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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) | Clinical Trials | Clareo Health

ACTIVE_NOT_RECRUITINGPHASE2

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)

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

NCT06128564•Hoffmann-La Roche

View on ClinicalTrials.gov

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

Age

2 - 3 years

Sex

MALE

Healthy Volunteers

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

Locations (7)

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

Key Dates

Start Date

November 29, 2023

Primary Completion Date

February 28, 2030

Completion Date

January 30, 2034

Last Updated

February 10, 2026

Enrollment

ACTUAL participants

Conditions

Duchenne Muscular Dystrophy

Interventions

delandistrogene moxeparvovec

GENETIC

A Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon 44 Skipping to Evaluate the Safety and Efficacy of ENTR-601-44

NCT07037862

Duchenne Muscular Dystrophy (DMD)

View study

RECRUITINGPHASE3

A Study of SGT-003 Gene Therapy in Ambulant Males With Duchenne Muscular Dystrophy (IMPACT DUCHENNE)

NCT07160634

Duchenne Muscular Dystrophy

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RECRUITINGPHASE2

A Study to Assess the Efficacy and Safety of Satralizumab in Duchenne Muscular Dystrophy (DMD)

Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: February 10, 2026Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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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)

Inclusion Criteria

Exclusion Criteria

A Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon 44 Skipping to Evaluate the Safety and Efficacy of ENTR-601-44

A Study of SGT-003 Gene Therapy in Ambulant Males With Duchenne Muscular Dystrophy (IMPACT DUCHENNE)

A Study to Assess the Efficacy and Safety of Satralizumab in Duchenne Muscular Dystrophy (DMD)

Fear of Falling in Muscular Dystrophy

Vasodilator and Exercise Study for DMD (VASO-REx)

DMD Voice: Qualitative Interviews With Patients and Caregivers