Rett Syndrome Clinical Trial
Official title:
Characterization of Translatable Neurophysiological Biomarkers to Enhance Therapeutic Development in Rett Syndrome
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.
The main goal of the project is to identify potential biomarkers that can become measures for intervention and other translational studies and, at the same time, provide insight into abnormal synaptic activity and pathogenesis of RTT. Therefore, the proposed assessments will be performed with females with RTT and age matched typically developing females. These electrophysiological assessments will be compared to established clinical outcome measures from previous work in the NIH funded Rett and Rett related disorders natural history study. The neurophysiological parameters for RTT will be correlated with each other and also to disease staging, overall clinical severity scores and through exploratory analyses with specific clinical features. The investigators will also be testing procedures to perform the recordings, electrode types and placement, and ways to reduce movement and artifact within the data to establish best practices. ;
| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Completed |
NCT04988867 -
An Open-Label Study of Trofinetide for the Treatment of Girls Two to Five Years of Age Who Have Rett Syndrome
|
Phase 2/Phase 3 | |
| Recruiting |
NCT00069550 -
Independent Studies of Dextromethorphan and of Donepezil Hydrochloride for Rett Syndrome
|
Phase 3 | |
| Enrolling by invitation |
NCT06139172 -
Promoting Prosocial Behavior in Syndromic Intellectual and Developmental Disabilities
|
N/A | |
| Not yet recruiting |
NCT04014985 -
Patients With RETT Syndrome
|
N/A | |
| Not yet recruiting |
NCT04041713 -
A Pilot Study of an Antioxidant Cocktail vs. Placebo in the Treatment of Children and Adolescents With Rett Syndrome
|
Phase 2 | |
| Completed |
NCT02705677 -
Biobanking of Rett Syndrome and Related Disorders
|
||
| Terminated |
NCT02790034 -
Evaluation of the Efficacy, Safety, and Tolerability of Sarizotan in Rett Syndrome With Respiratory Symptoms
|
Phase 2/Phase 3 | |
| Enrolling by invitation |
NCT03655223 -
Early Check: Expanded Screening in Newborns
|
||
| Recruiting |
NCT04463316 -
GROWing Up With Rare GENEtic Syndromes
|
||
| Completed |
NCT04776746 -
Open-Label Extension Study of Trofinetide for Rett Syndrome
|
Phase 3 | |
| Completed |
NCT04181723 -
Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome (LAVENDER™)
|
Phase 3 | |
| Enrolling by invitation |
NCT03836300 -
Parent and Infant Inter(X)Action Intervention (PIXI)
|
N/A | |
| Completed |
NCT04514549 -
ASSESSING EMERALD AND MC10 BIOSTAMP nPOINT BIOSENSORS FOR RETT SYNDROME
|
||
| Completed |
NCT02738281 -
Natural History of Rett Syndrome & Related Disorders
|
||
| Terminated |
NCT02562820 -
An Exploratory Trial of Ketamine for the Treatment of Rett Syndrome
|
Phase 1 | |
| Completed |
NCT05687214 -
Osteopathic Manipulative Treatment for Constipation in People With Rett Syndrome
|
N/A | |
| Recruiting |
NCT06199700 -
Esketamine for the Treatment of Rett Syndrome
|
Early Phase 1 | |
| Completed |
NCT03941444 -
ANAVEX2-73 Study in Patients With Rett Syndrome
|
Phase 3 | |
| Recruiting |
NCT06346106 -
The Diagnostic Experience of Male Rett Syndrome
|
||
| Not yet recruiting |
NCT06338267 -
Validation of Innovative Biosensors for Rett Autonomic Symptom Tracking
|