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)

Plain-language summary

This study evaluates the safety of Delandistrogene moxeparvovec, a gene therapy, and whether it enables dystrophin production in children under four with Duchenne muscular dystrophy (DMD). Children aged 2-4 with a genetically confirmed DMD diagnosis and a mutation between exons 18 and 79 took part. It is being conducted in Phase 2; it is active but not currently recruiting.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

Phase 2

Status

Active, not recruiting

Sponsor

Hoffmann-La Roche

Intervention

delandistrogene moxeparvovec

Start date

11/29/2023

Estimated completion

2/18/2030

Countries

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

Related genes

Locations

• Liège, Belgium
• Paris, France
• Essen, Germany
• Rome, Italy
• Esplugues de Llobregas, Spain
• London, United Kingdom
• Oxford, United Kingdom

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

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