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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT02823145




Registration number
NCT02823145
Ethics application status
Date submitted
13/06/2016
Date registered
6/07/2016

Titles & IDs
Public title
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
Scientific title
An Open-Label Extension Trial to Assess the Long-Term Safety of ZX008 (Fenfluramine Hydrochloride HCl) Oral Solution as an Adjunctive Therapy in Children and Young Adults With Dravet Syndrome
Secondary ID [1] 0 0
2016-002804-14
Secondary ID [2] 0 0
ZX008-1503
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Dravet Syndrome 0 0
Condition category
Condition code
Neurological 0 0 0 0
Epilepsy
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
Experimental: 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).
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Percentage of Participants With Treatment-emergent Adverse Events (TEAEs) During the Open-label Extension (OLE) Treatment Period
Timepoint [1] 0 0
From Day 1 to End of OLE Treatment Period - End of Study (EOS) Visit (Month 42)
Primary outcome [2] 0 0
Percentage of Participants With Treatment-emergent Adverse Events (TEAEs) Leading to Withdrawal From Investigational Medicinal Product (IMP) During the OLE Treatment Period
Timepoint [2] 0 0
From Day 1 to End of OLE Treatment Period - EOS Visit (Month 42)
Primary outcome [3] 0 0
Percentage of Participants With Serious Treatment-emergent Adverse Events (TEAEs) During the OLE Treatment Period
Timepoint [3] 0 0
From Day 1 to End of OLE Treatment Period - EOS Visit (Month 42)
Secondary outcome [1] 0 0
Change From Baseline (Core) in Convulsive Seizure Frequency Per 28 Days From Day 1 to End of Study (EOS) Visit (Month 42) in the OLE Treatment Period
Timepoint [1] 0 0
From Day 1 to End of OLE Treatment Period (EOS Visit - up to Month 42), compared to Baseline (Core)
Secondary outcome [2] 0 0
Change From Baseline (Core) in Convulsive Seizure Frequency Per 28 Days From Month 2 to EOS (Month 42) in the OLE Treatment Period
Timepoint [2] 0 0
From Month 2 to End of OLE Treatment Period (EOS Visit - up to Month 42), compared to Baseline (Core)
Secondary outcome [3] 0 0
Convulsive Seizure Frequency (CSF) Per 28 Days During the OLE Treatment Period (to Month 36)
Timepoint [3] 0 0
At Month 1, Month 2, Month 3, Month 4-6, Month 7-9, Month 10-12, Month 13-15, Month 16-18, Month 19-21, Month 22-24, Month 25-27, Month 28-30, Month 31-33, and Month 34-36
Secondary outcome [4] 0 0
Convulsive Seizure Frequency (CSF) by Mean Daily Dose During the Overall OLE Treatment Period
Timepoint [4] 0 0
From Day 1 to End of OLE Treatment Period - End of Study (EOS) Visit (Month 42)
Secondary outcome [5] 0 0
Percentage of Participants With Changes in Antiepileptic Drug (AED) Medications During First 6 Months of OLE Treatment Period
Timepoint [5] 0 0
At Month 1, 2, 3, 4 , 5, and 6 of OLE Treatment Period

Eligibility
Key inclusion criteria
Key

* Male or non-pregnant, non-lactating female, age 2 to 18 years, inclusive as of the day of the core study Screening Visit.
* 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).

Key
Minimum age
2 Years
Maximum age
35 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* Current or past history of cardiovascular or cerebrovascular disease, myocardial infarction or stroke.
* 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.

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
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
8/06/2016
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
27/01/2023
Sample size
Target
Accrual to date
Final
375
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Melbourne Brain Centre Austin Hospital - Heidelberg
Recruitment hospital [2] 0 0
Children's Health Queensland Hospital and Health Service at Lady Cilento Children's Hospital - South Brisbane
Recruitment hospital [3] 0 0
The Children's Hospital Westmead Dept. of Neurology and Neurosurgery - 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
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United States of America
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Arizona
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California
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Ohio
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Utah
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Washington
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Belgium
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Edegem
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Montréal
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Canada
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Vancouver
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Denmark
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Dianalund
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France
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Amiens
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France
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Bordeaux
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France
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Lille
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Paris
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Berlin
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Germany
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Bielefeld
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Germany
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Freiburg
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Jena
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Germany
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Kiel
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Radeberg
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Germany
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Tübingen
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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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Milano
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Roma
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Verona
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Saitama
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Japan
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Shizuoka
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Tokyo
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Heeze
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Zwolle
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Barcelona
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Madrid
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Spain
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Pamplona
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Birmingham
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Glasgow
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Liverpool
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London
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United Kingdom
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Sheffield

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc.
Address
Country

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 prespecified 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: https://www.Vivli.org


What supporting documents are/will be available?




Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results are available at https://clinicaltrials.gov/study/NCT02823145