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Gene Therapy
Gene Therapy (TSHA-102) for Rett Syndrome
Phase 1 & 2
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 clinical diagnosis of classical/typical Rett syndrome with a documented pathogenic mutation of the methyl-CpG-binding protein 2 (MECP2) gene that results in loss of function.
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline through week 52
Awards & highlights
Study Summary
This trial will evaluate the safety and effectiveness of an experimental gene therapy for adult female Rett syndrome patients. Two doses will be tested over a 63 month period.
Who is the study for?
This trial is for adult females with classical Rett syndrome confirmed by a specific MECP2 gene mutation. Candidates must be open to blood transfusions if needed and cannot require invasive breathing support, have uncontrolled seizures, other progressive genetic syndromes, significant brain injuries affecting the nervous system, or abnormal development in early infancy.Check my eligibility
What is being tested?
The REVEAL Adult Study tests TSHA-102, a gene therapy for Rett syndrome in women. It's an early-stage trial to see how safe it is and how well it works at two different doses over up to 63 months.See study design
What are the potential side effects?
As this is an investigational gene therapy study focusing on safety and tolerability, potential side effects are not fully known but may include reactions related to the drug infusion process or lumbar puncture procedure.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I have Rett syndrome with a confirmed MECP2 gene mutation.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ baseline through week 52
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline through week 52
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Rett Syndrome
Rett Syndrome
Change from baseline in participant's status after TSHA-102 administration as assessed by the Revised Motor Behavior Assessment (R-MBA)
+1 moreSecondary outcome measures
Change from Baseline in participant's status after TSHA-102 administration as assessed by Clinical Global Impressions-Severity (CGI-S)
Electroencephalography
Electroencephalography
+2 moreTrial 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
30 Total Patients Enrolled
1 Trials studying Rett Syndrome
6 Patients Enrolled for Rett Syndrome
Meredith Schultz, M.D.Study DirectorTaysha Gene Therapies
Suyash Prasad, MBBS, M.SC., MRCP, MRCPCH, FFPMStudy DirectorTaysha Gene Therapies
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I am willing to receive blood or blood products if needed for an adverse event.I have a MECP2 mutation, but it doesn't cause Rett syndrome.I need a machine to help me breathe.I cannot undergo spinal procedures or take certain medications due to my health conditions.You had significant delays in learning and movement during the first 6 months of life.I have had a brain injury that affects my nervous system.I have been diagnosed with atypical Rett syndrome.I have a neurodevelopmental disorder not related to MECP2 mutation.I have Rett syndrome with a confirmed MECP2 gene mutation.
Research Study Groups:
This trial has the following groups:- Group 1: Cohort 2
- Group 2: Cohort 1
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
What is the total number of participants in this clinical experiment?
"Affirmative. Clinicaltrials.gov displays that this clinical trial, first posted on November 8th 2022, is actively recruiting participants. In total, 12 patients are needed for the single medical centre hosting the study."
Answered by AI
Is there an ongoing recruitment period for this clinical trial?
"According to clinicaltrials.gov, this medical research is currently open for participants; the trial was initially published on November 8th 2022 and its information has been recently updated as of the same date."
Answered by AI
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