Dravet Syndrome clinical trials at UCSF
3 in progress, 2 open to eligible people
Dravet syndrome is a genetic epilepsy marked by frequent, prolonged seizures in young children. UCSF is enrolling infants and children in an early safety and efficacy study of a new medication. UCSF is also involved in a randomized, placebo-controlled study of an oral therapy.
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
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
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
Our lead scientists for Dravet Syndrome research studies include Ernesto Gonzalez-Giraldo, MD Adam Numis.
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