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Safety and Efficacy of TSHA-102 in Pediatric Females With Rett Syndrome (REVEAL Pediatric Study)

Sponsored by Taysha Gene Therapies, Inc.

Quick Self-Assessment

See if you qualify and express your interest

Preparing your pre-screening questions...

Current Status

Active, Not Recruiting

Auto-verified from ClinicalTrials.gov

Verified Oct 15, 2025

Phase

Clinical Trial

Sponsor

Taysha Gene Therapies, Inc.

Enrollment Target

20

Start Date

Dec 2023

๐Ÿ“Š
1 of 20 participants interested
5% interest

Express Your Interest Today

Interested in learning more? Complete the form below.

Secure & Confidential

Your information is protected and will only be shared with the research team.

Why Participate?

  • No-Cost Study Care

  • Advance Medical Science

    Contribute to groundbreaking research

  • Cutting-Edge Treatment

    Access to innovative therapies

  • Expert Medical Care

    Close monitoring by specialists

  • Possible Compensation*

    For time and travel (varies by study)

  • Help Others

    Benefit future patients

*Compensation varies by study. Confirm details with coordinator.

Simple Process

  1. 1Submit this form
  2. 2Phone screening
  3. 3In-person assessment if eligible
  4. 4Begin participation

About This Study

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.

Who Can Participate

Inclusion Criteria

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 โ‰ฅ5 to โ‰ค8 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.

Exclusion Criteria

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 syndro

โ“ Not sure if you qualify? Submit your interest and a study coordinator will help determine your eligibility during the screening process.

Study Details

๐Ÿ“‹

Study Type

INTERVENTIONAL

๐Ÿ‘ฅ

Target Enrollment

20 participants

๐ŸŽ‚

Age Range

5 Years - 8 Years

โšง

Gender

FEMALE

๐Ÿข

Sponsor

Taysha Gene Therapies, Inc.

Study Locations

Choose your preferred location or select flexible during enrollment

Unknown

Chicago

Illinois

Location available
View Chicago location page
Unknown

Saint Paul

Minnesota

Location available
View Saint Paul location page
Unknown

St Louis

Missouri

Location available
View St Louis location page

Frequently Asked Questions

Q:What is a clinical trial?

A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.

Q:Is it safe to participate?

Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.

Q:Will I be compensated?

Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.

Q:Will I receive a placebo instead of treatment?

When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.

Q:Can I leave a trial if I change my mind?

Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.

Q:How long does a clinical trial last?

Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.

Still have questions? Our study coordinators are here to help.

Ready to Make a Difference?

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Looking for Rett Syndrome Treatment in La Jolla?

Join others in California exploring innovative treatment options through clinical research

Rett Syndrome Treatment Options in La Jolla, California

If you're searching for Rett Syndrome treatment in La Jolla, participating in a clinical research study may provide access to innovative approaches under expert medical supervision. This study is actively recruiting participants in La Jolla, Chicago, Boston and surrounding areas.

Clinical trials offer participants the opportunity to receive cutting-edge treatments while contributing to medical research that may help future patients with Rett Syndrome. All study-related care is provided at no cost to participants.

Local Sites
3 locations in California
Now Enrolling
Up to 20 participants
Quick Start
Screening available now

Why Consider a Clinical Trial for Rett Syndrome?

Potential Benefits

  • โœ“Access to new treatment approaches before public availability
  • โœ“Close monitoring by experienced medical professionals
  • โœ“Study-related care provided at no cost
  • โœ“Contribute to medical research for Rett Syndrome

What to Expect

  • โ†’Initial screening to determine eligibility
  • โ†’Regular check-ups and monitoring visits
  • โ†’Possible compensation for time and travel
  • โ†’You can withdraw at any time

Frequently Asked Questions About This Rett Syndrome Study

Important Clinical Trial Information

This information is provided for educational purposes and does not constitute medical advice. Clinical trial participation involves potential risks and benefits. Eligibility requirements apply and will be assessed during the screening process.

Study identifier: NCT06152237. For complete study details, visit ClinicalTrials.gov. Always consult with your healthcare provider before making decisions about your medical care or participating in clinical research.