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A Study to Investigate the Efficacy and Safety of ZX008 (Fenfluramine Hydrochloride) as an Adjunctive Therapy in Children and Adults With Lennox-Gastaut 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: NCT03355209
Recruitment Status : Active, not recruiting
First Posted : November 28, 2017
Last Update Posted : June 18, 2021
Sponsor:
Information provided by (Responsible Party):
Zogenix, Inc. ( Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc. )

Brief Summary:
This is a two-part, multicenter, double-blind, parallel-group, placebo controlled study to evaluate the effect of ZX008 when used as adjunctive therapy for the treatment of uncontrolled seizures in children and adults with Lennox-Gastaut syndrome (LGS).

Condition or disease Intervention/treatment Phase
Lennox Gastaut Syndrome Drug: ZX008 0.2 or 0.8 mg/kg/day Drug: Matching Placebo Phase 3

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 296 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description: Part 1: Double-Blind ZX008 - (0.2 mg/kg/day or 0.8mg/kg/day) or Placebo and Part 2: Open-Label
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Masking Description: Part 1: Double-Blind Part 2: Open-Label
Primary Purpose: Treatment
Official Title: A Two-Part Study of ZX008 in Children and Adults With Lennox-Gastaut Syndrome (LGS); Part 1: A Randomized, Double-blind, Placebo-controlled Trial of Two Fixed Doses of ZX008 (Fenfluramine Hydrochloride) Oral Solution as Adjunctive Therapy for Seizures in Children and Adults With LGS, Followed by Part 2: An Open-label Extension to Assess Long-Term Safety of ZX008 in Children and Adults With LGS
Actual Study Start Date : November 27, 2017
Estimated Primary Completion Date : March 2024
Estimated Study Completion Date : March 2024

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Seizures

Arm Intervention/treatment
Experimental: ZX008 0.2 or 0.8 mg/kg/day
Part 1: ZX008 is supplied as an oral solution. Subjects will be randomized to receive 1 of 2 doses of ZX008 0.2 mg/kg/day or 0.8 mg/kg/day.
Drug: ZX008 0.2 or 0.8 mg/kg/day
ZX008 drug product is an oral aqueous solution of fenfluramine hydrochloride. The product is sugar free and is intended to be compatible with a Ketogenic Diet.

Placebo Comparator: Matching Placebo
Part 1: Matching ZX008 placebo is supplied as an oral solution.
Drug: Matching Placebo
Placebo will be administered twice a day (BID) in equally divided doses.
Other Name: Placebo Comparator

Experimental: Open-Label
Part 2: ZX008 is supplied as an oral solution. Study medication will be administered twice a day (BID) in equally divided doses.
Drug: ZX008 0.2 or 0.8 mg/kg/day
ZX008 drug product is an oral aqueous solution of fenfluramine hydrochloride. The product is sugar free and is intended to be compatible with a Ketogenic Diet.




Primary Outcome Measures :
  1. Part 1: Change from baseline in frequency of seizures that result in drops in subjects receiving ZX008 compared to placebo [ Time Frame: Up to 20 weeks maintenance and taper period (T+M) ]
    Parent/caregiver seizure diary record will be used to assess frequency, type and duration of seizure activity


Secondary Outcome Measures :
  1. Part 2: Adverse events and related safety parameters in subjects receiving ZX008 compared to placebo [ Time Frame: Up to 12 months open label ]
    Safety and tolerability evaluated by reported adverse events, laboratory parameters, physical and neurological examination, vital signs, electrocardiograms, echocardiograms, and body weight



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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 35 years, inclusive as of the day of the Screening Visit.
  • Clinical diagnosis of Lennox-Gastaut syndrome, where seizures that result in drops are not completely controlled by current antiepileptic treatments.
  • Onset of seizures at 11 years of age or younger.
  • Abnormal cognitive development.
  • Must be receiving at least 1 concomitant AED and up to 4 concomitant anti-epileptic treatments.

Key Exclusion Criteria:

  • Etiology of seizures is a degenerative neurological disease.
  • History of hemiclonic seizures in the first year of life.
  • Subject only has drop seizures in clusters, where individual seizures cannot be counted reliably.
  • Pulmonary arterial hypertension.
  • Current or past history of cardiovascular or cerebrovascular disease, such as cardiac valvulopathy, myocardial infarction or stroke.
  • 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.
  • Taking felbamate for less than 1 year prior to screening and/or does not have stable liver function and hematology laboratory tests, and/or the dose has not been stable for at least 60 days prior to the Screening Visit.
  • Currently receiving an investigational product.
  • Institutionalized in a general nursing home (ie, in a facility that does not specialize in epilepsy care).
  • 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.

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): NCT03355209


Locations
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United States, Arizona
Center for Neurosciences
Tucson, Arizona, United States, 85718
United States, California
University of California Los Angeles
Los Angeles, California, United States, 90095
University of California San Francisco
San Francisco, California, United States, 94143
United States, Colorado
The Children's Hospital Colorado
Aurora, Colorado, United States, 80045
United States, District of Columbia
Childrens National Medical Center
Washington, District of Columbia, United States, 20010
United States, Florida
NW FL Clinical Research Group, LLC
Gulf Breeze, Florida, United States, 32561
Nicklaus Children's Hospital
Miami, Florida, United States, 33155
Florida Hospital Orlando
Orlando, Florida, United States, 32803
Pediatric Neurology, PA
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 & Robert Lurie Children's Hospital of Chicago
Chicago, Illinois, United States, 80045
United States, Maryland
Mid-Atlantic Epilepsy and Sleep Center
Bethesda, Maryland, United States, 20817
United States, Massachusetts
Massachusetts General Hospital For Children
Boston, Massachusetts, United States, 02114
United States, Michigan
Spectrum Health
Grand Rapids, Michigan, United States, 49503
William Beaumont Hospital
Royal Oak, Michigan, United States, 48073
United States, Minnesota
Mayo Clinic
Rochester, Minnesota, United States, 55905
Minnesota Epilepsy Group, PA
Saint Paul, Minnesota, United States, 55102
United States, New Jersey
Northeast Regional Epilepsy Group
Hackensack, New Jersey, United States, 07601
Institute of Neurology and Neurosurgery at St. Barnabas
Livingston, New Jersey, United States, 07039
United States, New York
Icahn School of Medicine at Mount Sinai
Hartsdale, New York, United States, 10530
NYU Comprehensive Epilepsy Center
New York, New York, United States, 10016
United States, Ohio
University Hospital Cleveland
Cleveland, Ohio, United States, 44106
United States, Oregon
Oregon Health & Science University
Portland, Oregon, United States, 97239
United States, Pennsylvania
Wellspan Pediatric Neurology
Manchester, Pennsylvania, United States, 17345
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104-4318
United States, Texas
UT Southwestern Medical Center
Dallas, Texas, United States, 75235
Cook Children's Medical Center
Fort Worth, Texas, United States, 76104
Baylor College of Medicine
San Antonio, Texas, United States, 78207
United States, Utah
University of Utah
Salt Lake City, Utah, United States, 84108
United States, Washington
MultiCare Institute for Research & Innovation
Tacoma, Washington, United States, 98405
Australia
Melbourne Brain Centre Austin Hospital
Heidelberg, Australia, 3084
Children's Health Queensland Hospital and Health Service at Lady Cilento Children's Hospital
South Brisbane, Australia, QLD 4101
Belgium
Hôpital Universitaire Des Enfants Reine Fabiola
Brussels, Belgium, 1020
Universitair Ziekenhuis Brussel
Brussels, Belgium, 1090
Universitair Ziekenhuis Antwerpen
Edegem, Belgium, 2650
Canada, British Columbia
British Columbia Children's Hospital
Vancouver, British Columbia, Canada, 6H 3N1
Canada, Ontario
Sick Kids
Toronto, Ontario, Canada, M5G1X8
Denmark
Epilepsihospitalet Filadelfia
Dianalund, Denmark, DK4293
France
Hôpital des enfants- Pellegrin- CIC Pédiatrique
Bordeaux, France, 33076
Hôpital Femme Mère Enfant
Bron, France, 69677
CHRU de Lille Hopital Roger Salengro Neurophysiologie Clinique (RDC)
Lille, France, 59037
AP-HM - CHU TIMONE ENFANTS Service de Neurologie Pédiatrique
Marseille, France, 13385
Hôpital de la Pitié Salpétrière
Paris, France, 75013
Hôpital Universitaire Necker-Enfants Malades Service de neurologie pédiatrique
Paris, France, 75015
Hôpital Robert Debré Pédiatrie Médicale Service Neurologie Et Maladies Métaboliques Robert-debré
Paris, France, 75019
Germany
Krankenhaus Mara GmbH
Bielefeld, Germany, 33617
Universitätsklinikum Freiburg Klinik für Neuropädiatrie und Muskelerkrankungen
Freiburg, Germany, 79016
Universitätsklinikum Jena - Klinik für Neuropädiatrie /SPZ
Jena, Germany, 07747
Universitaetsklinikum Schleswig-Holstein, Campus Kiel
Kiel, Germany, 24105
Saechsisches Epilepsiezentrum Radeberg
Radeberg, Germany, 01454
Schoen Klinik Vogtareuth - Klinik fuer Neuropediatrie und Neurologische Rehabilitation
Vogtareuth, Germany, 83569
Italy
Istituto Scienze Neurologiche di Bologna, Ospedale Bellaria
Bologna, Italy, 40123
AOU Anna Meyer Clinica di Neurologia Pediatrica
Firenze, Italy, 50139
Istituto Pediatrico Giannina Gaslini
Genova, Italy, 16147
Ospedale Pediatrico Bambino Gesù, IRCS
Roma, Italy, 00165
Policlinico Universitario A.Gemelli
Roma, Italy, 00168
Japan
Fukuoka Children's Hospital
Fukuoka-shi, Fukuoka, Japan, 813-0017
Kanagawa Children's Medical Center
Yokohama, Kanagawa, Japan, 232-8555
National Hospital Organization Nagasaki Medical Center
Omura, Nagasaki, Japan, 856-8562
National Hospital Organization Nishi-Niigata Chuo National Hospital
Niigata-shi, Niigata, Japan, 950-2085
Okayama University Hospital
Okayama-shi, Okayama, Japan, 700-8558
Osaka City General Hospital
Osaka-shi, Osaka, Japan, 534-0021
Hokkaido University Hospital
Kita-ku, Sapporo Hokkaido, Japan, 060-8648
National Epilepsy Center Shizuoka Institute of Epilepsy and Neurological Disorders
Shizuoka-city, Shizuoka, Japan, 420-8688
Tokyo Women's Medical University Hospital
Shinjuku-Ku, Tokyo, Japan, 162-8666
Saitama Children's Medical Center
Saitama, Japan, 330-8777
Mexico
Antiguo Hospital Civil de Guadalajara Fray Antonio Alcalde
Jalisco, Guadalajara, Mexico, 44280
Hospital Aranda de la Parra
León, Mexico, 37000
Netherlands
Epilepsy center SEIN - Zwolle
Zwolle, Netherlands, 8025BV
Poland
NZOZ Vitamed
Bydgoszcz, Poland, 85-079
Centrum Medyczne Plejady
Kraków, Poland, 30-363
Spain
Hospital Universitario Vall d'Hebron
Barcelona, Spain, 08035
Hospital del la Mar
Barcelone, Spain, 08003
Hospital Ruber Internacional Servicio de Neurologia
Madrid, Spain, 28034
Clinica Universitaria de Navarra
Pamplona, Spain, 31008
Centro de Neurologia Avanzada
Sevilla, Spain, 41013
Sweden
Sahlgrenska University Hospital
Göteborg, Sweden, 413 45
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: NCT03355209    
Other Study ID Numbers: ZX008-1601
First Posted: November 28, 2017    Key Record Dates
Last Update Posted: June 18, 2021
Last Verified: June 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Zogenix, Inc. ( Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc. ):
LGS
Additional relevant MeSH terms:
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Lennox Gastaut Syndrome
Syndrome
Disease
Pathologic Processes
Epileptic Syndromes
Epilepsy
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Genetic Diseases, Inborn