TSHA-102 for Rett Syndrome

Recruiting at 11 trial locations

Overseen By Taysha Gene Therapies Medical Information

Age: < 18

Sex: Female

Trial Phase: Phase 1 & 2

Sponsor: Taysha Gene Therapies, Inc.

Disqualifiers: Other neurodevelopmental disorders, Brain injury, Atypical Rett, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Approved in 3 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

20 Participants Needed

Trial Summary

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

What is the purpose of this trial?

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.

Research Team

Laura Pisani, M.D.

Principal Investigator

Taysha Gene Therapies

Eligibility Criteria

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.

Inclusion Criteria

The person taking care of you must be okay with you getting a blood transfusion if the doctor thinks it's necessary.

I am up to date with all my vaccinations, with the last dose received over 42 days ago.

I have Rett Syndrome with a confirmed MECP2 gene mutation.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Dose-Escalation

Participants receive a single intrathecal administration of TSHA-102 at varying dose levels to evaluate safety and tolerability

52 weeks

Regular visits for monitoring

Dose-Expansion

Further evaluation of safety, tolerability, and preliminary efficacy at selected dose levels

52 weeks

Regular visits for monitoring

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 years

Treatment Details

Interventions

• TSHA-102

Trial Overview 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.

Participant Groups

Group I: Cohort 2 Experimental Treatment 1 Intervention

Dose Level 2

Group II: Cohort 1 Experimental Treatment 1 Intervention

Dose Level 1

TSHA-102 is already approved in United States, Canada, United Kingdom for the following indications:

Approved in United States as TSHA-102 for:

• Rett syndrome (investigational)

Approved in Canada as TSHA-102 for:

• Rett syndrome (investigational)

Approved in United Kingdom as TSHA-102 for:

• Rett syndrome (investigational)

Who Is Running the Clinical Trial?

Taysha Gene Therapies, Inc.

Lead Sponsor

Trials

5

Recruited

60+