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

NCT05232630 · Status: RECRUITING · Phase: PHASE4 · Type: INTERVENTIONAL · Enrollment: 20

Last updated 2024-02-28

No results posted yet for this study

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

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.

Sponsors & Collaborators

  • Zogenix, Inc.

    collaborator INDUSTRY

  • Hospital Ruber Internacional

    lead OTHER

Principal Investigators

  • Antonio Gil-Nagel, MD, PHD · Hospital Ruber Internacional

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

Min Age
2 Years
Max Age
35 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-10-20
Primary Completion
2025-02-20
Completion
2025-06-20

Countries

  • Spain

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05232630 on ClinicalTrials.gov