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 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

Status

Active, not recruiting

Phases

Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT06128564

Acronym

ENVOL

Enrollment

Registered

2023-11-13

Start date

2023-11-29

Completion date

2034-01-30

Last updated

2026-02-10

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Duchenne Muscular Dystrophy

Keywords

DMD, Duchenne, Dystrophin, Gene-Delivery, Pediatric

Brief 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.

Interventions

GENETICdelandistrogene moxeparvovec

Single IV infusion of delandistrogene moxeparvovec

Study design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

MALE

Age

2 Years to 3 Years

Healthy volunteers

Inclusion criteria

* 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)

Exclusion criteria

* 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

Design outcomes

Primary

Measure	Time frame
Percentage of Participants with a Treatment-emergent Adverse Event (TEAE), Serious Adverse Event (SAE), and Adverse Event of Special Interest (AESI)	Baseline up to Week 260

Secondary

Measure	Time frame
Change in Quantity of Delandistrogene Moxeparvovec Dystrophin as Measured by Western Blot	Baseline, Week 12

Countries

Belgium, France, Germany, Italy, Spain, United Kingdom

Contacts

STUDY_DIRECTORClinical Trials

Hoffmann-La Roche

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 11, 2026