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)
NCT ID: NCT06128564
Last Updated: 2025-12-24
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE2
21 participants
INTERVENTIONAL
2023-11-29
2034-01-30
Brief Summary
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Detailed Description
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Conditions
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Keywords
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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Delandistrogene Moxeparvovec
Participants will receive a single intravenous (IV) infusion of delandistrogene moxeparvovec on Day 1.
delandistrogene moxeparvovec
Single IV infusion of delandistrogene moxeparvovec
Interventions
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delandistrogene moxeparvovec
Single IV infusion of delandistrogene moxeparvovec
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Cohort B: \>=2 years of age to \<3 years of age
* Cohort C: \>6 months to \<2 years of age
* Cohort D: \<=6 months 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
* 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
3 Years
MALE
No
Sponsors
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Sarepta Therapeutics, Inc.
INDUSTRY
Hoffmann-La Roche
INDUSTRY
Responsible Party
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Principal Investigators
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Clinical Trials
Role: STUDY_DIRECTOR
Hoffmann-La Roche
Locations
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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, Italy
Hospital Sant Joan De Deu
Esplugues de Llobregas, Barcelona, Spain
Great Ormond Street Hospital for Children
London, , United Kingdom
John Radcliffe Hospital
Oxford, , United Kingdom
Countries
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Central Contacts
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Reference Study ID Number: BN43881 https://forpatients.roche.com/
Role: CONTACT
Phone: 888-662-6728 (U.S. and Canada)
Email: [email protected]
Other Identifiers
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2022-000691-19
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
2023-509901-57-00
Identifier Type: CTIS
Identifier Source: secondary_id
BN43881
Identifier Type: -
Identifier Source: org_study_id