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Brain Diseases clinical trials at UCSF

15 in progress, 10 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

  • Open-label, Long-term Safety Study of LP352 in Subjects With Developmental and Epileptic Encephalopathy

    open to eligible people ages 12-65

    The objective of this study is to assess the long-term safety, tolerability, and efficacy of adjunctive therapy of LP352 in subjects with developmental and epileptic encephalopathies who completed participation in Study LP352-201.

    San Francisco, California and other locations

  • Study to Evaluate NBI-921352 as Adjunctive Therapy in Subjects With SCN8A Developmental and Epileptic Encephalopathy Syndrome (SCN8A-DEE)

    open to eligible people ages 2-21

    The objective of this study is to assess the efficacy, safety, and pharmacokinetics of NBI-921352 as adjunctive therapy for seizures in subjects with SCN8A Developmental and Epileptic Encephalopathy Syndrome (SCN8A-DEE).

    San Francisco, California and other locations

  • Study to Investigate LP352 in Subjects With Developmental and Epileptic Encephalopathies

    open to eligible people ages 12-65

    The objective of this study is to assess the safety, tolerability, efficacy, and pharmacokinetics of adjunctive therapy of LP352 in adults and adolescents with developmental and epileptic encephalopathies.

    San Francisco, California and other locations

  • TReatment for ImmUne Mediated PathopHysiology

    open to eligible people ages 1-18

    TReatment for ImmUne Mediated PathopHysiology (TRIUMPH) is a multi-center, three arm, randomized, controlled trial of immunosuppressive therapy for children with acute liver failure. The study will determine if suppressing inflammatory responses with either corticosteroids or equine anti-thymocyte globulin therapy improves survival for children with this rare, life-threatening condition.

    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

  • Brain Development Research Program

    open to all eligible people

    Dr. Elliott Sherr and his collaborators at University of California, San Francisco (UCSF) are studying the genetic causes of disorders of cognition and epilepsy, in particular disorders of brain development that affect the corpus callosum, such as Aicardi syndrome, as well as two additional brain malformations, polymicrogyria and Dandy-Walker malformation. The goal of the investigators' research is to use a better understanding of the underlying genetic causes as a foundation to develop better treatments for these groups of patients.

    San Francisco, California

  • Longitudinal Study of Urea Cycle Disorders

    open to all eligible people

    Urea cycle disorders (UCD) are a group of rare inherited metabolism disorders. Infants and children with UCD commonly experience episodes of vomiting, lethargy, and coma. The purpose of this study is to perform a long-term analysis of a large group of individuals with various UCDs. The study will focus on the natural history, disease progression, treatment, and outcome of individuals with UCD.

    San Francisco, California and other locations

  • Neonatal Seizure Registry - Developmental Functional EValuation

    open to eligible people ages 2-8

    The NSR-DEV study is a longitudinal cohort study of around 280 Neonatal Seizure Registry participants that aims to evaluate childhood outcomes after acute symptomatic neonatal seizures, as well as examine risk factors for developmental disabilities and whether these are modified by parent well-being.

    San Francisco, California and other locations

  • Neonatal Seizure Registry, GEnetics of Post-Neonatal Epilepsy

    open to all eligible people

    The NSR-GENE study is a longitudinal cohort study of approximately 300 parent-child trios from the Neonatal Seizure Registry and participating site outpatient clinics that aims to evaluate whether and how genes alter the risk of post-neonatal epilepsy among children with acute provoked neonatal seizures. The researchers aim to develop prediction rules to stratify neonates into low, medium, and high risk for post-neonatal epilepsy based on clinical, electroencephalogram (EEG), magnetic resonance imaging (MRI), and genetic risk factors.

    San Francisco, California and other locations

  • A Study to Investigate the Long-Term Safety of ZX008 (Fenfluramine Hydrochloride) Oral Solution in Children and Adults With Epileptic Encephalopathy Including Dravet Syndrome and Lennox-Gastaut Syndrome

    Sorry, in progress, not accepting new patients

    This is an international, multicenter, open-label, long-term safety study of ZX008 in subjects with Dravet syndrome, Lennox-Gastaut syndrome or epileptic encephalopathy

    San Francisco, California and other locations

  • Extension Study to Evaluate How Safe and Tolerable NBI-921352 is as an Adjunctive Therapy for Subjects With SCN8A-DEE

    Sorry, accepting new patients by invitation only

    Extension Study to Evaluate how safe and tolerable the drug NBI-921352 is when used as Adjunctive Therapy in Subjects With SCN8A Developmental and Epileptic Encephalopathy Syndrome (SCN8A-DEE).

    San Francisco, California and other locations

  • TIME Study: Therapeutic Hypothermia for Infants With Mild Encephalopathy

    Sorry, not yet accepting patients

    The TIME study is a randomized, controlled trial to evaluate impact on early measures of neurodevelopment and the safety profile of therapeutic hypothermia in term neonates with Mild Hypoxic-Ischemic Encephalopathy who are < 6 hours of age. Neurodevelopmental outcome will be assessed at 12-14 months of age. The study will enroll 68 neonates randomized to therapeutic hypothermia or normothermia across 5 centers in California.

    Oakland, California and other locations

  • Cool Prime Comparative Effectiveness Study for Mild HIE

    Sorry, not yet accepting patients

    To determine effectiveness of therapy to improve neurodevelopmental outcomes in infants with mild HIE. To determine the adverse effects of Therapeutic Hypothermia (TH) in mild HIE on the neonate and his/her family. Determine heterogeneity of the treatment effect across key subgroups obtained in the first 6 hours after birth prior to the decision to initiate therapy.

  • Natural History Study of Infants and Children With Developmental and Epileptic Encephalopathies

    Sorry, in progress, not accepting new patients

    This is a multicenter, prospective, 2-year observational study in infants and children with developmental and epileptic encephalopathies (DEEs). The DEE currently being investigated is SCN1A-positive Dravet Syndrome.

    San Francisco, California and other locations

Our lead scientists for Brain Diseases research studies include .

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