Back to search results

TSHA-102 Gene Therapy for Rett Syndrome

This study is testing TSHA-102, a gene therapy, in young girls with Rett syndrome. TSHA-102 is given one time into the spinal fluid (intrathecal administration) and aims to address the genetic cause of Rett syndrome by helping cells properly use the MECP2 gene. Researchers want to see how safe TSHA-102 is and if it shows early signs of helping with Rett syndrome symptoms. You may be able to join if you are a girl between 2 and 4 years old with a confirmed diagnosis of classic Rett syndrome and a specific MECP2 gene mutation. The study is currently recruiting a small number of participants to understand these early effects.

Study design
This is an open-label study, meaning everyone knows what treatment is being given. It will include 3 pediatric females with typical Rett syndrome.
What's involved
Not specified in the trial record.
Compensation
Not stated in the trial record.
Follow-up
Each participant will be followed for 5 years after receiving TSHA-102.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT07480564

Safety and Preliminary Efficacy of TSHA-102 Gene Therapy in Pediatric Females Aged >2 to <4 Years With Rett Syndrome

Active, Not Recruiting
PHASE3Ages 2–3InterventionalTreatment
Taysha Gene Therapies, Inc.
~4 participants
Updated 2026-08-19 on ClinicalTrials.gov
What's tested:TSHA-102

At a glance

Recruiting sites
0 of 3 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Primary Safety
Measured over Baseline through Week 25

Conditions

Rett Syndrome

Explore related trials

Rett SyndromeMECP2 Gene

Where it's being run

3 sites across 3 states
Alabama1
Illinois1
Massachusetts1

Study leadership

  • Medical Monitor, M.D. · STUDY_DIRECTOR · Taysha Gene Therapies

Who to contact

This trial hasn't published a contact. View it on ClinicalTrials.gov

Do you actually qualify for this trial?

Add a private profile and we'll compare every criterion below against your situation — and tell you which ones are met, uncertain, or excluding.

Check eligibility for this trial ~2 min · HIPAA-protected · delete anytime
Eligibility criteria

Inclusion

Pediatric females between the ages of 2 and less than 4 years old.
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.

Exclusion

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.

What this trial measures

  • Primary SafetyBaseline through Week 25

    Safety and Tolerability of TSHA-102 Proportions of participants experiencing any treatment-emergent adverse events (AEs) and serious adverse events (SAEs)