Fenfluramine for Adult Dravet Patients

Dravet Syndrome Clinical Trial

Official title: Fenfluramine for the Treatment of Refractory Epilepsy in Adult Dravet Patients

Status Not yet recruiting

Clinical Trial 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

Clinical Trial Description

n/a

Related Conditions & MeSH terms

• Dravet Syndrome
• Epilepsies, Myoclonic
• Syndrome

• NCT number: NCT05560282
• Study type: Interventional
• Source: University Health Network, Toronto
• Contact: Sarah Selvadurai
• Phone: 4166035800
• Email: sarah.selvadurai2@uhn.ca
• Status: Not yet recruiting
• Phase: Phase 3
• Start date: December 10, 2022
• Completion date: January 31, 2024