NCT05560282

Fenfluramine for Adult Dravet Patients

Fenfluramine for the Treatment of Refractory Epilepsy in Adult Dravet Patients

• Status: Unknown
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 15 (estimated)
• Sponsor: University Health Network, Toronto · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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

Conditions

• Dravet Syndrome
• Dravet Syndrome, Intractable

Interventions

Type	Name	Description
DRUG	Fenfluramine	FINTEPLA oral solution contains 2.2 mg/mL fenfluramine, equivalent to 2.5 mg/mL of the hydrochloride salt. The active ingredient, fenfluramine hydrochloride, is designated chemically as N-ethyl-α- methyl-3-(trifluoromethyl) phenethylamine hydrochloride. Fenfluramine hydrochloride is a white to off-white crystalline solid. The pKa of fenfluramine is 10.2. FINTEPLA is a clear, colorless solution, pH 5.

Timeline

• Start date: 2022-12-10
• Primary completion: 2024-01-31
• Completion: 2024-01-31
• First posted: 2022-09-29
• Last updated: 2022-11-09

Locations

1 site across 1 country: Canada

Regulatory

• FDA-regulated drug study