NCT05606614
A Phase 1/2/3 Study of TSHA-102 Gene Therapy in Females With Rett Syndrome (REVEAL Pivotal Study)
Active, Not Recruiting
PHASE3Ages 6–21InterventionalTreatmentTaysha Gene Therapies, Inc.
~17 participants
Updated 2026-07-07 on ClinicalTrials.gov
What's tested:TSHA-102
At a glance
Recruiting sites
0 of 9 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Part A: Safety and Tolerability of TSHA-102
Measured over Baseline through Week 52
+1 more outcome measured
Conditions
Where it's being run
9 sites across 7 statesCalifornia3
Illinois1
Maryland1
Massachusetts1
Missouri1
Texas1
Quebec1
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?
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Eligibility criteria
Inclusion
Females between the ages of 12 and \<22 in Part A (closed) and females between the ages of 6 and \<22 in Part B (pivotal cohort).
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
- Part A: Safety and Tolerability of TSHA-102Baseline through Week 52
Proportions of participants experiencing any treatment-emergent adverse events (AEs) and serious adverse events (SAEs)
- Part B: Efficacy of TSHA-102Baseline through Week 52
Change from baseline in percentage of participants who gain or regain any one or more of the 28 items from the Developmental Milestones Assessment (DMA), which are video recorded and scored by independent, blinded central raters.