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

Running, not enrolling · Phase 3

Conditions studied: Rett Syndrome

In brief

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.

Key facts

Study ID

NCT05606614

Run by

Taysha Gene Therapies, Inc.

People needed

17

Starts

2023-03-06

Expected to finish

2031-06-01

Last updated by the study team

2026-07-07

Who can join

Age: 6 and older, up to 21. Sex: female. Healthy volunteers: not accepted.

You may qualify if…

• Females between the ages of 12 and <22 in Part A (closed) and females between the ages of 6 and <22 in Part B (pivotal cohort).
• Participant has a clinical diagnosis of classic/typical Rett syndrome with a documented pathogenic mutation of the methyl-CpG-binding protein 2 (MECP2) gene that results in loss of gene function.
• Participants must be willing to receive blood or blood products for the treatment of an AE if medically needed.
• Participants and parent/caregiver must agree to reside within easy access to the study site prior to the baseline visit and at least 3 months after TSHA-102 treatment

You may not qualify if…

• 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 or had grossly abnormal psychomotor development in the first 6 months of life.
• Participant has a diagnosis of atypical Rett syndrome or a MECP2 gene mutation that does not cause Rett syndrome.
• Participant requires invasive ventilatory support.
• Note: Other protocol defined inclusion/exclusion criteria may apply

Where it is running

• City of Hope — Duarte, California, United States
• UC San Diego — La Jolla, California, United States
• UC San Francisco Benioff Children's Hospital — Oakland, California, United States
• Rush University Medical Center — Chicago, Illinois, United States
• Kennedy Krieger Institute — Baltimore, Maryland, United States
• Boston Children's Hospital — Boston, Massachusetts, United States
• Washington University, St. Louis — St Louis, Missouri, United States
• UT Southwestern Children's Medical Center — Dallas, Texas, United States
• CHU St. Justine — Montreal, Quebec, Canada

Full record on ClinicalTrials.gov

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