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Neurophysiologic Biomarkers in Rett Syndrome
Sponsor: Children's Hospital of Philadelphia
Summary
The goal of this observational study is to identify candidate biomarkers in individuals with Rett Syndrome (RTT). The main questions it aims to answer are: * Do these biomarkers change during clinical changes in individuals with RTT? * Are biomarkers stable over time in clinically stable individuals? * Do these biomarkers correlate with severity of RTT? Participants will be asked to undergo an electroencephalogram (EEG) with measurements of Evoked Potentials (EP) to measure electrical activity in the brain. Researchers will compare findings in individuals with RTT to those in typically developing individuals to see if there are differences between the two groups.
Official title: Characterization of Translatable Neurophysiological Biomarkers to Enhance Therapeutic Development in Rett Syndrome
Key Details
Gender
FEMALE
Age Range
1 Year - 18 Years
Study Type
OBSERVATIONAL
Enrollment
202
Start Date
2023-10-11
Completion Date
2029-03-31
Last Updated
2025-10-20
Healthy Volunteers
Yes
Conditions
Interventions
EEG and Auditory and Visual Evoked Potentials (AEP and VEP)
Through up to eight standardized sessions, participants will undergo AEP and VEP, as well as resting state EEG.
Clinical assessment
Established clinical measures for RTT will be collected for RTT participants
Locations (6)
Children's Hospital of Los Angeles
Los Angeles, California, United States
Children's Hospital Colorado
Aurora, Colorado, United States
Boston Children's Hospital
Brookline, Massachusetts, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
Vanderbilt University Medical Center
Nashville, Tennessee, United States
Texas Children's Hospital
Houston, Texas, United States