RECRUITING

International CDKL5 Clinical Research Network

Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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

Quick Facts

Study Start:2021-02-15
Study Completion:2027-02-15
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT05558371

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:1 Month to 100 Years
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:CHILD, ADULT, OLDER_ADULT
Inclusion CriteriaExclusion Criteria
  1. * All children diagnosed with CDD age 1-month to 100 years of age that are receiving care at one of the study institutions or are registered with the International CDKL5 Disorder Database will be considered for the study population.
  1. * Individuals who do not meet study inclusion criteria.

Contacts and Locations

Study Contact

Sharon R Pincus, MA
CONTACT
303-949-7116
sharon.pincus@cuanschutz.edu
Scott T Demarest, MD MSCS
CONTACT
iccrn@childrenscolorado.org

Principal Investigator

Timothy A Benke, MD PhD
PRINCIPAL_INVESTIGATOR
University of Colorado, Denver

Study Locations (Sites)

University of California Los Angeles/UCLA Mattel Children's Hospital
Los Angeles, California, 90095
United States
University of Colorado Denver/Children's Hospital Colorado
Aurora, Colorado, 80045
United States
Harvard Medical School/Boston Children's Hospital
Boston, Massachusetts, 02115
United States
Washington University in St. Louis/St. Louis Children's Hospital
Saint Louis, Missouri, 63110
United States
NYU Langone Health
New York, New York, 10016
United States
Cleveland Clinic Foundation
Cleveland, Ohio, 44195
United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
United States
Baylor College of Medicine/ Texas Children's Hospital
Houston, Texas, 77030
United States

Collaborators and Investigators

Sponsor: University of Colorado, Denver

  • Timothy A Benke, MD PhD, PRINCIPAL_INVESTIGATOR, University of Colorado, Denver

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2021-02-15
Study Completion Date2027-02-15

Study Record Updates

Study Start Date2021-02-15
Study Completion Date2027-02-15

Terms related to this study

Additional Relevant MeSH Terms

  • CDKL5
  • CDKL5 Deficiency Disorder
  • CDD