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

NCT06152237 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 6

Last updated 2025-10-15

No results posted yet for this study

Summary

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.

Conditions

Interventions

GENETIC

TSHA-102

TSHA-102 is a recombinant, non-replicating, self-complementary AAV9 (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

  • Laura Pisani, M.D. · Taysha Gene Therapies

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
5 Years
Max Age
8 Years
Sex
FEMALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2023-12-12
Primary Completion
2028-11-02
Completion
2031-11-02
FDA Drug
Yes

Countries

  • United States
  • Canada
  • United Kingdom

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 NCT06152237 on ClinicalTrials.gov