To Investigate the Efficacy of Treatment With Oral NA-921 (Bionetide) Versus Placebo in Females With Rett Syndrome
Primary Objective • To investigate the efficacy of treatment with oral Bionetide versus placebo in girls and women with Rett syndrome Key Secondary Objective • To investigate the efficacy of treatme...
Safety and Efficacy of TSHA-102 in Pediatric Females With Rett Syndrome (REVEAL Pediatric 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 and Efficacy of TSHA-102 in Adolescent and Adult Females With Rett Syndrome (REVEAL Adult Study)
The REVEAL Adult Study is a multi-center, Phase 1/2 open-label, dose-escalation study of TSHA-102, an investigational gene therapy, in adult females with Rett syndrome. The safety, tolerability, and p...
A Novel, Regulated Gene Therapy (NGN-401) Study for Females With Rett Syndrome
This study will evaluate the safety profile of the investigational gene therapy, NGN-401, in females with typical Rett syndrome....
Top Cities for Rett Syndrome Clinical Trials
Rett Syndrome clinical trials are recruiting across 22 cities. Here are the cities with the most active studies:
About Rett Syndrome
Rett syndrome is a rare genetic neurological disorder that primarily affects girls, causing a regression in development after initially normal growth. It is caused by mutations in the MECP2 gene. Treatment is supportive, with gene therapies under investigation.
Clinical trials are advancing new treatments for rett syndrome. Currently, 4 studies are recruiting a combined 262 participants across the United States. Research is being conducted by 3 organizations including Biomed Industries, Inc., Taysha Gene Therapies, Inc., Neurogene Inc..
2026 Rett Syndrome Research Landscape
As of March 2026, the rett syndrome clinical trial landscape includes 4 actively recruiting studies across 22 cities in the United States. These studies are collectively seeking 262 participants, with an average enrollment target of 66 per study.
Research is being led by 3 different organizations, including Biomed Industries, Inc., Taysha Gene Therapies, Inc., Neurogene Inc..
Geographically, rett syndrome trials are most concentrated in Chicago, Illinois (4 trials); Boston, Massachusetts (4 trials); La Jolla, California (3 trials); Aurora, Colorado (2 trials); Saint Paul, Minnesota (2 trials) and 7 other cities.
Featured Rett Syndrome Studies
Highlighted recruiting studies for rett syndrome, selected by enrollment size and research scope.
To Investigate the Efficacy of Treatment With Oral NA-921 (Bionetide) Versus Placebo in Females With Rett Syndrome
Primary Objective • To investigate the efficacy of treatment with oral Bionetide versus placebo in girls and women with Rett syndrome Key Secondary Objective • To investigate the efficacy of treatment with oral Bionetide versus placebo on ability to communicate in girls and women with Rett syndrome
Safety and Efficacy of TSHA-102 in Pediatric Females With Rett Syndrome (REVEAL Pediatric 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.
Safety and Efficacy of TSHA-102 in Adolescent and Adult Females With Rett Syndrome (REVEAL Adult Study)
The REVEAL Adult Study is a multi-center, Phase 1/2 open-label, dose-escalation study of TSHA-102, an investigational gene therapy, in adult females with Rett syndrome. The safety, tolerability, and preliminary efficacy of two dose levels will be evaluated. The study duration is estimated to be up to 63 months.
Frequently Asked Questions About Rett Syndrome Clinical Trials
Are there rett syndrome clinical trials near me?
Yes, there are 4 rett syndrome clinical trials currently recruiting across 22+ cities in the United States, including Chicago, Illinois; Boston, Massachusetts; La Jolla, California. Browse the studies above to find one at a location convenient for you.
How do I join a rett syndrome clinical trial?
To join a rett syndrome clinical trial: 1) Browse the available studies on this page, 2) Click on a study that interests you, 3) Check the study locations to find a site near you, 4) Review the eligibility criteria, and 5) Contact the study site or complete the eligibility form. The process is free and you can withdraw at any time.
Are rett syndrome clinical trials free?
Yes, participation in rett syndrome clinical trials is free. Study-related treatments, medical tests, and doctor visits are provided at no cost to participants. Many studies also offer compensation for your time and travel expenses.
What types of rett syndrome treatments are being studied?
Current rett syndrome clinical trials are testing a range of approaches. These include new drugs, combination therapies, medical devices, and other interventions sponsored by 3 research organizations.
Is it safe to participate in rett syndrome clinical trials?
Clinical trials are carefully regulated by the FDA and institutional review boards (IRBs). All trials must follow strict safety protocols, and participants receive close medical monitoring throughout the study. You can withdraw from a trial at any time without penalty.
Data updated March 1, 2026 from ClinicalTrials.gov
About This Data
Clinical trial information on this page is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health (NIH) and National Library of Medicine (NLM). Study data is refreshed every hour to ensure accuracy.
Medical Disclaimer: The information provided on this page is for informational purposes only and does not constitute medical advice. Always consult with a qualified healthcare provider before making decisions about clinical trial participation or changes to your treatment plan.
Page reviewed by the HelloStudys Research Team · Last updated March 1, 2026 · Data from ClinicalTrials.gov