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An Open-Label Extension Trial to Assess the Long-Term Safety of ZX008 (Fenfluramine Hydrochloride HCl) Oral Solution in Children and Young Adults With Dravet Syndrome

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: NCT02823145
Recruitment Status : Enrolling by invitation
First Posted : July 6, 2016
Last Update Posted : January 6, 2020
Sponsor:
Information provided by (Responsible Party):
Zogenix, Inc. ( Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc. )

Brief Summary:
This is an international, multicenter, open-label, long-term safety study of ZX008 in subjects with Dravet syndrome.

Condition or disease Intervention/treatment Phase
Dravet Syndrome Drug: ZX008 (Fenfluramine Hydrochloride) Phase 3

Detailed Description:
This is an international, multicenter, open-label, long-term safety study of ZX008 in pediatric and young adult subjects with Dravet syndrome who participated in one of the core studies (ZX008-1501 and ZX008-1502) and are candidates for continuous treatment for an extended period of time. This trial will consist of a 36-month Open-Label Extension (OLE) Treatment Period and a 2-week Post-Dosing Period.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 340 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open-Label Extension Trial to Assess the Long-Term Safety of ZX008 (Fenfluramine Hydrochloride HCl) Oral Solution as an Adjunctive Therapy in Children and Young Adults With Dravet Syndrome
Study Start Date : June 2016
Estimated Primary Completion Date : December 2020
Estimated Study Completion Date : December 2020


Arm Intervention/treatment
Experimental: ZX008
ZX008 is supplied as an oral solution in a concentration of 2.5 mg/mL. Subjects will be titrated to an effective dose beginning with 0.2 mg/kg/day (maximum: 30 mg/day).
Drug: ZX008 (Fenfluramine Hydrochloride)



Primary Outcome Measures :
  1. Long-term safety and tolerability as measured by treatment emergent adverse events, including clinical labs, vital signs, and examination findings. [ Time Frame: Pre-baseline Up to 156 weeks ]
    Sensitivity of the key efficacy analysis will be assessed for changes in dose or type of concomitant AED medications


Secondary Outcome Measures :
  1. Proportion of subjects who achieve reduction from baseline in convulsive seizure frequency [ Time Frame: Baseline up to 156 weeks ]
  2. The longest interval between convulsive seizures will be calculated for each subject over the entire open-label treatment period. [ Time Frame: Up to 156 weeks ]


Information from the National Library of Medicine

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Ages Eligible for Study:   2 Years to 35 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Key Inclusion Criteria:

  • Male or non-pregnant, non-lactating female, age 2 to 18 years, inclusive as of the day of the core study Screening Visit.
  • Satisfactory completion of the core study in the opinion of the investigator and the sponsor.
  • Subjects who are >18 to ≤35 years of age at the time of screening and did not participate in one of the core studies may be eligible for participation.
  • A documented medical history to support a clinical diagnosis of Dravet syndrome, where convulsive seizures are not completely controlled by current antiepileptic drugs.
  • Parent/caregiver is willing and able to be compliant with diary completion, visit schedule and study drug accountability.
  • Subject's parent/caregiver has been compliant with diary completion during the core study, in the opinion of the investigator (eg, at least 90% compliant).

Key Exclusion Criteria:

  • Current or past history of cardiovascular or cerebrovascular disease, myocardial infarction or stroke.
  • Current cardiac valvulopathy or pulmonary hypertension that is clinically significant and warrants discontinuation of study medication.
  • Current or past history of glaucoma.
  • Moderate or severe hepatic impairment.
  • Receiving concomitant therapy with: centrally-acting anorectic agents; monoamineoxidase 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.
  • Currently taking carbamazepine, oxcarbamazepine, eslicarbazepine, phenobarbital, or phenytoin, or has taken any of these within the past 30 days, as maintenance therapy.
  • A clinically significant condition, or has had clinically relevant symptoms or a clinically significant illness at Visit 1, other than epilepsy, that would negatively impact study participation, collection of study data, or pose a risk to the subject.

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): NCT02823145


Locations
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United States, Arizona
Phoenix Children's Hospital
Phoenix, Arizona, United States, 85016
Center for Neurosciences - Tucson
Tucson, Arizona, United States, 85718
United States, California
Children's Hospital of Orange County
Orange, California, United States, 92868
Rady Children's Hospital San Diego
San Diego, California, United States, 92123
University of California San Francisco
San Francisco, California, United States, 94158
United States, Colorado
The Children's Hospital Colorado
Aurora, Colorado, United States, 80045
United States, Florida
NW FL Clinical Research Group, LLC
Gulf Breeze, Florida, United States, 32561
Miami Children's Hospital Brain Institute
Miami, Florida, United States, 33155
Neurology and Epilepsy Research Center
Orlando, Florida, United States, 32819
United States, Georgia
Clinical Integrative Research Center of Atlanta, Panda Neurology
Atlanta, Georgia, United States, 30328
United States, Illinois
Ann and Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States, 60611
United States, Massachusetts
Massachusetts General Hospital
Boston, Massachusetts, United States, 02114
Boston Children's Hospital
Boston, Massachusetts, United States, 02115
United States, Michigan
Children's Hospital Of Michigan
Detroit, Michigan, United States, 48201
United States, Minnesota
Mayo Clinic
Rochester, Minnesota, United States, 55905
Minnesota Epilepsy Group, P.A.
Saint Paul, Minnesota, United States, 55102
United States, New Jersey
Institute of Neurology and Neurosurgery at St. Barnabus
Livingston, New Jersey, United States, 07039
United States, Ohio
University Hospitals Cleveland Medical Center
Cleveland, Ohio, United States, 44103
United States, Pennsylvania
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
United States, Tennessee
Le Bonheur Children's Hospital
Memphis, Tennessee, United States, 38103
United States, Texas
Cook Children's Medical Center
Fort Worth, Texas, United States, 76104
United States, Utah
University of Utah
Salt Lake City, Utah, United States, 84113
United States, Washington
Seattle Children's Hospital
Seattle, Washington, United States, 98105
MultiCare Institute for Research & Innovation
Tacoma, Washington, United States, 98405
Australia
Melbourne Brain Centre Austin Hospital
Heidelberg, Australia
Children's Health Queensland Hospital and Health Service at Lady Cilento Children's Hospital
South Brisbane, Australia
The Children's Hospital Westmead Dept. of Neurology and Neurosurgery
Westmead, Australia
Belgium
Universitair Ziekenhuis Antwerpen
Edegem, Belgium, 2650
Canada
Centre Hospitalier Universitaire Sainte-Justine
Montréal, Canada
British Columbia Children's Hospital
Vancouver, Canada
Denmark
Danish National Epilepsy Centre
Dianalund, Denmark
France
Chu Amiens Picardie Service de Neurologie Pediatrique
Amiens, France
Chu De Bordeaux Service De Pédiatrie Médicale
Bordeaux, France
CHRU Lille Antenne Pédiatrique Du Cic - Hôpital Jeanne De Flandre
Lille, France
HOPITAL DEL LA TIMONE - HOPITAL HENRI GASTAUT Hôpital De La TimoneNeurologie Pédiatrique Pneumologie Pédiatrique Et Médecine Infantile
Marseille, France
HÔPITAL ROBERT DEBRÉ Pôle: Pédiatrie Médicale Service : Neurologie Et Maladies Métaboliques
Paris, France
Hôpital Universitaire Necker-Enfants Malades Service de neurologie pédiatrique Centre de référence épilepsies rares (CReER)
Paris, France
Germany
DRK Kliniken Berlin - Westend Epilepsiezentrum / Neuropaediatrie
Berlin, Germany
Krankenhaus Mara Epilepsie-Zentrum Bethel
Bielefeld, Germany
Universitaetsklinikum Freiburg Zentrum fuer Kinder- und Jugendmedizin
Freiburg, Germany
Universitaetsklinikum Jena Klinik fuer Kinder- und Jugendmedizin Neuropaediatrie
Jena, Germany
Universitaetsklinikum Schleswig-Holstein Campus Kiel Klinik fuer Neuropaediatrie
Kiel, Germany
Kleinwachau Saechsisches Epilepsiezentrum Radeberg gemeinnuetzige GmbH
Radeberg, Germany
Universitaetsklinik fuer Kinder- und Jugendmedizin Abteilung III
Tübingen, Germany
Schoen Klinik Vogtareuth Neuropaediatrie und Neurologische Rehabilitation, Epilepsiezentrum fuer Kinder und Jugendlische, Tagesklinik fuer Neuropaediatrie
Vogtareuth, Germany
Italy
Istituto Pediatrico Giannina Gaslini Dipartimento di Neurologia
Genova, Italy
A.O. Carlo Poma
Mantova, Italy
Istituto Neurologica Carlo Besta
Milano, Italy
Ospedale Fatebenefratelli e Oftalmico
Milano, Italy
Policlinico A . Gemelli
Roma, Italy
U.O. Neurologia Dipartimento di Neuroscienze Ospedale Pediatrico Bambino Gesù
Roma, Italy
AOU Anna Meyer Clinica di Neurologia Pediatrica
Verona, Italy
Ospedale Civile Maggiore di Borgo Trento - Ospedale della Donna e del Bambino
Verona, Italy
Netherlands
Kempenhaeghe
Heeze, Netherlands
Stichting Epilepsie Instellingen Nederland
Zwolle, Netherlands
Spain
Hospital Sant Joan de Déu
Barcelona, Spain
Hospital Ruber Internacional Primera Planta Servicio de Neurologia
Madrid, Spain
Clinica Universitaria de Navarra Fase 4. Segunda planta, Consulta de Pediatria
Pamplona, Spain
United Kingdom
Alder Hey Hospital
West Derby, Liverpool, United Kingdom, L12 2AP
Great Ormond Street Hospital for Children NHS Foundation Trust
Holborn, London, United Kingdom, WC1N 3JH
Birmingham Children Hospital NHS Foundation Trust
Birmingham, United Kingdom
Institute of Neurosciences Queen Elizabeth University Hospital
Glasgow, United Kingdom
Evelina Hospital
London, United Kingdom
Sheffield Children's Hospital
Sheffield, United Kingdom
Sponsors and Collaborators
Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc.
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Responsible Party: Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc.
ClinicalTrials.gov Identifier: NCT02823145    
Other Study ID Numbers: ZX008-1503
First Posted: July 6, 2016    Key Record Dates
Last Update Posted: January 6, 2020
Last Verified: January 2020
Keywords provided by Zogenix, Inc. ( Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc. ):
seizure
tonic clonic
epilepsy
myoclonic
encephalopathy
Additional relevant MeSH terms:
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Epilepsies, Myoclonic
Spasms, Infantile
Syndrome
Disease
Pathologic Processes
Epilepsy, Generalized
Epilepsy
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Epileptic Syndromes
Fenfluramine
Serotonin Uptake Inhibitors
Neurotransmitter Uptake Inhibitors
Membrane Transport Modulators
Molecular Mechanisms of Pharmacological Action
Neurotransmitter Agents
Serotonin Agents
Physiological Effects of Drugs