Award
National Institute of Neurological Disorders and Stroke 5U01NS114312-05
Multi-Site Validation of Biomarkers and Core Clinical Outcome Measures for Clinical Trials Readiness in CDKL5 Deficiency Disorder
Recipient
University of Colorado Denver
Award Amount
$782,432.00
Ceiling
$782,432.00
Awarded
February 13, 2025
Identifier
5U01NS114312-05
NIH grant awarded to University of Colorado Denver to develop and validate clinical outcome measures and biomarkers for CDKL5 deficiency disorder, enabling clinical trial readiness and testing of new therapeutics.
Description
This project aims to develop and validate clinical outcome measures (COMs) and biomarkers for assessing disease modification in CDKL5 deficiency disorder (CDD). The study will conduct a multi-site clinical trial readiness assessment to ensure the implementation, stability, and baseline data collection of these measures. The goal is to establish clinical trial readiness for CDD, facilitating the testing of new therapeutics, including gene therapy.