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Gene Therapy

Cohort 2 for Rett Syndrome

Phase 1
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

Study 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.Check my eligibility
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.See study design
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

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Primary Safety
Secondary outcome measures
Exploratory Efficacy

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
Benit Maru, Bsc, MB ChB, MSc, PhDStudy DirectorTaysha Gene Therapies

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

In how many locations is this trial currently being conducted?

"The Children's Hospital of Philadelphia Research Institute in Philadelphia, Pennsylvania; University of California San Diego (UCSD) in La Jolla, California; and Boston Children's at Brookline in Boston, Massachusetts are among the 3 sites participating in this trial. Additionally, there are 8 other locations involved as well."

Answered by AI

Are there any available vacancies for potential participants in this research study?

"Indeed, as per the information available on clinicaltrials.gov, this study is presently seeking eligible participants. The trial was originally made public on December 12th, 2023, and has been recently updated on December 19th, 2023."

Answered by AI

Am I eligible to be a part of this research trial?

"In order to be considered for participation in this clinical trial, individuals must have a diagnosis of Rett syndrome and fall within the age range of 5 to 8 years old. The study is currently seeking a total of six eligible candidates."

Answered by AI

How many individuals are enrolled as participants in this ongoing medical study?

"Indeed, according to the information available on clinicaltrials.gov, this trial is currently seeking participants. Originally posted on December 12th, 2023 and last updated on December 19th, 2023, the study aims to recruit a total of 6 patients from 8 different sites."

Answered by AI

Is the age criterion for this research study limited to individuals under 35 years old?

"This study is inviting participants who are over the age of 5 and under the age of 8 to enroll."

Answered by AI

What level of risk does Cohort 2 pose to patients?

"Based on our evaluation at Power, we assign a safety rating of 1 to Cohort 2. This decision is based on the limited data available in this Phase 1 trial regarding both safety and effectiveness."

Answered by AI

Who else is applying?

What site did they apply to?
Boston Children's at Brookline
What portion of applicants met pre-screening criteria?
Met criteria
Recent research and studies
clinicaltrials.govStudy
Safety and Efficacy of TSHA-102 in Pediatric Females With Rett Syndrome (REVEAL Pediatric Study)
2023. "Safety and Efficacy of TSHA-102 in Pediatric Females With Rett Syndrome (REVEAL Pediatric Study)". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT06152237.
All > Rett Syndrome > Paid Studies Minneapolis
~4 spots leftby Nov 2028
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