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

Description

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

Rett Syndrome

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Study Overview

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Study Details

Study overview

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.

A Multicenter, Open Label, Randomized, Dose-Escalation and Dose-Expansion Study of the Safety, Tolerability, and Efficacy of a Single Intrathecal Administration of TSHA-102, an AAV9-Delivered Gene Therapy, for the Treatment of Pediatric Females with Rett Syndrome

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

Condition
Rett Syndrome
Intervention / Treatment

-

Contacts and Locations

La Jolla

University of California San Diego (UCSD), La Jolla, California, United States, 92037

Chicago

Rush University Medical Center & Children's Hospital, Chicago, Illinois, United States, 60612

Boston

Boston Children's at Brookline, Boston, Massachusetts, United States, 02445

St. Paul

Gillette Children's Specialty Healthcare, St. Paul, Minnesota, United States, 55101

Saint Louis

Washington University, St. Louis, Saint Louis, Missouri, United States, 63110

Philadelphia

Children's Hospital of Philadelphia Research Institute, Philadelphia, Pennsylvania, United States, 19104

Nashville

Vanderbilt Kennedy Center, Nashville, Tennessee, United States, 37203

Dallas

University of Texas Southwestern Medical Center (UTSW), Dallas, Texas, United States, 75390

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility Criteria

  • * Participant has a confirmed diagnosis of classical/typical Rett Syndrome with a documented mutation of the MECP2 gene that results in loss of function.
  • * Participant is between ≥5 to ≤8 years of age at the time of consent.
  • * Participant must be up to date with all relevant local vaccination requirements, with last vaccination dose received at least 42 days prior to the start of the immunosuppression regimen.
  • * Participant's parent/caregiver must be willing to allow participant to receive blood or blood products for the treatment of an AE if medically needed.
  • * Participant has another neurodevelopmental disorder independent of the MECP2 gene loss of function mutation, or any other genetic syndrome with a progressive course.
  • * Participant has a history of brain injury that causes neurological problems.
  • * Participant had grossly abnormal psychomotor development in the first 6 months of life.
  • * Participant has a diagnosis of atypical Rett syndrome.
  • * Participant has an MECP2 mutation that does not cause Rett syndrome.
  • * Participant requires non-invasive and invasive ventilatory support.
  • * Participant has contraindications for IT administration of TSHA-102 or lumbar puncture procedure, other medical conditions, or contraindications to any medications required for IT administration.
  • * Participant has acute or chronic hepatitis B or C infections.

Ages Eligible for Study

5 Years to 8 Years

Sexes Eligible for Study

FEMALE

Accepts Healthy Volunteers

No

Collaborators and Investigators

Taysha Gene Therapies, Inc.,

Meredith Schultz, M.D., STUDY_DIRECTOR, Taysha Gene Therapies

Study Record Dates

2031-11-02