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Clinical Trials in the UK / NCT06128564
Active, not recruiting Phase 2

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)

NCT06128564 · tracked via the Priya Life Science UK tracker
Sponsor
Hoffmann-La Roche
Phase
Phase 2
Started
2023-11-29
Last updated
2026-09-09

Condition(s) studied

Duchenne Muscular Dystrophy

Investigational drug(s) / intervention(s)

delandistrogene moxeparvovec

delandistrogene moxeparvovec: Single IV infusion of delandistrogene moxeparvovec

Study 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.

Eligibility

Sex
MALE
Min age
2 Years
Max age
3 Years
Healthy volunteers
No
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

Primary outcome measure(s)

Trial sites (7)

FacilityCityRegionStatus
Chr de La Citadelle Liège Belgium
Hôpital Necker-Enfants Malades Paris France
Universitätsklinikum Essen Essen Germany
PU A. Gemelli, Università Cattolica del Sacro Cuore Rome Lazio
Hospital Sant Joan De Deu Esplugues de Llobregas Barcelona
Great Ormond Street Hospital for Children London United Kingdom
John Radcliffe Hospital Oxford United Kingdom
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06128564 on ClinicalTrials.gov ↗ ← All trials in the UK