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

Description

The REVEAL Adult Study is a multi-center, Phase 1/2 open-label, dose-escalation study of TSHA-102, an investigational gene therapy, in adult females with Rett syndrome. The safety, tolerability, and preliminary efficacy of two dose levels will be evaluated. The study duration is estimated to be up to 63 months.

Conditions

Rett Syndrome

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Study Overview

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Study Details

Study overview

The REVEAL Adult Study is a multi-center, Phase 1/2 open-label, dose-escalation study of TSHA-102, an investigational gene therapy, in adult females with Rett syndrome. The safety, tolerability, and preliminary efficacy of two dose levels will be evaluated. The study duration is estimated to be up to 63 months.

REVEAL Adult Study: an Open-label, Randomized, Doseescalation- and Dose-expansion Study of the Safety and Preliminary Efficacy of a Single Intrathecal Administration of TSHA-102, an AAV9-Delivered Gene Therapy, in the Treatment of Adult Females with Rett Syndrome

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

Condition
Rett Syndrome
Intervention / Treatment

-

Contacts and Locations

La Jolla

UC San Diego, La Jolla, California, United States, 92093

Chicago

Rush University Medical Center, Chicago, Illinois, United States, 60612

Boston

Boston Children's Hospital, Boston, Massachusetts, United States, 02115

St. Paul

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

Dallas

UT Southwestern Medical Center, Dallas, Texas, United States, 75930

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 clinical diagnosis of classical/typical Rett syndrome with a documented pathogenic mutation of the methyl-CpG-binding protein 2 (MECP2) gene that results in loss of function.
  • * Participants must be willing to receive blood or blood products for the treatment of an AE if medically needed.
  • * Participant has another neurodevelopmental disorder independent of the MECP2 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 a MECP2 mutation that does not cause Rett syndrome.
  • * Participant requires invasive ventilatory support.
  • * Participant has contraindications for IT administration of TSHA-102 or lumbar puncture procedure, or other medical conditions, or contraindications to any medications required for IT administration.
  • * Participant has uncontrolled seizures or a history of status epilepticus within the 3 months prior to enrollment.

Ages Eligible for Study

12 Years to

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

2032-01-05