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Gene Therapy

TSHA-102 for Rett Syndrome

Phase 1 & 2
Recruiting
Research Sponsored by Taysha Gene Therapies, Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Participant has a confirmed diagnosis of classical/typical Rett Syndrome with a documented mutation of the MECP2 gene that results in loss of function.
Participant is between ≥5 to ≤8 years of age at the time of consent.
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline through week 52
Awards & highlights
No Placebo-Only Group

Summary

This trial is testing a new gene therapy called TSHA-102 in girls with Rett Syndrome. It will evaluate the safety and effectiveness of two different doses of the therapy. The study will last for

Who is the study for?
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.
What is being tested?
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.
What are the potential side effects?
Possible side effects are not detailed here but typically include reactions related to gene therapy such as immune response, injection site reactions, or complications from long-term immunosuppression.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
I have Rett Syndrome with a confirmed MECP2 gene mutation.
Select...
I am between 5 and 8 years old.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline through week 52
This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline through week 52 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Primary Safety
Secondary study objectives
Exploratory Efficacy

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

2Treatment groups
Experimental Treatment
Group I: Cohort 2Experimental Treatment1 Intervention
Dose Level 2
Group II: Cohort 1Experimental Treatment1 Intervention
Dose Level 1

Find a Location

Who is running the clinical trial?

Taysha Gene Therapies, Inc.Lead Sponsor
4 Previous Clinical Trials
42 Total Patients Enrolled
1 Trials studying Rett Syndrome
18 Patients Enrolled for Rett Syndrome
Meredith Schultz, M.D.Study DirectorTaysha Gene Therapies
1 Previous Clinical Trials
18 Total Patients Enrolled
1 Trials studying Rett Syndrome
18 Patients Enrolled for Rett Syndrome
Benit Maru, Bsc, MB ChB, MSc, PhDStudy DirectorTaysha Gene Therapies
~13 spots leftby Nov 2028
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