TSHA-102 for Rett Syndrome

Recruiting in Palo Alto (17 mi)

+11 other locations

Age: < 18

Sex: Female

Travel: May be covered

Time Reimbursement: Varies

Trial Phase: Phase 1 & 2

Recruiting

Sponsor: Taysha Gene Therapies, Inc.

No Placebo Group

Approved in 3 jurisdictions

Trial Summary

What is the purpose of this trial?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.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

Eligibility Criteria

This trial is for young girls aged 5 to 8 with Rett Syndrome, a neurological disorder. They must have a specific gene mutation (MECP2) and be up-to-date on vaccinations. Their caregiver must agree to potential blood product treatments for side effects.

Inclusion Criteria

I have Rett Syndrome with a confirmed MECP2 gene mutation.

I am between 5 and 8 years old.

Participant Groups

The REVEAL Pediatric Study tests TSHA-102, a gene therapy, in two different doses to see how safe it is and if it works for treating Rett Syndrome in pediatric females over the course of up to six years.

2Treatment groups

Experimental Treatment

Group I: Cohort 2Experimental Treatment1 Intervention

Dose Level 2

Group II: Cohort 1Experimental Treatment1 Intervention

Dose Level 1

TSHA-102 is already approved in United States, Canada, United Kingdom for the following indications:

🇺🇸 Approved in United States as TSHA-102 for: