NCT05232630

Fenfluramine for the Treatment of Different Types of Developmental and Epileptic Encephalopathies: a Pilot Trial Exploring Epileptic and Non-epileptic Outcomes

• Status: Unknown
• Phase: Phase 4
• Study type: Interventional
• Enrollment: 20 (estimated)
• Sponsor: Hospital Ruber Internacional · Academic / Other
• Sex: All
• Age: 2 Years – 35 Years
• Healthy volunteers: Not accepted

Summary

This study is a pilot non-controlled clinical trial with adjunctive fenfluramine for the treatment of five different types of developmental and epileptic encephalopathies (DEEs) focused on epileptic and "non-epileptic outcomes": SYNGAP1 and STXBP1 encephalopathies, inv-dup(15) encephalopathy, multifocal or bilateral malformations of cortical development, and continuous spikes and waves during sleep. The main goal is to assess changes in seizure frequency comparing before and after treatment with fenfluramine in five specific types of developmental and epileptic encephalopathies (DEEs). Secondary objectives of this study are the analysis of changes in seizure intensity and duration, and "non-epileptic outcomes" such as variations in cognitive activity, level of alertness, impulsivity/self-control, gait stability and other alterations that might be detected during the interview and physical examination.

Conditions

• Refractory Epilepsy
• SYNGAP1 Encephalopathy
• STXBP1 Encephalopathy With Epilepsy
• Inv Dup(15) Encephalopathy
• Multifocal or Bilateral Malformations of Cortical Development
• Continuous Spike and Waves During Slow Sleep

Interventions

Type	Name	Description
DRUG	Fenfluramine	Administration of fenfluramine. Fenfluramine treatment dose: Between 0.2 and 0.7 mg/kg/day if no concomitant Stiripentol (STP), maximum dose: 40 mg/day \[or 0.5 mg/kg/day, maximum 30 mg/day, for subjects taking concomitant STP\]. Dosing will be started with 0.1mg/day per one week, then 0.2mg/kg/day per one week, then as investigator clinical decision-making, up to 0.4, 0.6 or 0.7mg/kg/day, with a maximum of 0.2mg/kg/day escalation every week. Visits: There will be four visits; (visit 1) screening; (visit 2) treatment initiation, +2 weeks; (visit 3, telematic) +8 weeks; (visit 4) +14 weeks.

Timeline

• Start date: 2022-10-20
• Primary completion: 2025-02-20
• Completion: 2025-06-20
• First posted: 2022-02-10
• Last updated: 2024-02-28

Locations

1 site across 1 country: Spain