Characterization of Translatable Neurophysiological Biomarkers to Enhance Therapeutic Development in Rett Syndrome

Status: Recruiting
Location: See all (6) locations...
Intervention Type: Other
Study Type: Observational
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.

Eligibility
Participation Requirements
Sex: Female
Minimum Age: 1
Maximum Age: 18
Healthy Volunteers: t
View:

• Rett Group: Females ages 3-18 (inclusive) with a clinical diagnosis of RTT with a likely pathogenic or known pathogenic variant in MECP2.

• Likely Rett Group: Females from 1 year to \< 5 years of age with MECP2 variant if regression has not yet occurred or child is within 6 months of last skill loss.

• Typically developing (TD) Group: Females age matched to RTT population (1-18) with no developmental or cognitive concerns as assessed using the Child/Adult Behavioral Checklist, Survey of Well-Being of Young Children (\<5yo), or the Wide Range Achievement Test-4 (\>5 yo).

Locations
United States
California
Children's Hospital of Los Angeles
RECRUITING
Los Angeles
Colorado
Children's Hospital Colorado
RECRUITING
Aurora
Massachusetts
Boston Children's Hospital
RECRUITING
Brookline
Pennsylvania
Children's Hospital of Philadelphia
RECRUITING
Philadelphia
Tennessee
Vanderbilt University Medical Center
RECRUITING
Nashville
Texas
Texas Children's Hospital
RECRUITING
Houston
Contact Information
Primary
Holly Dubbs, MS. CGC
dubbsh@chop.edu
215-590-1719
Time Frame
Start Date: 2023-10-11
Estimated Completion Date: 2029-03-31
Participants
Target number of participants: 202
Treatments
RTT Females
Females with Rett Syndrome
Controls
Females with typical development
Related Therapeutic Areas
Sponsors
Leads: Children's Hospital of Philadelphia
Collaborators: Vanderbilt University Medical Center, National Institute of Neurological Disorders and Stroke (NINDS)

This content was sourced from clinicaltrials.gov