Gene therapy hope for toddlers with duchenne MD

NCT ID NCT06128564

Ongoing Disease control Sponsor: Hoffmann-La Roche Source: ClinicalTrials.gov ↗

First seen Nov 01, 2025 · Last updated Apr 30, 2026 · Updated 29 times

Summary

This study tests a gene therapy (delandistrogene moxeparvovec) in children under 4 with Duchenne muscular dystrophy. The goal is to see if it is safe and helps produce a key muscle protein. About 13 children will receive the treatment and be followed for over 5 years.

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Contacts and locations

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Locations

Chr de La Citadelle

Liège, 3500, Belgium
Great Ormond Street Hospital for Children

London, WC1N 3JH, United Kingdom
Hospital Sant Joan De Deu

Esplugues de Llobregas, Barcelona, 08950, Spain
Hôpital Necker-Enfants Malades

Paris, 75015, France
John Radcliffe Hospital

Oxford, OX3 9DU, United Kingdom
PU A. Gemelli, Università Cattolica del Sacro Cuore

Rome, Lazio, 00168, Italy
Universitätsklinikum Essen

Essen, 45147, Germany

Conditions

Explore the condition pages connected to this study.

DUCHENNE MUSCULAR DYSTROPHY