View clinical trials related to Dravet Syndrome.
Filter by:EXPEDITION is a Phase 1/2 study in the UK to evaluate the safety and efficacy of ETX101 in participants with SCN1A-positive Dravet Syndrome aged 6 to < 48 months. The study follows and open-label, dose-escalation design.
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 patients with Developmental and Epileptic Encephalopathies (DEEs). The EAP study will allow continued treatment with LP352 for eligible patients diagnosed with treatment resistant DEEs who successfully completed an LP352 Clinical Trial (Enrollment by Invitation).
The primary purpose of this study is evaluate the safety and tolerability of fenfluramine hydrochloride (HCl) 0.2 to 0.8 mg/kg/day in infants 1 year to less than 2 years of age with Dravet syndrome.
WAYFINDER is a Phase 1/2 study in Australia to evaluate the safety and efficacy of ETX101 in participants with SCN1A-positive Dravet syndrome aged 36 to <84 months (3 to <7 years). The study follows an open-label, dose-escalation design.
This study will non-invasively obtain levels of GABA in the brain of children with SCN1A+DS and neurodeveloping children through evoked and induced cortical responses, correlate them with the BOLD responses, and with the levels of GABA in their blood.
To collect, preserve, and/or distribute annotated biospecimens and associated medical data to institutionally approved, investigator-directed biomedical research to discover and develop new treatments, diagnostics, and preventative methods for specific and complex conditions.
The objective of this study is to assess the long-term safety, tolerability, and efficacy of adjunctive therapy of LP352 in subjects with developmental and epileptic encephalopathies who completed participation in Study LP352-201.
Full Title: Fenfluramine for the treatment of refractory Epilepsy in Adult Dravet patients Short Title: Fenfluramine for Adult Dravet patients Clinical Phase: Phase III Sample Size: A total of 15 participants will be included in the study. Study Population: Adult patients (18 years and older) with drug-resistant epilepsy (maintained on their existing medications, with exception of cannabidiol) and genetically confirmed Dravet syndrome will be recruited to participate in the study. Accrual Period: 12 months Study Design: Open label, non-randomized and uncontrolled add-on trial in adults (18 years of age and older) residing in Ontario, with refractory motor seizures and maintained on their existing antiepileptic medications, with exception of cannabidiol. Study Duration: • Treatment period: 12 months Study duration: 28 months Study Agent/ Intervention/ Procedure: Name of study drug: fenfluramine (FINTEPLA) Dose and frequency: starting at 0.1 mg/kg twice daily, maximum 26 mg/day, in patients not taking concomitant stiripentol; starting at 0.1 mg/kg twice daily, maximum of 17 mg/day in patients taking concomitant stiripentol. All doses are divided to twice a day. Duration: Baseline phase: 4 weeks (no study drug) Titration phase: 2 weeks (if not taking stiripentol) to 3 weeks (if the patient is taking stiripentol) Treatment phase: 12 weeks Extension phase: up to 38 weeks, for patients who had at least a 50% decrease in seizure frequency Post-trial washout phase: 2 weeks (if not taking stiripentol) to 3 weeks (if the patient is taking stiripentol) Route of administration: Oral Efficacy and safety points of interest - Monthly convulsive seizure frequency (MCSF) reduction ≥ 50% - Improvement in motor function - Improvement in Cognition and Behavior - Improvement in Quality of Sleep - Improvement in Quality of life - Determination of Cardiovascular safety in adults - Responder analysis (≥25%, ≥75%, or 100% reduction in mean MCSF) - Longest period of seizure freedom - Number of Emergency room visits - Use of rescue medication (number of days in 28 day-periods) - Duration of post-ictal stage - Frequency of other seizure types - Body weight changes - Patient's global functioning prior to and after study (Clinical Global Impressions Scale) Trial registration: www.clinicaltrials.gov
The present study aims to collect data regarding the history of the disease, previous and current treatments, and the clinical status of Dravet patients during the 3 months prior to stiripentol initiation, the first 3 months on stiripentol and the last 3 months on stiripentol (irrespective of stiripentol discontinuation).
This is a prospective, observational study on approximately 70 Real World participants affected by LGS or DS, treated with Epidyolex® as prescribed in the summary of product characteristics. The eligible participants are expected to participate in the study for a duration of 56 weeks of treatment.