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Dravet Syndrome clinical trials at UCSF
3 in progress, 1 open to new patients

  • A Trial of Two Fixed Doses of ZX008 (Fenfluramine HCl) in Children and Young Adults With Dravet Syndrome

    open to eligible people ages 2–18

    This is a multicenter, double-blind, parallel-group, placebo-controlled, study to assess the efficacy, safety, and PK of ZX008 when used as adjunctive therapy for uncontrolled seizures in pediatric and young adult subjects with Dravet syndrome. After an initial Screening and Baseline charting of seizure frequency, subjects who qualify for the study will be randomized (1:1:1) to receive either ZX008 (0.2 mg/kg/day, 0.8 mg/kg/day; maximum dose: 30 mg/day) or placebo. Randomization will be stratified by age group (< 6 years, ≥6 to 18 years). All subjects will be titrated to their randomized dose over a 14-day Titration Period. Following titration, subjects will continue treatment at their randomly assigned dose over a 12-week Maintenance Period. Subjects exiting the study will undergo a 2-week taper, unless they enroll in a follow-on study. Subjects will be followed for post-study safety monitoring. Parents/caregivers will use a diary daily to record the number/type of seizures, dosing, and use of rescue medication.

    San Francisco, California and other locations

  • Cannabidiol Oral Solution as an Adjunctive Therapy for Treatment of Participants With Inadequately Controlled Dravet Syndrome

    Sorry, not yet accepting patients

    This Phase 3 study will enroll participants diagnosed with Dravet Syndrome (DS) who are still experiencing at least one tonic-clonic, clonic, and/or focal seizures with motor components (FSMC) per week, despite ongoing treatment with up to three antiepileptic drugs (AEDs), and meet the other inclusion/exclusion criteria. Following a 28-day baseline period, participants will begin an 84-day treatment period. Participants will be assigned to receive twice-daily doses of placebo or cannabidiol oral solution at the highest dose determined to be safe in a previous trial. Following study completion, all participants will be invited to receive Cannabidiol Oral Solution in an open label extension study (under a separate protocol).

  • An Open-Label Extension Trial to Assess the Long-Term Safety of ZX008 (Fenfluramine Hydrochloride HCl) Oral Solution in Children and Young Adults With Dravet Syndrome

    Sorry, accepting new patients by invitation only

    This is an international, multicenter, open-label, long-term safety study of ZX008 in pediatric and young adult subjects with Dravet syndrome who have successfully completed 14 weeks of treatment in the core study (ZX008-1501 and ZX008-1502).

    San Francisco, California and other locations