TSHA-102 Gene Therapy for Rett Syndrome
(ASPIRE Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
The primary objectives of this study are to evaluate the safety, tolerability and preliminary efficacy of a single intrathecal (IT) dose of TSHA-102 in pediatric females with typical Rett syndrome.
Who Is on the Research Team?
Medical Monitor, M.D.
Principal Investigator
Taysha Gene Therapies
Are You a Good Fit for This Trial?
This trial is for girls aged 2 to under 4 years old who have been diagnosed with classic Rett syndrome caused by a specific gene mutation. Participants must be able to stay near the study site and agree to receive blood products if needed.Inclusion Criteria
What Are the Treatments Tested in This Trial?
Interventions
- TSHA-102
Trial Overview
The study is testing a single dose of TSHA-102, a gene therapy given directly into the spinal fluid, to see if it is safe and shows early signs of helping young girls with Rett syndrome.
How Is the Trial Designed?
1
Treatment groups
Experimental Treatment
Participants receive a single intrathecal (IT) administration of TSHA-102 at 1.0 × 10¹⁵ total vector genomes (vg) adjusted for the participant's brain volume.
Find a Clinic Near You
Who Is Running the Clinical Trial?
Taysha Gene Therapies, Inc.
Lead Sponsor
Unbiased Results
We believe in providing patients with all the options.
Your Data Stays Your Data
We only share your information with the clinical trials you're trying to access.
Verified Trials Only
All of our trials are run by licensed doctors, researchers, and healthcare companies.