Skip to main content

Summary

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

Description

Summary

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

Details

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.

Keywords

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

Eligibility

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.

Locations

  • University of California San Francisco accepting new patients
    San Francisco, California, 94143, United States
  • British Columbia Children'S Hospital not yet accepting patients
    Vancouver, British Columbia, V6H3V4, Canada
  • The Children'S Hospital Colorado accepting new patients
    Aurora, Colorado, 80045, United States
  • Mayo Clinic accepting new patients
    Rochester, Minnesota, 55905, United States
  • Ann & Robert Lurie Children'S Hospital of Chicago accepting new patients
    Chicago, Illinois, 60611, United States
  • Centre Hospitalier Universitaire Sainte-Justine not yet accepting patients
    Montreal, Quebec, H3T 1C5, Canada
  • Institute of Neurosciences Queens Elizabeth University not yet accepting patients
    Glasgow, G51 4TF, United Kingdom
  • Alder Hey Children'S Nhs Foundation Trust not yet accepting patients
    Liverpool, L12 2AP, United Kingdom
  • Evelina Hospital not yet accepting patients
    London, SE1 7EH, United Kingdom
  • Great Ormonnd Street Hospital For Children Nhs Foundation Trust not yet accepting patients
    London, WC1N 3JH, United Kingdom
  • Kempenhaeghe not yet accepting patients
    Heeze, 5590 Ab, Netherlands
  • Stichting Epilepsie Instellingen Nederland accepting new patients
    Zwolle, 8025 BV, Netherlands
  • Chu Amiens Picardie Service de Neurologie Pediatrique accepting new patients
    Amiens, 80054, France
  • CHU DE BORDEAUX Service De Pédiatrie Médicale accepting new patients
    Bordeaux,, 33000, France
  • CHRU LILLE Centre Hospitalier Régional Universitaire De Lille 2 accepting new patients
    Lille, 59037, France
  • HÔPITAL FEMME-MÈRE-ENFANT Hôpital Femme-mère-enfant Service De Neurologie Pédiatrique not yet accepting patients
    Lyon Bron, 69500, France
  • Hôpital La Timone, Service de Neurologie Pédiatrique accepting new patients
    Marseille, 13385, France
  • Hôpital Necker accepting new patients
    Paris, 75743, France
  • Hôpital Robert Debré accepting new patients
    Paris, 75743, France
  • CENTRE REFERENT DES EPILEPSIES Hopital De Hautpierre not yet accepting patients
    Strasbourg Cedex, F-67098, France
  • HÔPITAL DES ENFANTS Pédiatrie accepting new patients
    Toulouse Cedex 9, 31059, France
  • Hopital Denfants Chru de Nancy accepting new patients
    Vandoeuvre Les Nancy Cedex, 54511, France
  • Krankenhaus Mara Ggmbh, Epilepsiezentrum Bethel not yet accepting patients
    Bielefeld, 33617, Germany
  • Universitätsklinikum Schleswig-Holstein, Klinik Für Neuropädiatrie not yet accepting patients
    Kiel, 24105, Germany
  • Kleinwachau Sächsisches Epilepsiezentrum Radeberg Gemeinnützige Gmbh not yet accepting patients
    Radeberg, 1454, Germany
  • Hospital Sant Joan de Déu not yet accepting patients
    Barcelona, 08950, Spain
  • Hospital Ruber Internacional Primera Planta Servicio de Neurologia not yet accepting patients
    Madrid, 28034, Spain
  • Clinica Universitaria de Navarra not yet accepting patients
    Pamplona, 31008, Spain

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc.
ID
NCT02926898
Phase
Phase 3
Lead Scientist
Joseph Sullivan
Study Type
Interventional
Last Updated
September 9, 2017
I’m interested in this study!