Dravet Syndrome clinical trials at UCSF

6 in progress, 4 open to eligible people

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 5391959, California 5332921 and other locations

  • Zorevunersen in Patients With Dravet Syndrome

    open to eligible people ages 2-17

    The purpose of the study is to evaluate the efficacy, safety, and tolerability of zorevunersen in Patients with Dravet syndrome.

    San Francisco 5391959, California 5332921 and other locations

  • LP352 in Children and Adults With Dravet Syndrome (DS)

    open to eligible people ages 2-65

    This (DEEp SEA Study) is a double-blind, randomized, placebo-controlled, multicenter study to investigate the efficacy, safety, and tolerability of LP352 in the treatment of seizures in children and adults with DS. The study consists of 3 main phases: Screening, Titration period, and Maintenance period, followed by a Taper period and Follow-Up. Participants will be randomized to LP352 or placebo. The total duration of the study will be approximately 24 months.

    San Francisco 5391959, California 5332921 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 hydrochloride (EPX-100) as adjunctive therapy in children and adult participants with Dravet syndrome (DS).

    San Francisco 5391959, California 5332921 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 5391959, California 5332921 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 5391959, California 5332921 and other locations

Our lead scientists for Dravet Syndrome research studies include .

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