A Trial of Two Fixed Doses of ZX008 (Fenfluramine HCl) in Children and Young Adults With Dravet Syndrome
NCT ID: NCT02682927
Last Updated: 2023-09-28
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE3
262 participants
INTERVENTIONAL
2016-01-15
2020-07-29
Brief Summary
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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ZX008 - 0.8 mg/kg/day
ZX008 (fenfluramine HCl) is supplied as an oral solution in concentrations of 1.25, 2.5, and 5 mg/mL. ZX008 will be administered twice a day (BID) in equally divided doses with food.
ZX008 (Fenfluramine Hydrochloride)
ZX008 drug product is an oral aqueous solution of fenfluramine hydrochloride buffered to pH 5. The product is sugar free and is intended to be compatible with a ketogenic diet.
ZX008 - 0.2 mg/kg/day
ZX008 (fenfluramine HCl) is supplied as an oral solution in concentrations of 1.25, 2.5, and 5 mg/mL. ZX008 will be administered twice a day (BID) in equally divided doses with food.
ZX008 (Fenfluramine Hydrochloride)
ZX008 drug product is an oral aqueous solution of fenfluramine hydrochloride buffered to pH 5. The product is sugar free and is intended to be compatible with a ketogenic diet.
Matching Placebo
Placebo will be administered twice a day (BID) in equally divided doses with food.
Matching Placebo
Placebo solution for ZX008. The product is sugar free and is intended to be compatible with a ketogenic diet.
Interventions
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ZX008 (Fenfluramine Hydrochloride)
ZX008 drug product is an oral aqueous solution of fenfluramine hydrochloride buffered to pH 5. The product is sugar free and is intended to be compatible with a ketogenic diet.
Matching Placebo
Placebo solution for ZX008. The product is sugar free and is intended to be compatible with a ketogenic diet.
Eligibility Criteria
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Inclusion Criteria
* Clinical diagnosis of Dravet syndrome, where convulsive seizures are not completely controlled by current antiepileptic drugs.
* Must have a minimum # of convulsive seizures per 4-week period for past 12 weeks prior to screening.
* All medications or interventions for epilepsy must be stable for at least 4 weeks prior to screening and expected to remain stable throughout the study.
* No cardiovascular or cardiopulmonary abnormality based on ECHO, ECG or physical examination.
* Parent/caregiver is willing and able to be compliant with diary completion, visit schedule and study drug accountability.
Exclusion Criteria
* Current or past history of cardiovascular or cerebrovascular disease, such as cardiac valvulopathy, myocardial infarction or stroke.
* Current or past history of glaucoma.
* Moderate or severe hepatic impairment.
* Receiving concomitant therapy with: anorectic agents; monoamine-oxidase inhibitors; medications that act via serotonin including serotonin reuptake inhibitors; atomoxetine, or other centrally-acting noradrenergic agonist; or cyproheptadine.
* Currently receiving or has received stiripentol in the past 21 days prior to Screening.
* Currently taking carbamazepine, oxcarbamazepine, eslicarbazepine, phenobarbital, or phenytoin, or has taken any of these within the past 30 days.
* Positive result on tetrahydrocannabinol (THC) or cannabidiol (CBD) test at the Screening Visit.
* A clinically significant medical condition,that would interfere with study participation, collection of study data, or pose a risk to the subject.
2 Years
18 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
Phoenix, Arizona, United States
Center for Neurosciences - Tucson
Tucson, Arizona, 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
Panda Neurology
Atlanta, Georgia, United States
Children's Hospital of Chicago
Chicago, Illinois, United States
Massachusetts General Hospital
Boston, Massachusetts, United States
Boston Children's Hospital
Boston, Massachusetts, United States
Mayo Clinic
Rochester, Minnesota, United States
Saint Barnabas Medical Center
Livingston, New Jersey, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 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
Melbourne, , 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
Antwerp, , Belgium
British Columbia Children's Hospital BCCH
Vancouver, British Columbia, Canada
Centre Hospitalier Universitaire Sainte-Justine
Montreal, , Canada
Danish National Epilepsy Centre
Dianalund, , Denmark
French Ref centre Necker Hospital Paris
Paris, , France
Epilepsiezentrum / Neuropädiatrie Hedwig-von-Rittberg-Zentrum Für Kinder und Jugendliche
Berlin, , Germany
Krankenhaus Mara Epilepsie-Zentrum Bethel
Bielefeld, , Germany
Epilepsiezentrum Freiburg
Freiburg im Breisgau, , Germany
Universitaetsklinikum Jena Klinik fuer Kinder- und Jugendmedizin Neuropaediatrie
Jena, , Germany
Klinik für Neuropädiatrie Universitätsklinikum Schleswig Holstein Campus Kiel
Kiel, , Germany
Kleinwachau Saechsisches Epilepsiezentrum Radeberggemeinnuetzige 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
Florence, , Italy
Istituto Pediatrico Giannina Gaslini Dipartimento di Neurologia
Genova, , Italy
A.O Carlo Poma
Mantova, , Italy
Instituto Neurologica Carlo Besta
Milan, , Italy
Ospedale Fatebenefratelli e Oftalmico
Milan, , Italy
U.O. Neurologia Dipartimento di Neuroscienze Ospedale Pediatrico Bambino Gesù, IRCS
Roma, , Italy
Ospedal Policlinico Giambattista Rossi diBorga Roma
Verona, , Italy
Okayama University Hospital
Okayama, Okayama-ken, Japan
Saitama Children's Medical Center
Saitama-shi, Saitama, Japan
National Epilepsy Center Shizuoka Institute
Shizuoka, Shizuoka, Japan
Tokyo Women's Medical University Hospital
Shinjuku-ku, Tokyo, Japan
Hospital Sant Joande 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
Birmingham, , United Kingdom
Institute of Neurosciences Queens Elizabeth University Hospital
Glasgow, , United Kingdom
Alder Hey Hospital
Liverpool, , United Kingdom
Evelina Hospital
London, , United Kingdom
Great Ormonnd Street Hospital for Children NHS Foundation Trust
London, , United Kingdom
Sheffield Children's Hospital
Sheffield, , United Kingdom
Countries
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References
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Sullivan J, Lagae L, Cross JH, Devinsky O, Guerrini R, Knupp KG, Laux L, Nikanorova M, Polster T, Talwar D, Ceulemans B, Nabbout R, Farfel GM, Galer BS, Gammaitoni AR, Lock M, Agarwal A, Scheffer IE; FAiRE DS Study Group. Fenfluramine in the treatment of Dravet syndrome: Results of a third randomized, placebo-controlled clinical trial. Epilepsia. 2023 Oct;64(10):2653-2666. doi: 10.1111/epi.17737. Epub 2023 Aug 17.
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.
Sullivan J, Specchio N, Devinsky O, Auvin S, Perry MS, Strzelczyk A, Gil-Nagel A, Dai D, Galer BS, Gammaitoni AR. Fenfluramine significantly reduces day-to-day seizure burden by increasing number of seizure-free days and time between seizures in patients with Dravet syndrome: A time-to-event analysis. Epilepsia. 2022 Jan;63(1):130-138. doi: 10.1111/epi.17106. Epub 2021 Oct 22.
Sullivan J, Perry MS, Wheless JW, Galer B, Gammaitoni A. Fenfluramine responder analyses and numbers needed to treat: Translating epilepsy trial data into clinical practice. Eur J Paediatr Neurol. 2021 Mar;31:10-14. doi: 10.1016/j.ejpn.2021.01.005. Epub 2021 Jan 22.
Lagae L, Sullivan J, Knupp K, Laux L, Polster T, Nikanorova M, Devinsky O, Cross JH, Guerrini R, Talwar D, Miller I, Farfel G, Galer BS, Gammaitoni A, Mistry A, Morrison G, Lock M, Agarwal A, Lai WW, Ceulemans B; FAiRE DS Study Group. Fenfluramine hydrochloride for the treatment of seizures in Dravet syndrome: a randomised, double-blind, placebo-controlled trial. Lancet. 2019 Dec 21;394(10216):2243-2254. doi: 10.1016/S0140-6736(19)32500-0. Epub 2019 Dec 17.
Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Other Identifiers
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ZX008-1502
Identifier Type: OTHER
Identifier Source: secondary_id
ZX008-1501
Identifier Type: -
Identifier Source: org_study_id
NCT02826863
Identifier Type: -
Identifier Source: nct_alias
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