REVEAL Adult Study: An Open-label, Randomized, Doseescalation- and Dose-expansion Study of the Safety and Preliminary Efficacy of a Single Intrathecal Administration of TSHA-102, an AAV9-Delivered Gene Therapy, in the Treatment of Adult Females With Rett Syndrome
Status: Recruiting
Location: See all (6) locations...
Intervention Type: Genetic
Study Type: Interventional
Study Phase: Phase 1/Phase 2
SUMMARY
The REVEAL Adult Study is a multi-center, Phase 1/2 open-label, dose-escalation study of TSHA-102, an investigational gene therapy, in adult females with Rett syndrome. The safety, tolerability, and preliminary efficacy of two dose levels will be evaluated. The study duration is estimated to be up to 63 months.
Eligibility
Participation Requirements
Sex: Female
Minimum Age: 12
Healthy Volunteers: f
View:
• Participant has a clinical diagnosis of classical/typical Rett syndrome with a documented pathogenic mutation of the methyl-CpG-binding protein 2 (MECP2) gene that results in loss of function.
• Participants must be willing to receive blood or blood products for the treatment of an AE if medically needed.
Locations
United States
California
UC San Diego
RECRUITING
La Jolla
Illinois
Rush University Medical Center
RECRUITING
Chicago
Massachusetts
Boston Children's Hospital
NOT_YET_RECRUITING
Boston
Minnesota
Gillette Children's Hospital
NOT_YET_RECRUITING
Saint Paul
Texas
UT Southwestern Medical Center
NOT_YET_RECRUITING
Dallas
Other Locations
Canada
CHU St. Justine
RECRUITING
Montreal
Contact Information
Primary
Taysha Gene Therapies Medical Information
medinfo@tayshagtx.com
833-489-8742
Time Frame
Start Date: 2023-03-06
Estimated Completion Date: 2032-01-05
Participants
Target number of participants: 18
Treatments
Experimental: Cohort 1
Dose Level 1
Experimental: Cohort 2
Dose Level 2
Related Therapeutic Areas
Sponsors
Leads: Taysha Gene Therapies, Inc.