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

NCT07480564 · Status: RECRUITING · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 3

Last updated 2026-05-22

No results posted yet for this study

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.

Conditions

Interventions

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 intrathecal (IT) administration.

Sponsors & Collaborators

  • Taysha Gene Therapies, Inc.

    lead INDUSTRY

Principal Investigators

  • Medical Monitor, M.D. · Taysha Gene Therapies

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
2 Years
Max Age
3 Years
Sex
FEMALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2026-05-08
Primary Completion
2031-06-30
Completion
2031-06-30
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07480564 on ClinicalTrials.gov