Safety and Efficacy of TSHA-102 in Pediatric Females with Rett Syndrome
Research type
Research Study
Full title
REVEAL Pediatric Study: A Multicenter, Open-Label, Randomized, Dose-Escalation and Dose-Expansion Study of the Safety, Tolerability, and Efficacy of a Single Intrathecal Administration of TSHA-102, an AAV9-Delivered Gene Therapy, for the Treatment of Pediatric Females with Rett Syndrome
IRAS ID
1007616
Contact name
Rumana Haque-Ahmed
Contact email
Sponsor organisation
Taysha Gene Therapies, Inc.
Clinicaltrials.gov Identifier
Research summary
This trial is to learn if an investigational gene therapy, TSHA-102, can help treat females ≥3 to ≤8 years with Rett Syndrome. Rett syndrome is a disabling genetic condition affecting 1:10,000 females, for which there are no treatments or cures. This trial aims to deliver new, working copies of the MECP2 gene to the cells that need it. TSHA-102 is a liquid that contains new, working copies of the MECP2 gene. In this study, a modified virus that cannot cause infection, called adeno-associated virus type 9 (AAV9), will be used as the vector. AAV9 can cross the blood-brain barrier, which is like a strict border that keeps blood vessels separate from the spinal cord and brain, allowing only certain things to pass through. TSHA-102 will be given as a one-time injection into the spinal fluid in the lower back. A thin needle will be used to inject TSHA-102 between 2 bones of the spine while the participant is under general anesthesia. The purpose of the study is to investigate the safety, possible harms and side effects of the gene transfer, and to see if there are any beneficial effects. The study will be open-label. There will be up to 27 participants enrolled from multiple centers worldwide. The study will test two different doses of TSHA-102 to help find the best dose that is most safe and effective. Part A: Six participants (at most twelve) will be randomized to receive the one-time gene therapy directly after the screening period. Part B: another six participants (at most nine) will be randomly assigned to receive the one-time gene therapy at a later time. Another six will be assigned to the Delayed Treatment Cohort. Each participant enrolled in the trial will receive the gene therapy (a one-time injection of TSHA-102) either immediately after eligibility requirements are met or after a delayed treatment phase. Participants will be monitored regularly for up to six years after receiving the gene therapy to check the effect of TSHA-102 on health and symptoms.
REC name
South Central - Oxford A Research Ethics Committee
REC reference
24/SC/0014
Date of REC Opinion
22 Dec 2023
REC opinion
Favourable Opinion