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

Launched by TAYSHA GENE THERAPIES, INC. · Nov 21, 2023

Keywords

ClinConnect Summary

The REVEAL Pediatric Study is a clinical trial that is exploring the safety and effectiveness of a new gene therapy called TSHA-102 for young girls with Rett Syndrome, a rare neurological disorder. This study is specifically looking at girls aged 5 to 8 years who have a confirmed diagnosis of typical Rett Syndrome caused by a mutation in the MECP2 gene. Researchers want to understand how well the therapy works and how safe it is for these children over a period of up to six years.

To participate, the girls must have the right type of Rett Syndrome and meet certain health criteria. For example, they should not have other neurological disorders or serious health issues that might interfere with the study. If eligible, participants will receive the treatment and will be closely monitored for any side effects or changes in their condition. This trial offers a chance to contribute to important research that might help improve the lives of children with Rett Syndrome.

Gender

FEMALE

Eligibility criteria

• Inclusion 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.
• Exclusion Criteria:
• • 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.