Brain gene therapy delivery system tested in kids with rare disorder
NCT ID NCT04903288
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tests a special needle (SmartFlow cannula) designed to safely deliver a gene therapy drug (eladocagene exuparvovec) into the brains of 13 children with AADC deficiency, a rare genetic disorder that affects movement and development. The main goals are to check if the procedure is safe and if it raises dopamine levels in the brain. The study includes short-term and long-term follow-up to see if the treatment improves motor skills and other symptoms.
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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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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.
Contacts and locations
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Locations
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Boston Children's Hospital
Boston, Massachusetts, 02115, United States
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Chaim Sheba Medical Center
Ramat Gan, 5262000, Israel
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Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229, United States
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Duke University Hospital
Durham, North Carolina, 27705, United States
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National Taiwan University Hospital, Department of Pediatrics and Medical Genetics
Taipei, 10041, Taiwan
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Texas Children's Hospital
Houston, Texas, 77030, United States
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