TerminatedPhase 3

Fenfluramine for Adult Dravet Patients

Stopped: Study recruitment was too slow, not enough funds to sustain it.

Canada7 participantsStarted 2023-01-11

Plain-language summary

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

Who can participate

Age range18 Years

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * 1\. Male and female patients 18 years of age and older. 2. Diagnosis of DS secondary to a genetically confirmed pathogenic variant. 3. Patients recruited from the Adult Epilepsy Genetics Clinic at Toronto Western Hospital. We will also accept referrals from other academic epilepsy centers across Ontario and that are referred to our program, making this a single site study. 4\. At least 4 convulsive seizures per month at the start of the study, despite having been treated with at least two different antiepileptic drugs (given concurrently or sequentially) for at least one year. 5\. At least 4 convulsive seizures per month during the prospective Baseline Phase (4 weeks). 6\. Able to provide written consent signed by study participant's caregiver prior to entering the study or undergoing any study procedures. 7\. Participant's caregivers should be able to record seizures and report adverse events for the full duration of the study. 8\. No evidence of non-compliance with ongoing ASM therapy. 9. Stable dose(s) of the same ASMs for one month prior to screening. 10. If participant has a vagal nerve stimulator or deep brain stimulator, it must have been implanted for at least six months prior to recruitment. Stimulator parameters may not be changed for at least one month prior to screening or thereafter during the study. Magnet use will be allowed but must be documented throughout the study. 11\. Patient's caregivers agree that patient will no…

What they're measuring

Seizure reduction

Timeframe: 16 week

Trial details

NCT IDNCT05560282

SponsorUniversity Health Network, Toronto

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2025-05-13