A Phase 1/2/3 Study of TSHA-102 Gene Therapy in Females With Rett Syndrome (REVEAL Pivotal Study)

NCTID	NCT05606614 (View at clinicaltrials.gov)
Description	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. (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 (Click here for study contact information)
Enrollment Count	15 (ESTIMATED)
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
Dose 2	1E15 vg
Dose 3
Dose 4
Dose 5

No.of Trial Sites	5
Locations	Canada,United States

Has US IND	True
FDA Designations	Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy
Recent Updates	Clinical data expected H1 2025