Dravet Syndrome clinical trials at UCSF

5 in progress, 2 open to eligible people

Dravet Syndrome is a genetic epilepsy disorder starting in infancy. UCSF is investigating a study for ETX101 to assess its effects on children with the syndrome. Another trial at UCSF is exploring the safety and efficacy of EPX-100 in patients with Dravet Syndrome.

Showing trials for

  • ETX101 in Infants and Children With SCN1A-Positive Dravet Syndrome

    open to eligible people ages 6 months to 47 months

    ENDEAVOR is a Phase 1/2, 2-part, multicenter study to evaluate the safety and efficacy of ETX101 in participants with SCN1A-positive Dravet syndrome aged ≥6 to <36 months (Part 1) and aged ≥6 to <48 months (Part 2). Part 1 follows an open-label, dose-escalation design, and Part 2 is a randomized, double-blind, sham delayed-treatment control, dose-selection study.

    San Francisco, California and other locations

  • EPX-100 (Clemizole Hydrochloride) in Participants With Dravet Syndrome

    open to eligible people ages 2 years and up

    This is a multicenter, Phase 3, randomized, double-blind, placebo-controlled study designed to evaluate the efficacy and safety of clemizole HCL (EPX-100) as adjunctive therapy in children and adult participants with Dravet syndrome.

    San Francisco, California and other locations

  • Soticlestat as an Add-on Therapy in Children and Adults With Dravet Syndrome or Lennox-Gastaut Syndrome

    Sorry, in progress, not accepting new patients

    The main aim of the study is to learn if soticlestat, when given as an add-on therapy, reduces the number of seizures in children and adults with Dravet Syndrome (DS) or Lennox-Gastaut Syndrome (LGS). Participants will receive their standard anti-seizure therapy, plus tablets of soticlestat. There will be scheduled visits and follow-up phone calls throughout the study.

    San Francisco, California and other locations

  • STK-001 for Patients With Dravet Syndrome

    Sorry, accepting new patients by invitation only

    Stoke Therapeutics is evaluating the long-term safety & tolerability of repeated doses of STK-001 in patients with Dravet syndrome who previously participated in studies of STK-001. Change in seizure frequency and overall clinical status, and quality of life will be measured as secondary endpoints in this open-label study.

    San Francisco, California and other locations

  • Fenfluramine in Children With Dravet Syndrome Under 24 Months of Age

    Sorry, not currently recruiting here

    Dravet syndrome is a genetic epilepsy associated with pathogenic variants in SCN1A that codes for Nav1.1, a protein necessary for sodium channels. Children with Dravet syndrome classically present in the first year of life with prolonged seizures, often hemiclonic and in the setting of fever or temperature changes such as getting in or out of bath water. Many anti-seizure medications are sodium channel blockers and exacerbate seizures in this patient population. This creates some limitations in medication choices for this patient population. Recently fenfluramine was approved for use in Dravet syndrome for people 2 years and older. Randomized studies demonstrated a 74.9% reduction of convulsive motor seizures compared to 19.2% in the placebo group. Additionally, 16% of children treated with fenfluramine were seizure free. Fenfluramine is likely to be as effective in children under the age of 2 years. The current study has proposed a treatment protocol to allow access to fenfluramine for children under 24 months of age.

    San Francisco, California and other locations

Our lead scientists for Dravet Syndrome research studies include .

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