CDKL5 Deficiency Disorder clinical trials at University of California Health

2 in progress, 1 open to eligible people

Showing trials for

  • 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.

    at UCLA

  • A Study of 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.

    at UCLA

Our lead scientists for CDKL5 Deficiency Disorder research studies include .

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