Gene therapy hope for girls with rare brain disorder
NCT ID NCT06152237
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tests a new gene therapy called TSHA-102 for girls aged 5 to 8 with Rett syndrome, a severe genetic disorder that affects brain development. The therapy is given as a single injection into the spinal fluid. The main goal is to check safety and side effects, while also looking for signs of improvement. The study involves 6 participants and will follow them for up to 6 years.
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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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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
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