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