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International CDKL5 Clinical Research Network
Sponsor: University of Colorado, Denver
Summary
Pathogenic variants in the Cyclin-dependent kinase like 5 (CDKL5) gene cause CDKL5 deficiency disorder (CDD, MIM 300672, 105830), a severe developmental and epileptic encephalopathy associated with cognitive and motor impairments and cortical visual impairment. While capability for disease modifying therapies is accelerating, there is a critical barrier for clinical trial readiness that may result in failure of these therapies, not due to lack of efficacy but due to lack of validated outcome measures and biomarkers. The measures and biomarkers validated here will be adaptable to other developmental and epileptic encephalopathies.
Official title: Multi-Site Validation of Biomarkers and Core Clinical Outcome Measures for Clinical Trials Readiness in CDKL5 Deficiency Disorder
Key Details
Gender
All
Age Range
1 Month - 100 Years
Study Type
OBSERVATIONAL
Enrollment
1000
Start Date
2021-02-15
Completion Date
2027-02-15
Last Updated
2023-12-06
Healthy Volunteers
No
Conditions
Interventions
No intervention.
No intervention administered as part of this study; observational only.
Locations (9)
University of California Los Angeles/UCLA Mattel Children's Hospital
Los Angeles, California, United States
University of Colorado Denver/Children's Hospital Colorado
Aurora, Colorado, United States
Harvard Medical School/Boston Children's Hospital
Boston, Massachusetts, United States
Washington University in St. Louis/St. Louis Children's Hospital
St Louis, Missouri, United States
NYU Langone Health
New York, New York, United States
Cleveland Clinic Foundation
Cleveland, Ohio, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
Baylor College of Medicine/ Texas Children's Hospital
Houston, Texas, United States
Telethon Kids Institute/Perth Children's Hospital
Perth, Nedlands Western Australia, Australia