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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

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
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
Secondary ID [1] 0 0
2019-001331-31
Secondary ID [2] 0 0
ZX008-1900
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Dravet Syndrome 0 0
Lennox Gastaut Syndrome 0 0
Epileptic Encephalopathy 0 0
Condition category
Condition code
Neurological 0 0 0 0
Epilepsy
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Other 0 0 0 0
Research that is not of generic health relevance and not applicable to specific health categories listed above

Intervention/exposure
Study type
Interventional
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.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Change in Adverse Events and Serious Adverse Events leading to study withdrawal in subjects receiving open label ZX008
Timepoint [1] 0 0
Up to 36 months open-label
Primary outcome [2] 0 0
Changes in laboratory test results
Timepoint [2] 0 0
Up to 36 months open-label
Primary outcome [3] 0 0
Changes in heart rate
Timepoint [3] 0 0
Up to 36 months open-label
Primary outcome [4] 0 0
Changes in respiratory rate
Timepoint [4] 0 0
Up to 36 months open-label
Primary outcome [5] 0 0
Changes in blood pressure
Timepoint [5] 0 0
Up to 36 months open-label
Primary outcome [6] 0 0
Change in body weight
Timepoint [6] 0 0
Up to 36 months open-label
Primary outcome [7] 0 0
Changes in heart rhythm
Timepoint [7] 0 0
Up to 36 months open-label
Primary outcome [8] 0 0
Changes in heart valve function
Timepoint [8] 0 0
Up to 36 months open-label
Secondary outcome [1] 0 0
Change in convulsive seizure response
Timepoint [1] 0 0
Up to 36 months open-label
Secondary outcome [2] 0 0
Change in Cognitive CGI by Parent/Caregiver
Timepoint [2] 0 0
Up to 36 months open-label
Secondary outcome [3] 0 0
Change in Behavioral CGI by Parent/Caregiver
Timepoint [3] 0 0
Up to 36 months open-label
Secondary outcome [4] 0 0
Change in Motor CGI by Parent/Caregiver
Timepoint [4] 0 0
Up to 36 months open-label
Secondary outcome [5] 0 0
Change in Cognitive CGI by Investigator
Timepoint [5] 0 0
Up to 36 months open-label
Secondary outcome [6] 0 0
Change in Behavioral CGI by Investigator
Timepoint [6] 0 0
Up to 36 months open-label
Secondary outcome [7] 0 0
Change in Motor CGI by Investigator
Timepoint [7] 0 0
Up to 36 months open-label
Secondary outcome [8] 0 0
Change in Clinical Global Impression by Parent/Caregiver
Timepoint [8] 0 0
Up to 36 months open-label
Secondary outcome [9] 0 0
Change in Clinical Global Impression by Investigator
Timepoint [9] 0 0
Up to 36 months open-label

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
Minimum age
2 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
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

Study design
Purpose of the study
Treatment
Allocation to intervention
NA
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
22/04/2019
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
25/04/2025
Actual
Sample size
Target
Accrual to date
Final
412
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Ep0215 301 - Heidelberg
Recruitment hospital [2] 0 0
Ep0215 302 - South Brisbane
Recruitment hospital [3] 0 0
Ep0215 303 - Westmead
Recruitment postcode(s) [1] 0 0
- Heidelberg
Recruitment postcode(s) [2] 0 0
- South Brisbane
Recruitment postcode(s) [3] 0 0
- Westmead
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
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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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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Massachusetts
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Minnesota
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New Jersey
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New York
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Ohio
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Tennessee
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Texas
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Utah
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United States of America
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Washington
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Belgium
State/province [17] 0 0
Bruxelles
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Belgium
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Edegem
Country [19] 0 0
Belgium
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Jette
Country [20] 0 0
Canada
State/province [20] 0 0
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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Dianalund
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France
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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
State/province [41] 0 0
Guadalajara
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Netherlands
State/province [42] 0 0
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

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Zogenix, Inc.
Address
Country
Other collaborator category [1] 0 0
Commercial sector/industry
Name [1] 0 0
Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc.
Address [1] 0 0
Country [1] 0 0

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
UCB Cares
Address 0 0
001 844 599 2273
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
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)
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.
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.
Available for what types of analyses?
How or where can data be obtained?
IPD available at link: http://www.Vivli.org


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