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)
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)
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
Trial sites (7)
Facility
City
Region
Status
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.
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