Gene therapy trial targets duchenne in toddlers

NCT ID NCT06128564

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

First seen Jun 25, 2026 · Last updated Aug 11, 2026 · Updated 3 times

Summary

This study tests a gene therapy called delandistrogene moxeparvovec in 13 children under age 4 with Duchenne muscular dystrophy. The goal is to see if it is safe and can help produce a key muscle protein. The children will be followed for about 5 years.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: delandistrogene moxeparvovec (gene therapy)
What this could lead to: If it works, this could slow muscle damage and delay disability in very young children with Duchenne muscular dystrophy.
What could go wrong: This is a small, early-phase study focused on safety, not proof of benefit. Gene therapy carries risks like immune reactions, and long-term effects are unknown.

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Conditions

The condition(s) this trial relates to.

Duchenne muscular dystrophy

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

DUCHENNE MUSCULAR DYSTROPHY

Contacts and locations

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

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