Gene therapy trial targets duchenne in toddlers
NCT ID NCT06128564
First seen Nov 01, 2025 · Last updated Jun 19, 2026 · Updated 34 times
Summary
This study tests a gene therapy called delandistrogene moxeparvovec in 13 children under 4 years old with Duchenne muscular dystrophy. The goal is to see if it is safe and helps their muscles produce dystrophin, a protein they lack. Participants receive a single IV infusion and are followed for about 5 years.
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This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Contacts and locations
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Locations
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Chr de La Citadelle
Liège, 3500, Belgium
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Great Ormond Street Hospital for Children
London, WC1N 3JH, United Kingdom
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Hospital Sant Joan De Deu
Esplugues de Llobregas, Barcelona, 08950, Spain
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Hôpital Necker-Enfants Malades
Paris, 75015, France
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John Radcliffe Hospital
Oxford, OX3 9DU, United Kingdom
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PU A. Gemelli, Università Cattolica del Sacro Cuore
Rome, Lazio, 00168, Italy
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Universitätsklinikum Essen
Essen, 45147, Germany
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 successful, this could provide a treatment that helps young children with Duchenne muscular dystrophy produce a key muscle protein, potentially slowing disease progression.
What could go wrong
This is an early phase 2 trial with only 13 participants, so results may not apply to all. Gene therapy carries risks like immune reactions, and long-term effects are unknown.
Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.