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Add-on Therapy With Low Dose Fenfluramine in Lennox Gastaut Epilepsy (FFA-LGS)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02655198
Recruitment Status : Active, not recruiting
First Posted : January 13, 2016
Last Update Posted : July 28, 2020
Sponsor:
Collaborator:
Zogenix, Inc.
Information provided by (Responsible Party):
Lieven Lagae, KU Leuven

Brief Summary:
In this trial, the potential anti-epileptic effect of low dose fenfluramine in Lennox Gastaut epilepsy will be studied. An exploratory dose finding add-on trial is proposed. At baseline and at the end of the study, ECG and ultrasound of the heart will be performed as part of the safety follow up.

Condition or disease Intervention/treatment Phase
Epilepsy Lennox Gastaut Syndrome Drug: Fenfluramine Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 13 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Add-on Therapy With Low Dose Fenfluramine in Lennox Gastaut Epilepsy
Study Start Date : January 2016
Actual Primary Completion Date : September 2018
Estimated Study Completion Date : December 2020


Arm Intervention/treatment
Experimental: fenfluramine

Experimental : one armed open label study :

Add-on fenfluramine in refractory Lennox Gastaut patients. Starting dose 0.2mg/kg/day. In non-responders (<50% seizure frequency decrease), dose will be uptitrated every 4 weeks from 0,2 to 0,4 and max 0,8 mg/kg/day (max 30 mg). Total duration study and max exposure to the drug 20 weeks

Drug: Fenfluramine
study of efficacy and safety of add-on fenfluramine at different dosages in refractory Lennox Gastaut patients : 0.2 - 0.4 and 0.8 mg/kg/day (max 30 mg).




Primary Outcome Measures :
  1. Efficacy of add-on FFA in Lennox Gastaut epilepsy: Number of responders and seizure free patients at each FFA dosage (0,2 or 0,4 or 0,8 mg/kg/day) [ Time Frame: up to 20 weeks ]

Secondary Outcome Measures :
  1. Seizure frequency change per patient and per major seizure type (Tonic Clonic Seizures (TCS), Tonic Seizures (TS), Atonic Seizures (AS), Focal Seizures (FS)) [ Time Frame: 20 weeks ]
  2. Adverse events (cardiac and general) [ Time Frame: 20 weeks ]
  3. Sleep quality : 10 point scale instrument to score sleep quality [ Time Frame: 20 weeks ]
  4. CGI (clinical global impression) scale at last visit , by patient/caregiver and treating physician [ Time Frame: 20 weeks ]


Information from the National Library of Medicine

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Ages Eligible for Study:   3 Years to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion criteria

Electro-clinical epilepsy syndrome compatible with Lennox Gastaut syndrome:

  • Minimum requirements (based on ILAE epilepsydiagnosis.org):

    • Multiple seizure types including in any case tonic seizures
    • EEG shows slow spike waves and abnormal background
    • Abnormal cognitive development
    • MRI compatible with Lennox Gastaut epilepsy : no progressive disease
  • Drug resistant:

    • at least 4 documented seizures in the last 4 weeks before inclusion (minimum 4 seizures in at least 2 separate weeks) Seizure types eligible for inclusion are : generalized tonic-clonic seizures GTC , tonic seizures TS , atonic seizures AS or clearly recognizable focal seizures FS.
    • on >= 2 AEDs (including VNS) during the 4 weeks before inclusion (no changes in treatment before inclusion and during the trial)
  • Age between 3 and 18 years
  • Subject is male or non-pregnant, non-lactating female. Female subjects of childbearing potential must not be pregnant or breast-feeding. Female subjects of childbearing potential must have a negative urine pregnancy test. Subjects of childbearing or child-fathering potential must be willing to use medically acceptable forms of birth control, which includes abstinence, while being treated on this study and for 90 days after the last dose of study drug.

Exclusion Criteria

  • Known clinical cardiovascular abnormalities (including valvular problems, shunts, pulmonary hypertension, exercise intolerance)
  • Any cardiac ultrasound/ECG abnormalities at baseline
  • Weight below percentile 3 for age at baseline
  • Subject is receiving concomitant therapy with: centrally-acting anorectic agents; monoamine oxidase inhibitors; any centrally-acting compound with clinically appreciable amount of serotonin agonist or antagonist properties, including serotonin reuptake inhibition; atomoxetine, or other centrally-acting noradrenergic agonist; cyproheptadine, and/or cytochrome P450 (CYP) 2D6/3A4/2B6 inhibitors/substrates.
  • Subject is unwilling to refrain from large or daily servings of grapefruits and/or Seville oranges, and their juices beginning with the Baseline Period and throughout the study.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02655198


Locations
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Belgium
University Hospitals UZ Leuven
Leuven, Belgium, 3000
Sponsors and Collaborators
KU Leuven
Zogenix, Inc.
Investigators
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Principal Investigator: Lieven G Lagae, MD, PhD Katolieke Universiteit Leuven, University Hospitals Gasthuisberg
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
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Responsible Party: Lieven Lagae, Professor Paediatric Neurology, KU Leuven
ClinicalTrials.gov Identifier: NCT02655198    
Other Study ID Numbers: S58545
First Posted: January 13, 2016    Key Record Dates
Last Update Posted: July 28, 2020
Last Verified: July 2020
Keywords provided by Lieven Lagae, KU Leuven:
refractory childhood epilepsy
Lennox Gastaut syndrome
fenfluramine
anti-epileptic treatment
Additional relevant MeSH terms:
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Epilepsy
Lennox Gastaut Syndrome
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Epileptic Syndromes
Genetic Diseases, Inborn
Fenfluramine
Serotonin Uptake Inhibitors
Neurotransmitter Uptake Inhibitors
Membrane Transport Modulators
Molecular Mechanisms of Pharmacological Action
Neurotransmitter Agents
Serotonin Agents
Physiological Effects of Drugs