Nervous System Diseases clinical trials at UCSF

5 in progress, 3 open to eligible people

Showing trials for

  • An Open-Label Extension of the Study XEN496 (Ezogabine) in Children With KCNQ2-DEE

    open to eligible people ages 1 month to 6 years

    To assess the long-term safety and tolerability of XEN496 in pediatric subjects with KCNQ2 developmental and epileptic encephalopathy (KCNQ2-DEE) who had participated in the primary study (XPF-009-301).

    San Francisco, California and other locations

  • XEN496 (Ezogabine) in Children With KCNQ2 Developmental and Epileptic Encephalopathy

    open to eligible people ages 1 month to 6 years

    To investigate the potential antiseizure effects of adjunctive XEN496 (ezogabine) compared with placebo in children with KCNQ2 Developmental and Epileptic Encephalopathy (KCNQ2-DEE).

    San Francisco, California and other locations

  • Rett Syndrome Registry

    open to eligible people ages 0-99

    The Rett Syndrome Registry is a longitudinal observational study of individuals with MECP2 mutations and a diagnosis of Rett syndrome. Designed together with the IRSF Rett Syndrome Center of Excellence Network medical directors, this study collects data on the signs and symptoms of Rett syndrome as reported by the Rett syndrome experts and by the caregivers of individuals with Rett syndrome. This study will be used to develop consensus based guidelines for the care of your loved ones with Rett syndrome and to facilitate the development of better clinical trials and other aspects of the drug development path for Rett syndrome.

    Oakland, California and other locations

  • Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy

    Sorry, in progress, not accepting new patients

    This study is a long-term study of ataluren in participants with nonsense mutation Duchenne muscular dystrophy.

    Oakland, California and other locations

  • Human Epilepsy Project 2: Resistant Focal Seizures Study

    Sorry, in progress, not accepting new patients

    The HEP2 study is designed to better understand the challenges of living with focal seizures that do not respond to medication, by following 205 people with medication-resistant focal epilepsy over two years to measure changes in health status, healthcare costs, quality of life, and biomarkers of epilepsy severity and treatment response.

    San Francisco, California and other locations

Our lead scientists for Nervous System Diseases research studies include .

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