An Open-label Phase 1/2/3 Study Consisting of a Phase 1/2 Safety and Dose-escalation and Phase 3 Dose-expansion Study to Evaluate Safety and Efficacy of a Single Intrathecal Administration of TSHA-102, an AAV9-Delivered Gene Therapy in Females With Rett Syndrome
Status: Recruiting
Location: See all (6) locations...
Intervention Type: Genetic
Study Type: Interventional
Study Phase: Phase 3
SUMMARY
The primary objectives of this study are to evaluate the safety of a single intrathecal (IT) dose of TSHA-102 in females with typical Rett syndrome, to select the TSHA-102 dose with the best benefit/risk profile based on the totality of safety and efficacy data and to evaluate the efficacy and safety of TSHA-102 at the selected dose.
Eligibility
Participation Requirements
Sex: Female
Minimum Age: 6
Maximum Age: 21
Healthy Volunteers: f
View:
• Females between the ages of 12 and \<22 in Part A (closed) and females between the ages of 6 and \<22 in Part B (pivotal cohort).
• Participant has a clinical diagnosis of classic/typical Rett syndrome with a documented pathogenic mutation of the methyl-CpG-binding protein 2 (MECP2) gene that results in loss of gene function.
• Participants must be willing to receive blood or blood products for the treatment of an AE if medically needed.
• Participants and parent/caregiver must agree to reside within easy access to the study site prior to the baseline visit and at least 3 months after TSHA-102 treatment
Locations
United States
California
UC San Diego
RECRUITING
La Jolla
Illinois
Rush University Medical Center
RECRUITING
Chicago
Massachusetts
Boston Children's Hospital
RECRUITING
Boston
Missouri
Washington University, St. Louis
RECRUITING
St Louis
Texas
UT Southwestern Children's Medical Center
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:2031-06
Participants
Target number of participants:15
Treatments
Experimental: Part A Cohort 1
TSHA-102 Dose Level 1: 5.7×10¹⁴ total vector genomes (vg). Participants receive a single intrathecal (IT) administration of TSHA-102 at Dose Level 1 (fully enrolled, 2 participants).
Experimental: Part A Cohort 2
TSHA-102 Dose Level 2: 1.0×10¹⁵ total vector genomes (vg)~Participants receive a single intrathecal (IT) administration of TSHA-102 at Dose Level 2 (fully enrolled, 4 participants).
Experimental: Part B Pivotal Cohort
TSHA-102 at Selected Dose (Dose Level 2): 1.0 × 10¹⁵ total vector genomes (vg)~Participants receive a single intrathecal (IT) administration of TSHA-102 at Dose Level 2 (1.0 × 10¹⁵).