Baseline Characteristics and Feasibility of Clinical Outcome Measures in CDKL5 Deficiency Disorder: The CANDID Observational Study
| Journal | Epilepsia |
| Study Type | Observational Study |
| Population | Human participants |
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CDKL5 deficiency disorder (CDD) is a rare X-linked developmental and epileptic encephalopathy caused by loss-of-function variants in the CDKL5 gene. Preclinical experiments using enzyme replacement or gene therapies show promise and could be transformative therapies. This precompetitive consortium sought to harmonize nonseizure clinical endpoint selection for efficacy trials. Clinical Assessment of Neurodevelopmental Measures in CDD (CANDID) is an ongoing study evaluating the feasibility and suitability of neurocognitive tests and functioning scales in CDD patients. CANDID is a 3-year, longitudinal, noninterventional global study involving children and adults with CDD. On-site and remote visits include clinical, behavioral, developmental, and quality of life assessments. We enrolled 112 patients (111 included in analyses); mean age = 8.3 years (range 16 seizures; six were seizure-free. Median seizure onset was at 1.5 months (range = 0-66). Patients used an average of 2.6 antiseizure me
“This is a development worth tracking. The clinical implications will become clearer as more evidence accumulates.”
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