Safety and Efficacy of TSHA-102 in Pediatric Females with Rett Syndrome (REVEAL Pediatric Study)

NCTID	NCT06152237 (View at clinicaltrials.gov)
Description	The REVEAL Pediatric Study is a multi-center, Phase 1/2 open-label, dose-escalation and dose-expansion study of TSHA-102, an investigational gene therapy, in pediatric females with Rett Syndrome. The safety, tolerability, and preliminary efficacy of two dose levels will be evaluated. The study duration is up to 6 years. (Show More)
Development Status	Active
Indication	Rett Syndrome
Disease Ontology Term	DOID:1206
Compound Name	TSHA-102
Sponsor	Taysha Gene Therapies, Inc.
Funder Type	Industry
Recruitment Status	Recruiting
Enrollment Count	20
Results Posted	Not Available

Target Gene/Variant	MiniMECP2
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Intrathecal
Drug Product Type	Viral vector
Target Tissue/Cell
Delivery System	Viral transduction
Vector Type	scAAV9
Editor Type	none
Dose 1	5.7E14 vg (n=4)
Dose 2	1E15 vg (n=6)
Dose 3
Dose 4
Dose 5

No.of Trial Sites	10
Locations	Canada,United States,United Kingdom

Has US IND	True
FDA Designations	Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy
Recent Updates	Update on pivotal trial design expected in 1H2025