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:
- Rett syndrome (investigational)
🇨🇦 Approved in Canada as TSHA-102 for:
- Rett syndrome (investigational)
🇬🇧 Approved in United Kingdom as TSHA-102 for:
- Rett syndrome (investigational)
Find A Clinic Near You
Research locations nearbySelect from list below to view details:
Rush University Medical Center & Children's HospitalChicago, IL
Gillette Children's Specialty HealthcareSt. Paul, MN
CHU Ste-JustineMontréal, Canada
Rush University Medical CenterChicago, IL
More Trial Locations
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Who is running the clinical trial?
Taysha Gene Therapies, Inc.Lead Sponsor