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NCT Number: NCT06128564

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)

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.

Active, Not Recruiting

This study is active but is not currently recruiting participants.

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Key information

Age range

2 year–3 year

Sex eligibility

Male

Study type

Interventional

Phase

Phase 2

Primary location

Chr de La Citadelle, Liège, Belgium

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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 exclusion criteria could apply

Treatment and study plan

delandistrogene moxeparvovec

Genetic

Single IV infusion of delandistrogene moxeparvovec

Other names: SRP-9001, ELEVIDYS, RO7494222, delandistrogene moxeparvovec-rokl

Primary outcomes

  1. 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

Secondary outcomes

  1. Change in Quantity of Delandistrogene Moxeparvovec Dystrophin as Measured by Western Blot

    Time frame: Baseline, Week 12

Sponsors and collaborators

Lead sponsor

Hoffmann-La Roche

Industry

Collaborators

  • Sarepta Therapeutics, Inc.

Registry information

Official study 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

Acronym: ENVOL

Important dates

Study start
2023
Primary completion
2026
Study completion
2030
First posted
Nov 13, 2023
Registry last updated
May 11, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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