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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
Date last updated
25/06/2024

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
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United States of America
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Arizona
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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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Illinois
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Massachusetts
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Minnesota
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Oregon
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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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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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France
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Bron
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Marseilles
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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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Okayama
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Omura
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Osaka
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Sapporo-city
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Shinjuku-ku
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Shizuoka
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Guadalajara
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Zwolle
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Bydgoszcz
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Barcelona
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Mirasierra
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Pamplona
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Sweden
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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
This is a two-part, multicenter, double-blind, parallel-group, placebo controlled study to evaluate the effect of ZX008 when used as adjunctive therapy for the treatment of uncontrolled seizures in children and adults with Lennox-Gastaut syndrome (LGS).
Trial website
https://clinicaltrials.gov/study/NCT03355209
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



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT03355209