Dravet Syndrome clinical trials at University of California Health
9 in progress, 4 open to eligible people
- 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. - at UCSF 
- 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. - at UCSF 
- 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. - at UCLA UCSF 
- 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). - at UC Irvine UCSF 
- 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 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. - at UCSF 
- 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. - at UCSF 
- Access Program With Lorcaserin For The Treatment of Dravet Syndrome and Other Refractory Epilepsies- Sorry, not accepting new patients - The primary purpose of this study is to provide continued access of lorcaserin to participants with Dravet syndrome and other refractory epilepsies. - at UCLA 
- Intermediate-Size Expanded Access Protocol (EAP) for LP352- Sorry, not accepting new patients - This is an intermediate-size expanded access program (EAP) study. The purpose of this EAP is to provide continued access to LP352, an investigational drug product being investigated in participants with DEEs. The EAP study will allow continued treatment with LP352 for eligible participants diagnosed with treatment resistant DEEs who successfully completed an LP352 Clinical Trial (Enrollment by Invitation) or an immediate family member who has the exact same gene mutation resulting in the same DEE epilepsy syndrome phenotype or a patient who previously participated in the lorcaserin EAP. - at UCLA UCSF 
- Treatment of Dravet Syndrome With Fenfluramine (Expanded Access Protocol)- Sorry, not accepting new patients - The purpose of this research study is to (1) provide access to fenfluramine for patients with intractable epilepsy associated with Dravet syndrome, and (2) evaluate the safety of fenfluramine. - at UCLA 
Our lead scientists for Dravet Syndrome research studies include Shaun Hussain, MD Joseph Sullivan.
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