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for people ages 2–18 (full criteria)
at San Francisco, California and other locations
study started
estimated completion:



The primary purpose of this study is to evaluate the safety, tolerability and efficacy of a single dose of ZX008 (Fenfluramine Hydrochloride) when added to standard of care and when added to Adjunctive Antiepleptic therapy to Stiripentol treatment in children and young adults with Dravet Syndrome

Official Title

A Multicenter, 2-Cohort Trial to First Assess the Pharmacokinetic and Safety Profile of a Single Dose of ZX008 (Fenfluramine Hydrochloride) Oral Solution When Added to Standard of Care , Followed by a Randomized, Double-blind, Placebo-controlled Parallel Group Evaluation of the Efficacy, Safety, and Tolerability of ZX008 as Adjunctive Antiepileptic Therapy to Stiripentol Treatment in Children and Young Adults With Dravet Syndrome


This is a multicenter, two-cohort trial to assess the pharmacokinetic and safety profile of a single dose of ZX008 (fenfluramine hydrochloride) oral solution when added to Dravet syndrome treatment regimen containing VPA and CLB, with or without STP (Cohort 1), followed by a randomized, double-blind, placebo-controlled parallel group evaluation of the efficacy, safety, and tolerability of ZX008 as adjunctive therapy for seizures in children and young adults with Dravet syndrome (Cohort 2). Cohort 2 will not be dosed until the PK and safety data from Cohort 1 have been collected and evaluated. The PK and safety data from Cohort 1 will determine the dose of ZX008 to be used in Cohort 2.


Dravet Syndrome seizure tonic clonic epilepsy myoclonic encephalopathy Syndrome Epilepsies, Myoclonic Pharmaceutical Solutions Fenfluramine


For people ages 2–18

Key Inclusion Criteria:

  • Subject must be male or non-pregnant, non-lactating female, age 2 to 18 years(inclusive)
  • Subject must have documented medical history to support a clinical diagnosis of Dravet syndrome, where convulsive seizures are not completely controlled by current antiepileptic drugs.
  • Subject must be receiving a therapeutically relevant and stable dose of CLB, VP, and STP for at least 4 weeks prior to screening and are expected to remain stable throughout the study (Cohort 2 only).
  • Subject must be receiving a stable dose of CLB and VPA, administered twice daily, to be eligible for Dose Regimen 1 and 2 or subject must be receiving a stable dose of CLB, VPA, and STP, administered twice daily, to be eligible for Dose Regimen 3 (Cohort 1 only).

Key Exclusion Criteria:

  • Subject has a known hypersensitivity to fenfluramine or any of the excipients in the study medication.
  • Subject has pulmonary arterial hypertension.
  • Subject has a current or past history of cardiovascular or cerebrovascular disease,such as cardiac valvulopathy, myocardial infarction or stroke.
  • Subject has a current or recent history of anorexia nervosa, bulimia, or depression within the prior year that required medical treatment or psychological treatment for a duration greater than 1 month.
  • Subject has a current or past history of glaucoma.
  • 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 re-uptake inhibition; triptans, atomoxetine, or other centrally-acting noradrenergic agonist; cyproheptadine, and/or CYP 2D6/3A4/2B6 inhibitors/substrates.
  • Subject is currently taking carbamazepine, oxcarbamazepine, eslicarbazepine,phenobarbital, or phenytoin, or has taken any of these within the past 30 days, as maintenance therapy.
  • Subject has a positive result on urine THC Panel or whole blood CBD at the Screening Visit.
  • Subject has a clinically significant condition, or has had clinically relevant symptoms or a clinically significant illness in the 4 weeks prior to the Screening Visit, other than epilepsy, that would negatively impact study participation,collection of study data, or pose a risk to the subject.


  • University of California San Francisco
    San Francisco, California, 94143, United States
  • British Columbia Children'S Hospital
    Vancouver, British Columbia, V6H3V4, Canada
  • The Children'S Hospital Colorado
    Aurora, Colorado, 80045, United States
  • Mayo Clinic
    Rochester, Minnesota, 55905, United States
  • Ann & Robert Lurie Children'S Hospital of Chicago
    Chicago, Illinois, 60611, United States
  • Centre Hospitalier Universitaire Sainte-Justine
    Montreal, Quebec, H3T 1C5, Canada
  • Institute of Neurosciences Queens Elizabeth University
    Glasgow, G51 4TF, United Kingdom
  • Alder Hey Children'S Nhs Foundation Trust
    Liverpool, L12 2AP, United Kingdom
  • Evelina Hospital
    London, SE1 7EH, United Kingdom
  • Great Ormonnd Street Hospital For Children Nhs Foundation Trust
    London, WC1N 3JH, United Kingdom
  • Kempenhaeghe
    Heeze, 5590 Ab, Netherlands
  • Stichting Epilepsie Instellingen Nederland
    Zwolle, 8025 BV, Netherlands
  • Krankenhaus Mara Ggmbh, Epilepsiezentrum Bethel
    Bielefeld, 33617, Germany
  • Universitätsklinikum Schleswig-Holstein, Klinik Für Neuropädiatrie
    Kiel, 24105, Germany
  • Kleinwachau Sächsisches Epilepsiezentrum Radeberg Gemeinnützige Gmbh
    Radeberg, 1454, Germany
  • Chu Amiens Picardie Service de Neurologie Pediatrique
    Amiens, 80054, France
  • CHU DE BORDEAUX Service De Pédiatrie Médicale
    Bordeaux, 33000, France
  • CHRU LILLE Centre Hospitalier Régional Universitaire De Lille 2
    Lille, 59037, France
  • HÔPITAL FEMME-MÈRE-ENFANT Hôpital Femme-mère-enfant Service De Neurologie Pédiatrique
    Lyon, 69500, France
  • Hôpital La Timone, Service de Neurologie Pédiatrique
    Marseille, 13385, France
  • Hôpital Necker
    Paris, 75743, France
  • Hôpital Robert Debré
    Paris, 75743, France
    Strasbourg Cedex, F-67098, France
    Toulouse Cedex 9, 31059, France
  • Hopital Denfants Chru de Nancy
    Vandoeuvre Les Nancy Cedex, 54511, France
  • Hospital Sant Joan de Déu
    Barcelona, 08950, Spain
  • Hospital Ruber Internacional Primera Planta Servicio de Neurologia
    Madrid, 28034, Spain
  • Clinica Universitaria de Navarra
    Pamplona, 31008, Spain


in progress, not accepting new patients
Start Date
Completion Date
Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc.
Phase 3
Lead Scientist
Joseph Sullivan
Study Type
Last Updated
January 4, 2018