Dravet Syndrome clinical trials at UCSF
5 in progress, 3 open to eligible people
ETX101 in Infants and Children With SCN1A-Positive Dravet Syndrome
open to eligible people ages 6 months to 17 years
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 1A), aged ≥48 months to <18 years (Part 1B), and aged ≥6 to <48 months (Part 2). Part 1A follows an open-label, dose-escalation design, Part 1B follows an open-label design, and Part 2 is a randomized, double-blind, sham delayed-treatment control study.
San Francisco, California 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, 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 hydrochloride (EPX-100) as adjunctive therapy in children and adult participants with Dravet syndrome (DS).
San Francisco, California and other locations
STK-001 for Patients With Dravet Syndrome
Sorry, in progress, not accepting new patients
Stoke Therapeutics is evaluating the long-term safety & tolerability of repeated doses of zorevunersen (STK-001) in patients with Dravet syndrome who previously participated in studies of zorevunersen. 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 Joseph Sullivan, MD Adam Numis.
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