Rett Syndrome Registry

Recruiting now

Conditions studied: Rett Syndrome, Rett Syndrome, Atypical, Genetic Disease, Genetic Diseases, X-Linked, Intellectual Disability, Neurobehavioral Manifestations, Neurologic Manifestations, Neurologic Disorder, Neurodevelopmental Disorders, Nervous System Diseases

In brief

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 study will be used to develop consensus based guidelines for the care of your loved ones with Rett syndrome and to facilitate the development of better clinical trials and other aspects of the drug development path for Rett syndrome.

Key facts

Study ID

NCT05432349

Run by

International Rett Syndrome Foundation

People needed

3000

Starts

2022-08-02

Expected to finish

2028-07-01

Last updated by the study team

2026-06-30

Who can join

Age: any, up to 99. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Male or female with a pathologic loss of function alteration of MECP2

You may not qualify if…

• Male or female with a gain of function alteration of MECP2, including those with MEPC2 duplication or triplication

Where it is running

• University of Alabama — Birmingham, Alabama, United States (enrolling)
• Children's Hospital Los Angeles — Los Angeles, California, United States (enrolling)
• UCSF Benioff Children's Hospital — Oakland, California, United States (enrolling)
• Rady Children's Hospital - San Diego — San Diego, California, United States (enrolling)
• Children's Hospital Colorado — Denver, Colorado, United States (enrolling)
• Nicklaus Children's Hospital — Miami, Florida, United States (enrolling)
• Rush University Medical Center — Chicago, Illinois, United States (enrolling)
• Kennedy Krieger Institute — Baltimore, Maryland, United States (enrolling)
• Boston Children's Hospital — Boston, Massachusetts, United States (enrolling)
• Gillette Children's Specialty Healthcare — Saint Paul, Minnesota, United States (enrolling)
• Washington University in St. Louis — St Louis, Missouri, United States (enrolling)
• The University of North Carolina at Chapel Hill — Chapel Hill, North Carolina, United States (enrolling)
• Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio, United States (enrolling)
• Nationwide Children's Hospital — Columbus, Ohio, United States (enrolling)
• Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States (enrolling)
• Greenwood Genetic Center — Greenwood, South Carolina, United States (enrolling)
• Vanderbilt Kennedy Center — Nashville, Tennessee, United States (enrolling)
• Children's Health — Dallas, Texas, United States (enrolling)
• Texas Children's Hospital — Houston, Texas, United States (enrolling)

Full record on ClinicalTrials.gov

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