NCT05898620API RE2T
A Novel, Regulated Gene Therapy (NGN-401) Study for Females With Rett Syndrome
This study evaluates the efficacy and safety of the investigational gene therapy NGN-401 in females with typical Rett syndrome.
Ongoing · not recruitingINTERVENTIONAL
Record summary
This study evaluates the efficacy and safety of the investigational gene therapy NGN-401 in females with typical Rett syndrome.
What this record cannot establish
Registry data requiring human review; it cannot determine eligibility or individual benefit.
Study level
Documented countryAU · GB · US
Centre level16 Sites named in the sources
Original sourceClinicalTrials.gov
Human reviewHuman-reviewed
Centre level
Centre level · 16 Sites named in the sources · AU · GB · US
University of Alabama at Birmingham · Birmingham · USSite opening to confirm
UCSF Benioff Children's Hospital Oakland · Oakland · USSite opening to confirm
Children's Hospital Colorado · Aurora · USSite opening to confirm
Nicklaus Children's Hospital Research Institute · Miami · USSite opening to confirm
Rush University Medical Center · Chicago · USSite opening to confirm
Kennedy Krieger Institute · Baltimore · USSite opening to confirm
Boston Children's Hospital · Boston · USSite opening to confirm
Montefiore Medical Center · New York · USSite opening to confirm
UNC at Chapel Hill · Chapel Hill · USSite opening to confirm
Nationwide Children's Hospital · Columbus · USSite opening to confirm
Children's Hospital of Philadelphia · Philadelphia · USSite opening to confirm
Vanderbilt University Medical Center · Nashville · USSite opening to confirm
Texas Children's Hospital · Houston · USSite opening to confirm
The Children's Hospital at Westmead · Sydney · AUSite opening to confirm
Royal Hospital for Children and Young People · Edinburgh · GBSite opening to confirm
Manchester University NHS Foundation Trust · Manchester · GBSite opening to confirm
Source and review
Résumé ou traduction assisté par IA : peut contenir des erreurs et ne remplace ni une relecture humaine ni un avis médical.
Source updated · Jun 10, 2026
Source updated · Jun 9, 2026
Synced · Sep 19, 2026
Published · Sep 19, 2026
Human review · Sep 19, 2026