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, California and other locations
Soticlestat in Adults and Children With Rare Epilepsies
Sorry, in progress, not accepting new patients
The main aim is to assess the long-term safety and tolerability of soticlestat when used along with other anti-seizure treatment. Participants will receive soticlestat twice a day. Participants will visit the study clinic every 2-6 months throughout the study. Study treatments may continue as long as the participant is receiving benefit from it.
Los Angeles, California 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, California and other locations
Observational Study in 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, California and other locations
Our lead scientists for CDKL5 Deficiency Disorder research studies include Rajsekar Rajaraman, MD.
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