RecruitingPhase 3NCT07480564

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

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

Sponsor

Taysha Gene Therapies, Inc.

Enrollment

3 participants

Start Date

May 8, 2026

Study Type

INTERVENTIONAL

Conditions

Rett Syndrome

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

Sex: FEMALEMin Age: 2 YearsMax Age: 3 Years

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying TSHA-102 for people with rett syndrome. The study is currently recruiting participants at 1 location. People eligible for this study include women aged 2 Years to 3 Years.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

GENETICTSHA-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 intrathecal (IT) administration.