NCT06152237

Brief Summary

The REVEAL Pediatric Study is a multi-center, Phase 1/2 open-label, dose-escalation and dose-expansion study of TSHA-102, an investigational gene therapy, in pediatric females with Rett Syndrome. The safety, tolerability, and preliminary efficacy of two dose levels will be evaluated. The study duration is up to 6 years.

Trial Health

78
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
6

participants targeted

Target at below P25 for phase_1

Timeline
67mo left

Started Dec 2023

Longer than P75 for phase_1

Geographic Reach
3 countries

5 active sites

Status
active not recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress30%
Dec 2023Nov 2031

First Submitted

Initial submission to the registry

November 21, 2023

Completed
9 days until next milestone

First Posted

Study publicly available on registry

November 30, 2023

Completed
12 days until next milestone

Study Start

First participant enrolled

December 12, 2023

Completed
4.9 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

November 2, 2028

Expected
3 years until next milestone

Study Completion

Last participant's last visit for all outcomes

November 2, 2031

Last Updated

October 15, 2025

Status Verified

October 1, 2025

Enrollment Period

4.9 years

First QC Date

November 21, 2023

Last Update Submit

October 10, 2025

Conditions

Rett Syndrome

Keywords

Rett SyndromeNeurodevelopmental DisorderMECP2

Outcome Measures

Primary Outcomes (1)

  • Primary Safety

    The incidence of participants experiencing any treatment-emergent adverse events (AEs) and serious adverse events (SAEs)

    Baseline through week 52

Secondary Outcomes (6)

  • Exploratory Efficacy

    Baseline through week 52

  • Exploratory Efficacy

    Baseline through week 52

  • Exploratory Efficacy

    Baseline through week 52

  • Exploratory Efficacy

    Baseline through week 52

  • Exploratory Efficacy

    Baseline through week 52

  • +1 more secondary outcomes

Study Arms (2)

Cohort 1

EXPERIMENTAL

Dose Level 1

Genetic: TSHA-102

Cohort 2

EXPERIMENTAL

Dose Level 2

Genetic: TSHA-102

Interventions

TSHA-102GENETIC

TSHA-102 is a recombinant, non-replicating, self-complementary AAV9 (scAAV9) vector encoding for the miniMECP2 gene. TSHA-102 is a one-time intrathecal (IT) administration.

Cohort 1Cohort 2

Eligibility Criteria

Age5 Years - 8 Years
Sexfemale
Healthy VolunteersNo
Age GroupsChild (0-17)

You may qualify if:

  • 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.
  • Participant must be up to date with all relevant local vaccination requirements, with last vaccination dose received at least 42 days prior to the start of the immunosuppression regimen.
  • Participant's parent/caregiver must be willing to allow participant to receive blood or blood products for the treatment of an AE if medically needed.

You may not qualify if:

  • Participant has another neurodevelopmental disorder independent of the MECP2 gene loss of function mutation, or any other genetic syndrome with a progressive course.
  • Participant has a history of brain injury that causes neurological problems.
  • Participant had grossly abnormal psychomotor development in the first 6 months of life.
  • Participant has a diagnosis of atypical Rett syndrome.
  • Participant has an MECP2 mutation that does not cause Rett syndrome.
  • Participant requires non-invasive and invasive ventilatory support.
  • Participant has contraindications for IT administration of TSHA-102 or lumbar puncture procedure, other medical conditions, or contraindications to any medications required for IT administration.
  • Participant has acute or chronic hepatitis B or C infections.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (5)

Rush University Medical Center & Children's Hospital

Chicago, Illinois, 60612, United States

Location

Gillette Children's Specialty Healthcare

Saint Paul, Minnesota, 55101, United States

Location

Washington University, St. Louis

St Louis, Missouri, 63110, United States

Location

CHU Ste-Justine

Montreal, Quebec, H3T 1C5, Canada

Location

Children's Neurosciences, Evelina London Children's Hospital, Guy's and St Thomas' NHS Foundation Trust

London, United Kingdom

Location

MeSH Terms

Conditions

Rett SyndromeNeurodevelopmental Disorders

Condition Hierarchy (Ancestors)

X-Linked Intellectual DisabilityIntellectual DisabilityNeurobehavioral ManifestationsNeurologic ManifestationsNervous System DiseasesGenetic Diseases, X-LinkedGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesHeredodegenerative Disorders, Nervous SystemMental Disorders

Study Officials

  • Laura Pisani, M.D.

    Taysha Gene Therapies

    STUDY DIRECTOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
SEQUENTIAL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

November 21, 2023

First Posted

November 30, 2023

Study Start

December 12, 2023

Primary Completion (Estimated)

November 2, 2028

Study Completion (Estimated)

November 2, 2031

Last Updated

October 15, 2025

Record last verified: 2025-10

Data Sharing

IPD Sharing
Will not share

Locations

US(3)GB(1)CA(1)