University Health Network, Toronto
Danielle Andrade
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
Driven Syndrome
Dravet Syndrome, Intractable
Fenfluramine
Phase 3
Study Type : | Interventional |
Estimated Enrollment : | 15 participants |
Masking : | None (Open Label) |
Primary Purpose : | Treatment |
Official Title : | Fenfluramine for the Treatment of Refractory Epilepsy in Adult Dravet Patients |
Actual Study Start Date : | December 10, 2022 |
Estimated Primary Completion Date : | January 31, 2024 |
Estimated Study Completion Date : | January 31, 2024 |
Arm | Intervention/treatment |
---|---|
Experimental: Fenfluramine Fenfluramine, oral, starting at 0.1mg/kg twice daily, maximum 26mg/day in patients not taking concomitant stiripentol starting at 0.1mg/kg twice daily, maximum 17mg/day in patients taking concomitant stiripentol |
Drug: Fenfluramine |
Ages Eligible for Study: | 18 Years |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
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Not yet recruiting
Toronto Western Hospital
Toronto, Ontario, Canada, M5T 2S8