International CDKL5 Clinical Research Network
Part of paid clinical trials in Los Angeles, California.
- Sponsor
- University of Colorado, Denver
- Study ID
- NCT05558371
- Status
- Recruiting
Conditions
- CDD
- CDKL5
- CDKL5 Deficiency Disorder
Eligibility Criteria
- Sex
- ALL
- Age
- 1 Month - 100 Years
- Healthy Volunteers
- Not accepted
Interventions
- No intervention. — OTHERNo intervention administered as part of this study; observational only.
Study Details
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.
Key Dates
- Start date
- Feb 15, 2021
- Status verified
- Dec 2023
- Primary completion
- Feb 15, 2026
- Completion
- Feb 15, 2027
Study Design
- Enrollment
- 1,000 participants (estimated)
Primary Outcome Measure
CDKL5 Deficiency Disorder (CDD) Clinical Severity Assessment - Clinician (CCSA-Clinician) [ Time Frame: 5 years ]
Central Contacts
- Sharon R Pincus, MA303-949-7116
- Scott T Demarest, MD MSCS
Locations (8)
| Facility | City | State | ZIP | Site coordinators |
|---|---|---|---|---|
| University of California Los Angeles/UCLA Mattel Children's Hospital | Los Angeles | California | 90095 | Angela Martinez Rajsekar Rajaraman, MD (PRINCIPAL_INVESTIGATOR) |
| University of Colorado Denver/Children's Hospital Colorado | Aurora | Colorado | 80045 | Timothy A Benke, MD PhD (PRINCIPAL_INVESTIGATOR) Scott T Demarest, MD MSCS (SUB_INVESTIGATOR) |
| Harvard Medical School/Boston Children's Hospital | Boston | Massachusetts | 02115 | Isa Haviland Jenna Lukash Heather E Olson, MD (PRINCIPAL_INVESTIGATOR) Will Hong, MD (SUB_INVESTIGATOR) |
| Washington University in St. Louis/St. Louis Children's Hospital | St Louis | Missouri | 63110 | Olga Novak Ali Vonderheid Judith Weisenberg, MD (PRINCIPAL_INVESTIGATOR) |
| NYU Langone Health | New York | New York | 10016 | Jacob Sloane Juliana Laze Dana Price, MD (PRINCIPAL_INVESTIGATOR) |
| Cleveland Clinic Foundation | Cleveland | Ohio | 44195 | Xiaoming Zhang Honglian Huang Elia Pestana Knight, MD (PRINCIPAL_INVESTIGATOR) |
| Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | Holly Dubbs Eric Marsh, MD PhD (PRINCIPAL_INVESTIGATOR) Erin Prange, MSN CRNP (SUB_INVESTIGATOR) |
| Baylor College of Medicine/ Texas Children's Hospital | Houston | Texas | 77030 | Elif Dundar Bernhard Suter (PRINCIPAL_INVESTIGATOR) |