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
NCT ID: NCT02823145
Last Updated: 2025-07-14
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE3
375 participants
INTERVENTIONAL
2016-06-08
2023-01-27
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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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).
ZX008 (Fenfluramine Hydrochloride)
Interventions
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ZX008 (Fenfluramine Hydrochloride)
Eligibility Criteria
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Inclusion Criteria
* 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).
Exclusion Criteria
* 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.
2 Years
35 Years
ALL
No
Sponsors
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Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc.
INDUSTRY
Responsible Party
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Principal Investigators
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UCB Cares
Role: STUDY_DIRECTOR
001 844 599 2273
Locations
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Phoenix Children's Hospital
Phoenix, Arizona, United States
Center for Neurosciences - Tucson
Tucson, Arizona, United States
Children's Hospital of Orange County
Orange, California, United States
Rady Children's Hospital San Diego
San Diego, California, United States
University of California San Francisco
San Francisco, California, United States
The Children's Hospital Colorado
Aurora, Colorado, United States
NW FL Clinical Research Group, LLC
Gulf Breeze, Florida, United States
Miami Children's Hospital Brain Institute
Miami, Florida, United States
Neurology and Epilepsy Research Center
Orlando, Florida, United States
Clinical Integrative Research Center of Atlanta, Panda Neurology
Atlanta, Georgia, United States
Ann and Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
Massachusetts General Hospital
Boston, Massachusetts, United States
Boston Children's Hospital
Boston, Massachusetts, United States
Children's Hospital Of Michigan
Detroit, Michigan, United States
Mayo Clinic
Rochester, Minnesota, United States
Minnesota Epilepsy Group, P.A.
Saint Paul, Minnesota, United States
Institute of Neurology and Neurosurgery at St. Barnabus
Livingston, New Jersey, United States
University Hospitals Cleveland Medical Center
Cleveland, Ohio, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
Le Bonheur Children's Hospital
Memphis, Tennessee, United States
Cook Children's Medical Center
Fort Worth, Texas, United States
University of Utah
Salt Lake City, Utah, United States
Seattle Children's Hospital
Seattle, Washington, United States
MultiCare Institute for Research & Innovation
Tacoma, Washington, United States
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
Universitair Ziekenhuis Antwerpen
Edegem, , Belgium
Centre Hospitalier Universitaire Sainte-Justine
Montreal, , Canada
British Columbia Children's Hospital
Vancouver, , Canada
Danish National Epilepsy Centre
Dianalund, , Denmark
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 Timone Neurologie 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
DRK Kliniken Berlin - Westend Epilepsiezentrum / Neuropaediatrie
Berlin, , Germany
Krankenhaus Mara Epilepsie-Zentrum Bethel
Bielefeld, , Germany
Universitaetsklinikum Freiburg Zentrum fuer Kinder- und Jugendmedizin
Freiburg im Breisgau, , 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
AOU Anna Meyer Clinica di Neurologia Pediatrica
Florence, , Italy
Istituto Pediatrico Giannina Gaslini Dipartimento di Neurologia
Genova, , Italy
A.O. Carlo Poma
Mantova, , Italy
Istituto Neurologica Carlo Besta
Milan, , Italy
Ospedale Fatebenefratelli e Oftalmico
Milan, , Italy
Policlinico A. Gemelli
Roma, , Italy
U.O. Neurologia Dipartimento di Neuroscienze Ospedale Pediatrico Bambino Gesù
Roma, , Italy
Ospedale Civile Maggiore di Borgo Trento - Ospedale della Donna e del Bambino
Verona, , Italy
Saitama Children's Medical Center
Saitama, , Japan
National Epilepsy Center Shizuoka Institute of Epilepsy and Neurological Disorders
Shizuoka, , Japan
Tokyo Women's Medical University Hospital
Tokyo, , Japan
Kempenhaeghe
Heeze, , Netherlands
Stichting Epilepsie Instellingen Nederland
Zwolle, , Netherlands
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
Birmingham Children Hospital NHS Foundation Trust
Birmingham, , United Kingdom
Institute of Neurosciences Queen Elizabeth University Hospital
Glasgow, , United Kingdom
Alder Hey Hospital
Liverpool, , United Kingdom
Great Ormond Street Hospital for Children NHS Foundation Trust
London, , United Kingdom
St. Thomas Hospital
London, , United Kingdom
Sheffield Children's Hospital
Sheffield, , United Kingdom
Countries
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References
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Scheffer IE, Nabbout R, Lagae L, Devinsky O, Auvin S, Thiele EA, Wirrell EC, Polster T, Specchio N, Pringsheim M, Imai K, Lock MD, Langlois M, Roper RZ, Lothe A, Sullivan J. Long-term safety and effectiveness of fenfluramine in children and adults with Dravet syndrome. Epilepsia. 2025 Jun;66(6):1919-1932. doi: 10.1111/epi.18342. Epub 2025 Mar 12.
Cross JH, Galer BS, Gil-Nagel A, Devinsky O, Ceulemans B, Lagae L, Schoonjans AS, Donner E, Wirrell E, Kothare S, Agarwal A, Lock M, Gammaitoni AR. Impact of fenfluramine on the expected SUDEP mortality rates in patients with Dravet syndrome. Seizure. 2021 Dec;93:154-159. doi: 10.1016/j.seizure.2021.10.024. Epub 2021 Nov 2.
Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Other Identifiers
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2016-002804-14
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
ZX008-1503
Identifier Type: -
Identifier Source: org_study_id
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