New gene therapy hopes to restore milestones in rett syndrome
NCT ID NCT05606614
First seen Jun 27, 2026 · Last updated Jul 08, 2026 · Updated 2 times
Summary
This study tests a one-time gene therapy called TSHA-102 in females aged 6 to under 22 with classic Rett syndrome. The goal is to see if it is safe and can help them regain or gain new developmental skills like walking or talking. Participants receive the therapy through a spinal injection and are monitored for side effects and improvements.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for RETT SYNDROME are added.
By submitting, you agree to our Terms of use
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
Locations
-
Boston Children's Hospital
Boston, Massachusetts, 02115, United States
-
CHU St. Justine
Montreal, Quebec, Canada
-
City of Hope
Duarte, California, 91010, United States
-
Kennedy Krieger Institute
Baltimore, Maryland, 21205, United States
-
Rush University Medical Center
Chicago, Illinois, 60612, United States
-
UC San Diego
La Jolla, California, 92093, United States
-
UC San Francisco Benioff Children's Hospital
Oakland, California, 94609, United States
-
UT Southwestern Children's Medical Center
Dallas, Texas, 75930, United States
-
Washington University, St. Louis
St Louis, Missouri, 63110, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- A private facebook group may ease the emotional burden of caring for a child with rett syndrome
- Antioxidant cocktail may ease motor deficits in rett syndrome — a trial investigates
- Can robot legs help Non-Walking kids join in at school?
- Can early parent coaching help infants with rare genetic disorders thrive?
- Newborn screening study aims to catch rare diseases at birth
- Massive global registry aims to unlock secrets of rett syndrome