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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT03936777




Registration number
NCT03936777
Ethics application status
Date submitted
12/04/2019
Date registered
3/05/2019
Date last updated
17/09/2020

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
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 - Evaluate the long term safety and tolerability of oral dose administration of ZX008
Timepoint [1] 0 0
Up to 36 months open-label
Primary outcome [2] 0 0
Changes in laboratory test results - The analysis will include abnormal hematology and chemistry laboratory results outside of reference ranges
Timepoint [2] 0 0
Up to 36 months open-label
Primary outcome [3] 0 0
Changes in heart rate - The analysis will include change in heart rate using standard measure
Timepoint [3] 0 0
Up to 36 months open-label
Primary outcome [4] 0 0
Changes in respiratory rate - The analysis will include change in resting respiratory rate using standard measure
Timepoint [4] 0 0
Up to 36 months open-label
Primary outcome [5] 0 0
Changes in blood pressure - The analysis will include change in resting blood pressure using standard measure
Timepoint [5] 0 0
Up to 36 months open-label
Primary outcome [6] 0 0
Change in body weight - The analysis will include change in body weight and height by report of BMI in kg/m^2
Timepoint [6] 0 0
Up to 36 months open-label
Primary outcome [7] 0 0
Changes in heart rhythm - The analysis will include changes in heart beat as measured with 12-lead electrocardiogram
Timepoint [7] 0 0
Up to 36 months open-label
Primary outcome [8] 0 0
Changes in heart valve function - The analysis will include change in heart valves as measured with standard echocardiogram
Timepoint [8] 0 0
Up to 36 months open-label
Secondary outcome [1] 0 0
Change in convulsive seizure response - The analysis will include percent improvement per investigator rating
Timepoint [1] 0 0
Up to 36 months open-label
Secondary outcome [2] 0 0
Change in Cognitive CGI by Parent/Caregiver - 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".
Timepoint [2] 0 0
Up to 36 months open-label
Secondary outcome [3] 0 0
Change in Behavioral CGI by Parent/Caregiver - 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".
Timepoint [3] 0 0
Up to 36 months open-label
Secondary outcome [4] 0 0
Change in Motor CGI by Parent/Caregiver - 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".
Timepoint [4] 0 0
Up to 36 months open-label
Secondary outcome [5] 0 0
Change in Cognitive CGI by Investigator - 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".
Timepoint [5] 0 0
Up to 36 months open-label
Secondary outcome [6] 0 0
Change in Behavioral CGI by Investigator - 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".
Timepoint [6] 0 0
Up to 36 months open-label
Secondary outcome [7] 0 0
Change in Motor CGI by Investigator - 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".
Timepoint [7] 0 0
Up to 36 months open-label
Secondary outcome [8] 0 0
Change in Clinical Global Impression by Parent/Caregiver - 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"
Timepoint [8] 0 0
Up to 36 months open-label
Secondary outcome [9] 0 0
Change in Clinical Global Impression by Investigator - 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"
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
Gender
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
N/A
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
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
QLD
Recruitment hospital [1] 0 0
Queensland Children's Hospital - South Brisbane
Recruitment postcode(s) [1] 0 0
4101 - South Brisbane
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arizona
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
Colorado
Country [4] 0 0
United States of America
State/province [4] 0 0
Florida
Country [5] 0 0
United States of America
State/province [5] 0 0
Georgia
Country [6] 0 0
United States of America
State/province [6] 0 0
Illinois
Country [7] 0 0
United States of America
State/province [7] 0 0
Maryland
Country [8] 0 0
United States of America
State/province [8] 0 0
Massachusetts
Country [9] 0 0
United States of America
State/province [9] 0 0
Minnesota
Country [10] 0 0
United States of America
State/province [10] 0 0
New Jersey
Country [11] 0 0
United States of America
State/province [11] 0 0
New York
Country [12] 0 0
United States of America
State/province [12] 0 0
Ohio
Country [13] 0 0
United States of America
State/province [13] 0 0
Texas
Country [14] 0 0
United States of America
State/province [14] 0 0
Utah
Country [15] 0 0
United States of America
State/province [15] 0 0
Washington
Country [16] 0 0
Belgium
State/province [16] 0 0
Antwerp
Country [17] 0 0
Canada
State/province [17] 0 0
British Columbia
Country [18] 0 0
Denmark
State/province [18] 0 0
Dianalund
Country [19] 0 0
Spain
State/province [19] 0 0
Madrid
Country [20] 0 0
Spain
State/province [20] 0 0
Pamplona
Country [21] 0 0
United Kingdom
State/province [21] 0 0
Birmingham
Country [22] 0 0
United Kingdom
State/province [22] 0 0
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
This is an international, multicenter, open-label, long-term safety study of ZX008 in
subjects with Dravet syndrome, Lennox-Gastaut syndrome or epileptic encephalopathy
Trial website
https://clinicaltrials.gov/show/NCT03936777
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
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/show/NCT03936777