Recruiting Studies
Advanced SearchData: ClinicalTrials.gov · Source of record for eligibility and locations
Top Cities for Rett Syndrome Clinical Trials
Rett Syndrome clinical trials are recruiting across 33 cities. Here are the cities with the most active studies:
Rett Syndrome Trial Phases
14 research organizations are sponsoring rett syndrome trials.
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, 17 studies are recruiting a combined 18,948 participants across the United States. Research is being conducted by 14 organizations including University of Colorado, Denver, Rett Syndrome Research Trust, International Rett Syndrome Foundation and 11 others.
2026 Rett Syndrome Research Landscape
As of July 2026, the rett syndrome clinical trial landscape includes 17 actively recruiting studies across 33 cities in the United States. These studies are collectively seeking 18,948 participants, with an average enrollment target of 1,115 per study.
The research spans multiple phases of development: 3 studies are in NA, 2 studies are in PHASE3, 1 study is in PHASE2. The presence of Phase 3 trials indicates that some rett syndrome treatments are in late-stage testing and may be approaching regulatory review.
Research is being led by 14 different organizations, including University of Colorado, Denver, Rett Syndrome Research Trust, International Rett Syndrome Foundation, Midwest Biomedical Research Foundation, Case Western Reserve University, and 9 others. The large number of sponsors reflects significant research interest and investment in rett syndrome treatment advancement.
Geographically, rett syndrome trials are most concentrated in Boston, Massachusetts (5 trials); Los Angeles, California (4 trials); Chicago, Illinois (4 trials); St Louis, Missouri (3 trials); Baltimore, Maryland (3 trials) and 7 other cities.
Featured Rett Syndrome Studies
Highlighted recruiting studies for rett syndrome, selected by enrollment size and research scope.
TREAT-BE Study (Treatment With Resection and Endoscopic Ablation Techniques for Barrett's Esophagus)
A prospective outcomes study in patients with and esophageal cancer (EAC) and Barrett's esophagus (BE) associated neoplasia being evaluated for endoscopic eradication therapy (EET).
The Rett Syndrome Global Registry
The Rett Global Registry is a fully remote, global, caregiver-reported registry to collect information about caring for a loved one with Rett syndrome. In addition, caregivers have the ability to track and graph their loved one's symptoms and care strategies over time, store information for central access, and opt-in to complete medical record consolidation and summary. Qualified researchers and t...
Rett Syndrome Registry
The Rett Syndrome Registry is a longitudinal observational study of individuals with MECP2 mutations and a diagnosis of Rett syndrome. Designed together with the IRSF Rett Syndrome Center of Excellence Network medical directors, this study collects data on the signs and symptoms of Rett syndrome as reported by the Rett syndrome experts and by the caregivers of individuals with Rett syndrome. This ...
Frequently Asked Questions About Rett Syndrome Clinical Trials
Are there rett syndrome clinical trials near me?
Yes, there are 17 rett syndrome clinical trials currently recruiting across 33+ cities in the United States, including Boston, Massachusetts; Los Angeles, California; Chicago, Illinois. 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 across NA (3 studies), PHASE3 (2 studies), PHASE2 (1 study). These include new drugs, combination therapies, medical devices, and other interventions sponsored by 14 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 July 15, 2026 from ClinicalTrials.gov
About This Data
Data: ClinicalTrials.gov · Source of record for eligibility and locations
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 July 15, 2026 · Data from ClinicalTrials.gov