CDKL5 Deficiency Disorder clinical trials at UCLA
4 in progress, 1 open to eligible people
International CDKL5 Clinical Research Network
open to eligible people ages 1 month to 100 years
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.
Los Angeles 5368361, California 5332921 and other locations
Intermediate-Size Expanded Access Protocol (EAP) for LP352
Sorry, not accepting new patients
This is an intermediate-size expanded access program (EAP) study. The purpose of this EAP is to provide continued access to LP352, an investigational drug product being investigated in participants with DEEs. The EAP study will allow continued treatment with LP352 for eligible participants diagnosed with treatment resistant DEEs who successfully completed an LP352 Clinical Trial (Enrollment by Invitation) or an immediate family member who has the exact same gene mutation resulting in the same DEE epilepsy syndrome phenotype or a patient who previously participated in the lorcaserin EAP.
Los Angeles 5368361, California 5332921 and other locations
ZX008 in Subjects With CDKL5 Deficiency Disorder
Sorry, in progress, not accepting new patients
This is a multicenter, double-blind, parallel-group, placebo controlled, 2-part study to evaluate the efficacy and safety of ZX008 when used as adjunctive therapy for the treatment of uncontrolled seizures in children and adults with cyclin-dependent kinase like-5 (CDKL5) deficiency disorder (CDD).
Los Angeles 5368361, California 5332921 and other locations
Patients With Cyclin-dependent Kinase-like 5 Deficiency Disorder
Sorry, in progress, not accepting new patients
Observational, noninterventional, 3-year study to examine the presence of detectable differences in neurocognitive, developmental, motor, neurophysiologic, and quality of life measures over time in patients with cyclin-dependent kinase-like 5 deficiency disorder.
Los Angeles 5368361, California 5332921 and other locations
Our lead scientists for CDKL5 Deficiency Disorder research studies include Shaun A. Hussain, MD.
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