Clinical Trial: Safety and Preliminary Efficacy of TSHA-102 Gene Therapy in Pediatric Females Aged >2 to <4 Years With Rett Syndrome

U.S., March 19 -- ClinicalTrials.gov registry received information related to the study (NCT07480564) titled 'Safety and Preliminary Efficacy of TSHA-102 Gene Therapy in Pediatric Females Aged >2 to <4 Years With Rett Syndrome' on March 13.

Brief 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.

Study Start Date: March, 2026

Study Type: INTERVENTIONAL

Condition: Rett Syndrome

Intervention: GENETIC: TSHA-102

TSHA-102 is a recombinant, non-replicating, self-complementary adeno-associated virus serotype 9 (scAAV9) vector encoding for the miniMECP2 gene. TSHA-102 is a one-time intra...