle IV infusion of delandistrogene moxeparvovec | Genetic | Delandistrogene Moxeparvovec |
Primary outcome
Percentage of Participants with a Treatment-emergent Adverse Event (TEAE), Serious Adverse Event (SAE), and Adverse Event of Special Interest (AESI)
Time frame: Baseline up to Week 260
1 secondary outcome
Change in Quantity of Delandistrogene Moxeparvovec Dystrophin as Measured by Western Blot
Time frame: Baseline, Week 12
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 9 Sep 2026.
- Individual participant dataSharing planned: Yes. For eligible studies, qualified researchers may request access to individual patient level clinical data. See Roche's commitment to transparency of clinical study information here: https://go.roche.com/data_sharing
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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.
Clinical Trials study director
Hoffmann-La Roche
7 sites in 6 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing delandistrogene moxeparvovec in people with Duchenne Muscular Dystrophy, run by Hoffmann-La Roche at 7 sites in 6 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 5 inclusion and 6 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 9 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites in United Kingdom, Belgium, France, Germany, Italy and 1 more country. 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. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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?
Percentage of Participants with a Treatment-emergent Adverse Event (TEAE), Serious Adverse Event (SAE), and Adverse Event of Special Interest (AESI) (time frame: Baseline up to Week 260). 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 9 Sep 2026.