One-Shot gene therapy trial offers hope for devastating childhood disorder
NCT ID NCT06152237
Summary
This study is testing a single injection of an experimental gene therapy called TSHA-102 for girls aged 5 to 8 with Rett syndrome. The main goal is to see if the treatment is safe and well-tolerated at two different dose levels. Researchers will also look for early signs that it might help improve symptoms like movement, behavior, and seizures over a follow-up period of up to six years.
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Contacts and locations
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Locations
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CHU Ste-Justine
Montreal, Quebec, H3T 1C5, Canada
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Children's Neurosciences, Evelina London Children's Hospital, Guy's and St Thomas' NHS Foundation Trust
London, United Kingdom
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Gillette Children's Specialty Healthcare
Saint Paul, Minnesota, 55101, United States
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Rush University Medical Center & Children's Hospital
Chicago, Illinois, 60612, United States
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Washington University, St. Louis
St Louis, Missouri, 63110, United States
Conditions
Explore the condition pages connected to this study.