Brain surgery delivers gene therapy to children with rare neurological disorder
NCT ID NCT04903288
Summary
This study is testing the safety of a new surgical tool and a one-time gene therapy for children with Aromatic L-amino Acid Decarboxylase (AADC) deficiency, a rare genetic disorder that severely affects brain development and movement. The therapy, called eladocagene exuparvovec, is delivered directly into the brain during surgery to try to correct the underlying genetic cause. Researchers will monitor 13 children for safety and check if the treatment improves their motor skills and brain chemistry over several years.
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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
Conditions
Explore the condition pages connected to this study.