ASPIRE Study: A Multicenter, Open-Label Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of a Single Intrathecal Administration of TSHA-102, an AAV9-Delivered Gene Therapy, for the Treatment of Pediatric Females Aged >2 to <4 Years With Rett Syndrome
Status: Recruiting
Location: See location...
Intervention Type: Genetic
Study Type: Interventional
Study Phase: Phase 3
SUMMARY
The primary objectives of this study are to evaluate the safety, tolerability and preliminary efficacy of a single intrathecal (IT) dose of TSHA-102 in pediatric females with typical Rett syndrome.
Eligibility
Participation Requirements
Sex: Female
Minimum Age: 2
Maximum Age: 3
Healthy Volunteers: f
View:
• Pediatric females between the ages of 2 and less than 4 years old.
• 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
Massachusetts
Boston Children's Hospital
RECRUITING
Boston
Contact Information
Primary
Taysha Gene Therapies Medical Information
medinfo@tayshagtx.com
833-489-8742
Time Frame
Start Date: 2026-05-08
Estimated Completion Date: 2031-06
Participants
Target number of participants: 3
Treatments
Experimental: Treatment
Participants receive a single intrathecal (IT) administration of TSHA-102 at 1.0 × 10¹⁵ total vector genomes (vg) adjusted for the participant's brain volume.
Related Therapeutic Areas
Sponsors
Leads: Taysha Gene Therapies, Inc.