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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. |
ClinicalTrials.gov Identifier: NCT03355209 |
Recruitment Status :
Recruiting
First Posted : November 28, 2017
Last Update Posted : September 17, 2020
|
- Study Details
- Tabular View
- No Results Posted
- Disclaimer
- How to Read a Study Record
Condition or disease | Intervention/treatment | Phase |
---|---|---|
Lennox Gastaut Syndrome | Drug: ZX008 0.2 or 0.8 mg/kg/day Drug: Matching Placebo | Phase 3 |
Study Type : | Interventional (Clinical Trial) |
Estimated Enrollment : | 225 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 : | December 2020 |
Estimated Study Completion Date : | December 2020 |

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) or placebo.
|
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 |
Placebo Comparator: 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. |
- 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
- 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

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
Ages Eligible for Study: | 2 Years to 35 Years (Child, Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
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.

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
Contact: ZX008 Clinical Trials Information Desk | 510-338-9968 | ClinStudyInfo@zogenix.com |

United States, Arizona | |
Center for Neurosciences | Active, not recruiting |
Tucson, Arizona, United States, 85718 | |
United States, California | |
University of California Los Angeles | Active, not recruiting |
Los Angeles, California, United States, 90095 | |
University of California San Francisco | Active, not recruiting |
San Francisco, California, United States, 94143 | |
United States, Colorado | |
The Children's Hospital Colorado | Active, not recruiting |
Aurora, Colorado, United States, 80045 | |
United States, District of Columbia | |
Childrens National Medical Center | Active, not recruiting |
Washington, District of Columbia, United States, 20010 | |
United States, Florida | |
NW FL Clinical Research Group, LLC | Active, not recruiting |
Gulf Breeze, Florida, United States, 32561 | |
Nicklaus Children's Hospital | Active, not recruiting |
Miami, Florida, United States, 33155 | |
Florida Hospital Orlando | Active, not recruiting |
Orlando, Florida, United States, 32803 | |
Pediatric Neurology, PA | Active, not recruiting |
Orlando, Florida, United States, 32819 | |
United States, Georgia | |
Clinical Integrative Research Center of Atlanta, Panda Neurology | Active, not recruiting |
Atlanta, Georgia, United States, 30328 | |
United States, Illinois | |
Ann & Robert Lurie Children's Hospital of Chicago | Completed |
Chicago, Illinois, United States, 80045 | |
United States, Maryland | |
Mid-Atlantic Epilepsy and Sleep Center | Active, not recruiting |
Bethesda, Maryland, United States, 20817 | |
United States, Massachusetts | |
Massachusetts General Hospital For Children | Active, not recruiting |
Boston, Massachusetts, United States, 02114 | |
United States, Michigan | |
Spectrum Health | Completed |
Grand Rapids, Michigan, United States, 49503 | |
William Beaumont Hospital | Active, not recruiting |
Royal Oak, Michigan, United States, 48073 | |
United States, Minnesota | |
Mayo Clinic | Active, not recruiting |
Rochester, Minnesota, United States, 55905 | |
Minnesota Epilepsy Group, PA | Active, not recruiting |
Saint Paul, Minnesota, United States, 55102 | |
United States, New Jersey | |
Northeast Regional Epilepsy Group | Active, not recruiting |
Hackensack, New Jersey, United States, 07601 | |
Institute of Neurology and Neurosurgery at St. Barnabas | Active, not recruiting |
Livingston, New Jersey, United States, 07039 | |
United States, New York | |
Icahn School of Medicine at Mount Sinai | Active, not recruiting |
Hartsdale, New York, United States, 10530 | |
NYU Comprehensive Epilepsy Center | Active, not recruiting |
New York, New York, United States, 10016 | |
United States, Ohio | |
University Hospital Cleveland | Active, not recruiting |
Cleveland, Ohio, United States, 44106 | |
United States, Oregon | |
Oregon Health & Science University | Active, not recruiting |
Portland, Oregon, United States, 97239 | |
United States, Pennsylvania | |
Wellspan Pediatric Neurology | Active, not recruiting |
Manchester, Pennsylvania, United States, 17345 | |
Children's Hospital of Philadelphia | Active, not recruiting |
Philadelphia, Pennsylvania, United States, 19104-4318 | |
United States, Texas | |
UT Southwestern Medical Center | Active, not recruiting |
Dallas, Texas, United States, 75235 | |
Cook Children's Medical Center | Active, not recruiting |
Fort Worth, Texas, United States, 76104 | |
Baylor College of Medicine | Active, not recruiting |
San Antonio, Texas, United States, 78207 | |
United States, Utah | |
University of Utah | Active, not recruiting |
Salt Lake City, Utah, United States, 84108 | |
United States, Washington | |
MultiCare Institute for Research & Innovation | Active, not recruiting |
Tacoma, Washington, United States, 98405 | |
Australia | |
Melbourne Brain Centre Austin Hospital | Active, not recruiting |
Heidelberg, Australia, 3084 | |
Children's Health Queensland Hospital and Health Service at Lady Cilento Children's Hospital | Active, not recruiting |
South Brisbane, Australia, QLD 4101 | |
Belgium | |
Universitair Ziekenhuis Antwerpen | Active, not recruiting |
Antwerpen, Belgium, 2650 | |
Hôpital Universitaire Des Enfants Reine Fabiola | Active, not recruiting |
Brussels, Belgium, 1020 | |
Universitair Ziekenhuis Brussel | Active, not recruiting |
Brussels, Belgium, 1090 | |
Canada, British Columbia | |
British Columbia Children's Hospital | Active, not recruiting |
Vancouver, British Columbia, Canada, 6H 3N1 | |
Canada, Ontario | |
Sick Kids | Active, not recruiting |
Toronto, Ontario, Canada, M5G1X8 | |
Denmark | |
Epilepsihospitalet Filadelfia | Active, not recruiting |
Dianalund, Denmark, DK4293 | |
France | |
Hôpital des enfants- Pellegrin- CIC Pédiatrique | Active, not recruiting |
Bordeaux, France, 33076 | |
Hôpital Femme Mère Enfant | Active, not recruiting |
Bron, France, 69677 | |
CHRU de Lille Hopital Roger Salengro Neurophysiologie Clinique (RDC) | Active, not recruiting |
Lille, France, 59037 | |
AP-HM - CHU TIMONE ENFANTS Service de Neurologie Pédiatrique | Active, not recruiting |
Marseille, France, 13385 | |
Hôpital Universitaire Necker-Enfants Malades Service de neurologie pédiatrique | Active, not recruiting |
Paris, France, 75015 | |
Hôpital Robert Debré Pédiatrie Médicale Service Neurologie Et Maladies Métaboliques Robert-debré | Active, not recruiting |
Paris, France, 75019 | |
Germany | |
Krankenhaus Mara GmbH | Active, not recruiting |
Bielefeld, Germany, 33617 | |
Universitetsklinik Freiburg | Active, not recruiting |
Friedberg, Germany | |
Universitätsklinikum Jena - Klinik für Neuropädiatrie /SPZ | Active, not recruiting |
Jena, Germany | |
Universitaetsklinikum Schleswig-Holstein, Campus Kiel | Active, not recruiting |
Kiel, Germany | |
Sächsisches Epilepsie-Zentrum Radeberg | Active, not recruiting |
Radeberg, Germany | |
Schoen Klinik Vogtareuth - Klinik fuer Neuropediatrie und Neurologische Rehabilitation | Active, not recruiting |
Vogtareuth, Germany | |
Italy | |
Ospedale Bellaria | Active, not recruiting |
Bologna, Italy | |
AOU Anna Meyer Clinica di Neurologia Pediatrica | Active, not recruiting |
Firenze, Italy, 50139 | |
Istituto Pediatrico Giannina Gaslini | Active, not recruiting |
Genova, Italy, 16148 | |
Ospedale Bambino Gesù, IRCS | Active, not recruiting |
Roma, Italy, 00165 | |
Unita Operativa di Neuropsichiatria Infantile, Universita Cattolica del Sacro Cuore, Fondazione Poli | Active, not recruiting |
Roma, Italy, 00168 | |
Japan | |
National Hospital Organization Nagasaki Medical Center | Recruiting |
Omura, Nagasaki, Japan | |
National Hospital Organization Nishi-Niigata Chuo National Hospital | Recruiting |
Niigata-shi, Niigata, Japan | |
Okayama University Hospital | Recruiting |
Okayama-shi, Okayama, Japan | |
National Epilepsy Center Shizuoka | Recruiting |
Shizuoka-city, Shizuoka, Japan | |
Mexico | |
Antiguo Hospital Civil de Guadalajara Fray Antonio Alcalde | Active, not recruiting |
Jalisco, Guadalajara, Mexico, 44280 | |
Hospital Aranda de la Parra | Active, not recruiting |
Mexico, Mexico | |
Netherlands | |
Epilepsy center SEIN - Zwolle | Active, not recruiting |
Zwolle, Netherlands, 8025BV | |
Poland | |
NZOZ Vitamed | Active, not recruiting |
Bydgoszcz, Poland, 85-079 | |
Centrum Medyczne Plejady | Active, not recruiting |
Kraków, Poland, 30-363 | |
Spain | |
Hospital del Mar | Active, not recruiting |
Barcelona, Spain, 08022 | |
Hospital Universitario Vall d'Hebron | Active, not recruiting |
Barcelona, Spain | |
Hospital Ruber Internacional Servicio de Neurologia | Active, not recruiting |
Madrid, Spain, 28034 | |
Clinica Universitaria de Navarra | Active, not recruiting |
Pamplona, Spain, 31008 | |
Sweden | |
Sahlgrenska University Hospital | Active, not recruiting |
Göteborg, Sweden |
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: | September 17, 2020 |
Last Verified: | September 2020 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | No |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
LGS |
Lennox Gastaut Syndrome Syndrome Disease Pathologic Processes Epileptic Syndromes |
Epilepsy Brain Diseases Central Nervous System Diseases Nervous System Diseases Genetic Diseases, Inborn |