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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT03936777
Registration number
NCT03936777
Ethics application status
Date submitted
12/04/2019
Date registered
3/05/2019
Date last updated
10/06/2025
Titles & IDs
Public title
A Study to Investigate the Long-Term Safety of ZX008 (Fenfluramine Hydrochloride) Oral Solution in Children and Adults With Epileptic Encephalopathy Including Dravet Syndrome and Lennox-Gastaut Syndrome
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Scientific title
An Open-Label Extension Trial to Assess the Long-Term Safety of ZX008 (Fenfluramine Hydrochloride) Oral Solution as an Adjunctive Therapy for Seizures in Patients With Rare Seizure Disorders Such as Epileptic Encephalopathies Including Dravet Syndrome and Lennox-Gastaut Syndrome
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Secondary ID [1]
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2019-001331-31
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Secondary ID [2]
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ZX008-1900
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Dravet Syndrome
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Lennox Gastaut Syndrome
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Epileptic Encephalopathy
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Condition category
Condition code
Neurological
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Epilepsy
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Human Genetics and Inherited Disorders
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Other human genetics and inherited disorders
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Other
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Research that is not of generic health relevance and not applicable to specific health categories listed above
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - ZX008 (Fenfluramine Hydrochloride)
Experimental: ZX008 (Fenfluramine Hydrochloride) - ZX008 is supplied as an open-label oral solution.Doses will include up to 0.8 mg/kg/day divided into 2 daily doses, up to a maximum of 30 mg/day (subjects taking concomitant STP will receive up to 0.5 mg/kg/day, up to a maximum of 20 mg/day) in a concentration of 2.5 mg/mL.
Treatment: Drugs: ZX008 (Fenfluramine Hydrochloride)
Fenfluramine hydrochloride provided in a concentration of 2.5 mg/mL.
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Change in Adverse Events and Serious Adverse Events leading to study withdrawal in subjects receiving open label ZX008
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Assessment method [1]
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Evaluate the long term safety and tolerability of oral dose administration of ZX008
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Timepoint [1]
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Up to 36 months open-label
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Primary outcome [2]
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Changes in laboratory test results
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Assessment method [2]
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The analysis will include abnormal hematology and chemistry laboratory results outside of reference ranges
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Timepoint [2]
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Up to 36 months open-label
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Primary outcome [3]
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Changes in heart rate
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Assessment method [3]
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The analysis will include change in heart rate using standard measure
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Timepoint [3]
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Up to 36 months open-label
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Primary outcome [4]
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Changes in respiratory rate
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Assessment method [4]
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The analysis will include change in resting respiratory rate using standard measure
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Timepoint [4]
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Up to 36 months open-label
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Primary outcome [5]
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Changes in blood pressure
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Assessment method [5]
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The analysis will include change in resting blood pressure using standard measure
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Timepoint [5]
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Up to 36 months open-label
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Primary outcome [6]
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Change in body weight
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Assessment method [6]
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The analysis will include change in body weight and height by report of BMI in kg/m\^2
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Timepoint [6]
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Up to 36 months open-label
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Primary outcome [7]
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Changes in heart rhythm
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Assessment method [7]
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The analysis will include changes in heart beat as measured with 12-lead electrocardiogram
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Timepoint [7]
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Up to 36 months open-label
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Primary outcome [8]
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Changes in heart valve function
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Assessment method [8]
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The analysis will include change in heart valves as measured with standard echocardiogram
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Timepoint [8]
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Up to 36 months open-label
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Secondary outcome [1]
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Change in convulsive seizure response
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Assessment method [1]
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The analysis will include percent improvement per investigator rating
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Timepoint [1]
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Up to 36 months open-label
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Secondary outcome [2]
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Change in Cognitive CGI by Parent/Caregiver
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Assessment method [2]
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The rating by parent/caregiver of the patient's change in cognitive symptoms since the beginning of study as "very much improved", "much improved", "minimally improved", "no change", "minimally worse", "much worse", or "very much worse".
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Timepoint [2]
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Up to 36 months open-label
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Secondary outcome [3]
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Change in Behavioral CGI by Parent/Caregiver
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Assessment method [3]
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The rating by parent/caregiver of the patient's change in behavioral symptoms since the beginning of study as "very much improved", "much improved", "minimally improved", "no change", "minimally worse", "much worse", or "very much worse".
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Timepoint [3]
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Up to 36 months open-label
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Secondary outcome [4]
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Change in Motor CGI by Parent/Caregiver
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Assessment method [4]
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The rating by parent/caregiver of the patient's change in motor symptoms since the beginning of study as "very much improved", "much improved", "minimally improved", "no change", "minimally worse", "much worse", or "very much worse".
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Timepoint [4]
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Up to 36 months open-label
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Secondary outcome [5]
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Change in Cognitive CGI by Investigator
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Assessment method [5]
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The rating by investigator of the patient's change in cognitive symptoms since the beginning of study as "very much improved", "much improved", "minimally improved", "no change", "minimally worse", "much worse", or "very much worse".
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Timepoint [5]
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Up to 36 months open-label
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Secondary outcome [6]
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Change in Behavioral CGI by Investigator
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Assessment method [6]
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The rating by investigator of the patient's change in behavioral symptoms since the beginning of study as "very much improved", "much improved", "minimally improved", "no change", "minimally worse", "much worse", or "very much worse".
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Timepoint [6]
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Up to 36 months open-label
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Secondary outcome [7]
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Change in Motor CGI by Investigator
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Assessment method [7]
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The rating by investigator of the patient's change in motor symptoms since the beginning of study as "very much improved", "much improved", "minimally improved", "no change", "minimally worse", "much worse", or "very much worse".
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Timepoint [7]
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Up to 36 months open-label
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Secondary outcome [8]
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Change in Clinical Global Impression by Parent/Caregiver
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Assessment method [8]
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The rating by parent/caregiver of the patient's overall change in symptoms since the beginning of study as "very much improved", "much improved", "minimally improved", "no change", "minimally worse", "much worse", or "very much worse"
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Timepoint [8]
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Up to 36 months open-label
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Secondary outcome [9]
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Change in Clinical Global Impression by Investigator
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Assessment method [9]
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The rating by investigator of the patient's overall change in symptoms since the beginning of study as "very much improved", "much improved", "minimally improved", "no change", "minimally worse", "much worse", or "very much worse"
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Timepoint [9]
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Up to 36 months open-label
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Eligibility
Key inclusion criteria
* Male or nonpregnant, nonlactating female
* Satisfactory completion of a core study
* Has a rare seizure disorder, such as epileptic encephalopathy and has successfully completed another Zogenix-sponsored clinical trials with ZX008
* Subject's caregiver is willing and able to be compliant with study procedures, visit schedule and study drug accountability
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Minimum age
2
Years
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Maximum age
No limit
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
* Current cardiac valvulopathy or pulmonary hypertension that is clinically significant
* Moderate or severe hepatic impairment
* Receiving monoamine oxidase inhibitors, serotonin agonists, serotonin antagonists, and serotonin reuptake inhibitors within 14 days of receiving ZX008
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Not applicable
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Single group
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Other design features
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Phase
Phase 3
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Completed
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
22/04/2019
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
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Actual
8/05/2025
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Sample size
Target
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Accrual to date
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Final
412
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Recruitment in Australia
Recruitment state(s)
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Recruitment hospital [1]
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Ep0215 301 - Heidelberg
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Recruitment hospital [2]
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Ep0215 302 - South Brisbane
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Recruitment hospital [3]
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Ep0215 303 - Westmead
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Recruitment postcode(s) [1]
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- Heidelberg
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Recruitment postcode(s) [2]
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- South Brisbane
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Recruitment postcode(s) [3]
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- Westmead
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Recruitment outside Australia
Country [1]
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United States of America
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Arizona
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United States of America
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California
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United States of America
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Colorado
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United States of America
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Florida
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United States of America
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Georgia
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Illinois
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Maryland
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United States of America
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Massachusetts
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Minnesota
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New Jersey
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New York
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United States of America
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Ohio
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Tennessee
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Texas
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Utah
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Washington
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Belgium
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Bruxelles
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Belgium
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Edegem
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Belgium
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Jette
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Canada
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Montreal
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Canada
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Toronto
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Canada
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Vancouver
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Bordeaux Cedex
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France
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Lille
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France
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Marseille Cedex 05
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France
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Paris
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France
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Salouel
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Germany
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Bielefeld
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Germany
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Freiburg Im Breisgau
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Germany
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Jena
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Germany
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Kiel
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Germany
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Radeberg
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Germany
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Vogtareuth
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Italy
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Firenze
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Italy
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Genova
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Italy
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Mantova
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Italy
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Milano
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Italy
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Roma
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Italy
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Verona
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Mexico
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Guadalajara
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Netherlands
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Heeze
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Netherlands
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Zwolle
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Poland
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Bydgoszcz
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Poland
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Krakow
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Spain
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Barcelona
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Spain
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Esplugues de Llobregat
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Spain
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Madrid
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Spain
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Pamplona
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Sweden
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Göteborg
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United Kingdom
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Birmingham
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United Kingdom
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Glasgow
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United Kingdom
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Liverpool
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United Kingdom
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London
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Funding & Sponsors
Primary sponsor type
Commercial sector/industry
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Name
Zogenix, Inc.
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Address
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Country
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Other collaborator category [1]
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Commercial sector/industry
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Name [1]
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Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc.
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Address [1]
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Ethics approval
Ethics application status
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Summary
Brief summary
This is an international, multicenter, open-label, long-term safety study of ZX008 in subjects with Dravet syndrome, Lennox-Gastaut syndrome or epileptic encephalopathy
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Trial website
https://clinicaltrials.gov/study/NCT03936777
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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UCB Cares
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Address
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001 844 599 2273
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Fax
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Email
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Contact person for public queries
Name
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Address
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Fax
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Email
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Contact person for scientific queries
Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
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What data in particular will be shared?
Data from this trial may be requested by qualified researchers six months after product approval in the US and/or Europe, or global development is discontinued, and 18 months after trial completion. Investigators may request access to anonymized individual patient-level data and redacted trial documents which may include: analysis-ready datasets, study protocol, annotated case report form, statistical analysis plan, dataset specifications, and clinical study report. Prior to use of the data, proposals need to be approved by an independent review panel at www.Vivli.org and a signed data sharing agreement will need to be executed. All documents are available in English only, for a pre-specified time, typically 12 months, on a password protected portal. This plan may change if the risk of re-identifying trial participants is determined to be too high after the trial is completed; in this case and to protect participants, individual patient-level data would not be made available.
Supporting document/s available: Study protocol, Statistical analysis plan (SAP), Clinical study report (CSR)
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When will data be available (start and end dates)?
Data from this trial may be requested by qualified researchers six months after product approval in the US and/or Europe or global development is discontinued, and 18 months after trial completion.
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Available to whom?
Qualified researchers may request access to anonymized IPD and redacted study documents which may include: raw datasets, analysis-ready datasets, study protocol, blank case report form, annotated case report form, statistical analysis plan, dataset specifications, and clinical study report. Prior to use of the data, proposals need to be approved by an independent review panel at www.Vivli.org and a signed data sharing agreement will need to be executed. All documents are available in English only, for a pre-specified time, typically 12 months, on a password protected portal.
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Available for what types of analyses?
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How or where can data be obtained?
IPD available at link: http://www.Vivli.org
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What supporting documents are/will be available?
No Supporting Document Provided
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results not provided in
https://clinicaltrials.gov/study/NCT03936777
Download to PDF