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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT03355209




Registration number
NCT03355209
Ethics application status
Date submitted
19/06/2017
Date registered
28/11/2017

Titles & IDs
Public title
A Study to Investigate the Efficacy and Safety of ZX008 (Fenfluramine Hydrochloride) as an Adjunctive Therapy in Children and Adults With Lennox-Gastaut Syndrome
Scientific title
A Two-Part Study of ZX008 in Children and Adults With Lennox-Gastaut Syndrome (LGS); Part 1: A Randomized, Double-blind, Placebo-controlled Trial of Two Fixed Doses of ZX008 (Fenfluramine Hydrochloride) Oral Solution as Adjunctive Therapy for Seizures in Children and Adults With LGS, Followed by Part 2: An Open-label Extension to Assess Long-Term Safety of ZX008 in Children and Adults With LGS
Secondary ID [1] 0 0
2017-002628-26
Secondary ID [2] 0 0
ZX008-1601
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Lennox Gastaut Syndrome 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 0.2 or 0.8 mg/kg/day
Treatment: Drugs - Matching Placebo

Experimental: ZX008 0.2 or 0.8 mg/kg/day - Part 1: ZX008 is supplied as an oral solution. Subjects will be randomized to receive 1 of 2 doses of ZX008 0.2 mg/kg/day or 0.8 mg/kg/day.

Placebo comparator: Matching Placebo - Part 1: Matching ZX008 placebo is supplied as an oral solution.

Experimental: Open-Label - Part 2: ZX008 is supplied as an oral solution. Study medication will be administered twice a day (BID) in equally divided doses.


Treatment: Drugs: ZX008 0.2 or 0.8 mg/kg/day
ZX008 drug product is an oral aqueous solution of fenfluramine hydrochloride. The product is sugar free and is intended to be compatible with a Ketogenic Diet.

Treatment: Drugs: Matching Placebo
Placebo will be administered twice a day (BID) in equally divided doses.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Part 1: Change from baseline in frequency of seizures that result in drops in subjects receiving ZX008 compared to placebo
Timepoint [1] 0 0
Up to 20 weeks maintenance and taper period (T+M)
Secondary outcome [1] 0 0
Part 2: Adverse events and related safety parameters in subjects receiving ZX008 compared to placebo
Timepoint [1] 0 0
Up to 12 months open label

Eligibility
Key inclusion criteria
Key

* Male or non-pregnant, non-lactating female, age 2 to 35 years, inclusive as of the day of the Screening Visit.
* Clinical diagnosis of Lennox-Gastaut syndrome, where seizures that result in drops are not completely controlled by current antiepileptic treatments.
* Onset of seizures at 11 years of age or younger.
* Abnormal cognitive development.
* Must be receiving at least 1 concomitant AED and up to 4 concomitant anti-epileptic treatments.

Key
Minimum age
2 Years
Maximum age
35 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* Etiology of seizures is a degenerative neurological disease.
* History of hemiclonic seizures in the first year of life.
* Subject only has drop seizures in clusters, where individual seizures cannot be counted reliably.
* Pulmonary arterial hypertension.
* Current or past history of cardiovascular or cerebrovascular disease, such as cardiac valvulopathy, myocardial infarction or stroke.
* 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.
* Taking felbamate for less than 1 year prior to screening and/or does not have stable liver function and hematology laboratory tests, and/or the dose has not been stable for at least 60 days prior to the Screening Visit.
* Currently receiving an investigational product.
* Institutionalized in a general nursing home (ie, in a facility that does not specialize in epilepsy care).
* 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.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
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
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
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
27/11/2017
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
23/05/2024
Sample size
Target
Accrual to date
Final
296
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Ep0214 301 - Heidelberg
Recruitment hospital [2] 0 0
Ep0214 302 - South Brisbane
Recruitment postcode(s) [1] 0 0
- Heidelberg
Recruitment postcode(s) [2] 0 0
- South Brisbane
Recruitment outside Australia
Country [1] 0 0
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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Colorado
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District of Columbia
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Florida
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Georgia
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Illinois
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Maryland
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Massachusetts
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Michigan
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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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Oregon
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Pennsylvania
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Texas
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Utah
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Washington
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Belgium
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Brussels
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Belgium
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Bruxelles
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Belgium
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Edegem
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Canada
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Toronto
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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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Bordeaux Cedex
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France
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Bron
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France
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Lille Cedex
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France
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Marseilles
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France
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Paris
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Germany
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Bielefeld
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Germany
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Freiburg
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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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Bologna
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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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Roma
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Japan
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Fukuoka
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Japan
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Niigata-city
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Japan
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Okayama
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Japan
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Omura
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Japan
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Osaka
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Japan
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Sapporo-city
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Japan
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Shinjuku-ku
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Japan
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Shizuoka
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Mexico
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Guadalajara
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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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Mirasierra
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Spain
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Pamplona
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Sweden
State/province [56] 0 0
Göteborg

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 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: https://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/NCT03355209