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A Trial of Two Fixed Doses of ZX008 (Fenfluramine HCl) as an Adjunctive Therapy in Children and Young Adults With Dravet Syndrome

This study is currently recruiting participants.
See Contacts and Locations
Verified July 2017 by Zogenix, Inc. ( Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc. )
Sponsor:
Information provided by (Responsible Party):
Zogenix, Inc. ( Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc. )
ClinicalTrials.gov Identifier:
NCT02826863
First received: June 13, 2016
Last updated: July 3, 2017
Last verified: July 2017
  Purpose
This is a multicenter, double-blind, parallel-group, placebo-controlled, study to assess the efficacy, safety, and pharmacokinetics of ZX008 when used as adjunctive therapy in pediatric and young adult subjects with Dravet syndrome. Subjects who qualify for the study will be randomized (1:1:1) in a double-blind manner to receive 1 of 2 doses of ZX008 or placebo. All subjects will be titrated to their randomized dose over a 14-day Titration Period. Following titration, subjects will continue treatment at their randomly assigned dose over a 12-week Maintenance Period. Total treatment time from the beginning of the Titration Period through the end of the Maintenance Period is 14 weeks.

Condition Intervention Phase
Dravet Syndrome Drug: ZX008 - 0.8 mg/kg/day Drug: ZX008 - 0.2 mg/kg/day Drug: Placebo Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Multicenter, Randomized, Double-blind, Parallel Group, Placebo-controlled Trial of Two Fixed Doses of ZX008 (Fenfluramine Hydrochloride) Oral Solution as an Adjunctive Therapy in Children and Young Adults With Dravet Syndrome

Resource links provided by NLM:


Further study details as provided by Zogenix, Inc. ( Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc. ):

Primary Outcome Measures:
  • Change from baseline in frequency of convulsive seizures in subjects receiving ZX008 0.8mg/kg/day compared to placebo [ Time Frame: Time between 6-week baseline assessment period and 14 week treatment and maintenance period ]
    Parent/caregiver seizure diary record will be used to assess frequency, type and duration of seizure activity


Secondary Outcome Measures:
  • Change from baseline in frequency of convulsive seizures for subjects receiving ZX008 0.2mg/kg/day compared to placebo [ Time Frame: Time between 6-week baseline assessment period and 14 week treatment and maintenance period ]
    Parent/caregiver seizure diary record will be used to assess frequency, type and duration of seizure activity

  • Proportion of subjects achieving a ≥40% or ≥50% reduction from baseline in convulsive seizure frequency and longest seizure-free interval in subjects receiving ZX008 0.2 and 0.8 mg/kg/day compared to placebo [ Time Frame: Time between 6-week baseline assessment period and combined 14 week treatment and maintenance period ]
    Parent/caregiver seizure diary record will be used to assess frequency, type and duration of seizure activity

  • Frequency and severity of seizure activity for subjects receiving ZX008 0.2mg/kg/day and 0.8mg/kg/day compared to placebo [ Time Frame: Time between 6-week baseline assessment period and 14 week treatment and maintenance period ]
    Seizure severity evaluated using parent/caregiver seizure diary to record frequency and severity of seizure activity

  • Safety and tolerability of ZX008 0.2 and 0.8 mg/kg/day compared to placebo [ Time Frame: Week 1 through Week 14 ]
    Safety and tolerability will be evaluated by reported adverse events, laboratory parameters, physical and neurological examination, vital signs, electrocardiograms, echocardiograms, and body weight. (Cognitive function will be assessed using age-appropriate versions of the Brief Rating Inventory of Executive Function [BRIEF].)


Estimated Enrollment: 130
Study Start Date: June 2016
Estimated Study Completion Date: December 2018
Estimated Primary Completion Date: December 2018 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Experimental: ZX008 - 0.8 mg/kg/day
ZX008 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.
Drug: ZX008 - 0.8 mg/kg/day
ZX008 drug product is an oral aqueous solution of fenfluramine hydrochloride buffered to pH 5 and provided in concentrations of 1.25 mg/mL, 2.5 mg/mL, and 5 mg/mL. The product is sugar free and is intended to be compatible with KD.
Experimental: Experimental: ZX008 - 0.2 mg/kg/day
ZX008 is supplied as an oral solution in concentrations of 0.2 mg/kg/day ZX008 will be administered twice a day (BID) in equally divided doses with food.
Drug: ZX008 - 0.2 mg/kg/day
ZX008 drug product is an oral aqueous solution of fenfluramine hydrochloride buffered to pH 5 and provided in concentrations of 1.25 mg/mL, 2.5 mg/mL, and 5 mg/mL. The product is sugar free and is intended to be compatible with KD.
Placebo Comparator: Placebo Comparator: Matching Placebo
Placebo will be administered twice a day (BID) in equally divided doses with food.
Drug: Placebo
Placebo will be administered twice a day (BID) in equally divided doses with food.

  Eligibility

Ages Eligible for Study:   2 Years to 18 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 Screening Visit.
  • 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 (including KD and VNS) must be stable for at least 4 weeks prior to screening and are expected to remain stable throughout the study.
  • Parent/caregiver is willing and able to be compliant with diary completion, visit schedule and study drug accountability.

Key Exclusion Criteria

  • Pulmonary arterial hypertension.
  • 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: centrally-acting anorectic agents; monoamine-oxidase inhibitors; medications that act via serotonin including serotonin reuptake inhibitors; atomoxetine, or other centrally-acting noradrenergic agonist; cyproheptadine, and/or 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.
  • Subject is unwilling to refrain from large or daily servings of grapefruits and/or Seville oranges, and their juices beginning with the Baseline Period and throughout the study.
  • 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.
  • Currently receiving an investigational product.
  Contacts and Locations
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, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02826863

Contacts
Contact: ZX008 Clinical Trials Disclosure Desk Medinfo@zogenix.com
Contact: Betty Quarles, B.S. BQuarles@zogenix.com

Locations
Australia
Melbourne Brain Centre Austin Hospital Recruiting
Melbourne, Australia
Princes Margaret Hospital Not yet recruiting
Perth, Australia
Children's Health Queensland Hospital and Health Service at Lady Cilento Children's Hospital Recruiting
South Brisbane, Australia
The Children's Hospital Westmead Dept. of Neurology and Neurosurgery Recruiting
Westmead, Australia
Belgium
Universitair Ziekenhuis Antwerpen Recruiting
Antwerp, Belgium
Denmark
Danish National Epilepsy Centre Recruiting
Dianalund, Denmark
France
French Ref centre Necker Hospital Paris Recruiting
Paris, France
Germany
Epilepsiezentrum / Neuropädiatrie Hedwig-von-Rittberg-Zentrum Für Kinder und Jugendliche Recruiting
Berlin, Germany
Krankenhaus Mara Epilepsie-Zentrum Bethel Recruiting
Bielefeld, Germany
Epilepsiezentrum Freiburg Recruiting
Freiburg, Germany
Universitaetsklinikum Jena Klinik fuer Kinder- und Jugendmedizin Neuropaediatrie Recruiting
Jena, Germany
Klinik für Neuropädiatrie Universitätsklinikum Schleswig Holstein Campus Kiel Recruiting
Kiel, Germany
Kleinwachau Saechsisches Epilepsiezentrum Radeberg gemeinnuetzige GmbH Recruiting
Radeberg (Sachsen), Germany
Universitaetsklinik fuer Kinder- und Jugendmedizin Abteilung III Recruiting
Tübingen, Germany
Schoen Klinik Vogtareuth Neuropaediatrie und Neurologische Rehabilitation, Epilepsiezentrum fuer Kinder und Jugendlische, Tagesklinik fuer Neuropaediatrie Recruiting
Vogtareuth, Germany
Italy
AOU Anna Meyer Recruiting
Firenze, Italy, 50139
Istituto Pediatrico Giannina Gaslini Dipartimento di Neurologia Recruiting
Genova, Italy
A.O Carlo Poma Recruiting
Mantova, Italy, 46100
Instituto Neurologica Carlo Besta Recruiting
Milano, Italy, 20133
Ospedale Fatebenefratelli e Oftalmico Recruiting
Milano, Italy
U.O. Neurologia Dipartimento di Neuroscienze Ospedale Pediatrico Bambino Gesù, IRCS Recruiting
Roma, Italy, 00165
Ospedal Policlinico Giambattista Rossi diBorga Roma Recruiting
Verona, Italy, 37134
Spain
Hospital Sant Joan de Déu Recruiting
Barcelona, Spain
Hospital Ruber Internacional Primera Planta Servicio de Neurologia Recruiting
Madrid, Spain
Clinica Universitaria de Navarra Fase 4. Segunda planta, Consulta de Pediatria Recruiting
Pamplona, Spain
United Kingdom
Birmingham Children Hospital Recruiting
Birmingham, United Kingdom
Institute of Neurosciences Queens Elizabeth University Hospital Recruiting
Glasgow, United Kingdom
Alder Hey Hospital Recruiting
Liverpool, United Kingdom
Evelina Hospital Recruiting
London, United Kingdom
Great Ormonnd Street Hospital for Children NHS Foundation Trust Recruiting
London, United Kingdom
Sheffield Children's Hospital Recruiting
Sheffield, United Kingdom
Sponsors and Collaborators
Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc.
  More Information

Responsible Party: Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc.
ClinicalTrials.gov Identifier: NCT02826863     History of Changes
Other Study ID Numbers: ZX008-1502
Study First Received: June 13, 2016
Last Updated: July 3, 2017

Keywords provided by Zogenix, Inc. ( Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc. ):
seizure
tonic-atonic
epilepsy
myoclonic
encephalopathy

Additional relevant MeSH terms:
Syndrome
Epilepsies, Myoclonic
Disease
Pathologic Processes
Epilepsy
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Pharmaceutical Solutions
Fenfluramine
Serotonin Uptake Inhibitors
Neurotransmitter Uptake Inhibitors
Membrane Transport Modulators
Molecular Mechanisms of Pharmacological Action
Neurotransmitter Agents
Serotonin Agents
Physiological Effects of Drugs

ClinicalTrials.gov processed this record on September 21, 2017