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

NCT05606614 · Status: RECRUITING · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 15

Last updated 2025-12-30

No results posted yet for this study

Summary

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.

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

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

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
SINGLE
Model
SEQUENTIAL

Eligibility

Min Age
6 Years
Max Age
21 Years
Sex
FEMALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2023-03-06
Primary Completion
2031-06-30
Completion
2031-06-30
FDA Drug
Yes

Countries

  • United States
  • Canada

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 NCT05606614 on ClinicalTrials.gov