Actively Recruiting
A Phase 1/2/3 Study of TSHA-102 Gene Therapy in Females With Rett Syndrome (REVEAL Pivotal Study)
Led by Taysha Gene Therapies, Inc. · Updated on 2025-12-30
15
Participants Needed
6
Research Sites
429 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
The primary objectives of this study are to evaluate the safety of a single intrathecal (IT) dose of TSHA-102 in females with typical Rett syndrome, to select the TSHA-102 dose with the best benefit/risk profile based on the totality of safety and efficacy data and to evaluate the efficacy and safety of TSHA-102 at the selected dose.
CONDITIONS
Official Title
A Phase 1/2/3 Study of TSHA-102 Gene Therapy in Females With Rett Syndrome (REVEAL Pivotal Study)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Female participants aged 12 to under 22 years in Part A and 6 to under 22 years in Part B
- Clinical diagnosis of typical Rett syndrome with a pathogenic MECP2 gene mutation causing loss of function
- Willingness to receive blood or blood products if medically needed for adverse events
- Participant and caregiver agree to live near the study site before baseline and for at least 3 months after treatment
You will not qualify if you...
- Diagnosis of another neurodevelopmental disorder unrelated to MECP2 mutation or progressive genetic syndrome
- History of brain injury causing neurological problems or abnormal psychomotor development within first 6 months of life
- Diagnosis of atypical Rett syndrome or MECP2 mutation not causing Rett syndrome
- Requirement for invasive ventilatory support
- Other protocol-defined exclusion criteria may also apply
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 6 locations
1
UC San Diego
La Jolla, California, United States, 92093
Actively Recruiting
2
Rush University Medical Center
Chicago, Illinois, United States, 60612
Actively Recruiting
3
Boston Children's Hospital
Boston, Massachusetts, United States, 02115
Actively Recruiting
4
Washington University, St. Louis
St Louis, Missouri, United States, 63110
Actively Recruiting
5
UT Southwestern Children's Medical Center
Dallas, Texas, United States, 75930
Actively Recruiting
6
CHU St. Justine
Montreal, Quebec, Canada
Actively Recruiting
Research Team
T
Taysha Gene Therapies Medical Information
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
SINGLE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
3
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