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Trial details imported from ClinicalTrials.gov

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




Registration number
NCT02823145
Ethics application status
Date submitted
13/06/2016
Date registered
6/07/2016
Date last updated
6/01/2020

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
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
Treatment: Drugs - ZX008 (Fenfluramine Hydrochloride)

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).


Treatment: Drugs: ZX008 (Fenfluramine Hydrochloride)


Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Long-term safety and tolerability as measured by treatment emergent adverse events, including clinical labs, vital signs, and examination findings. - Sensitivity of the key efficacy analysis will be assessed for changes in dose or type of concomitant AED medications
Timepoint [1] 0 0
Pre-baseline Up to 156 weeks
Secondary outcome [1] 0 0
Proportion of subjects who achieve reduction from baseline in convulsive seizure frequency
Timepoint [1] 0 0
Baseline up to 156 weeks
Secondary outcome [2] 0 0
The longest interval between convulsive seizures will be calculated for each subject over the entire open-label treatment period.
Timepoint [2] 0 0
Up to 156 weeks

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
Gender
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
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)
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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Arizona
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Utah
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Washington
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Belgium
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Edegem
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Lille
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Paris
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Bielefeld
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Freiburg
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Jena
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Kiel
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Radeberg
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Tübingen
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Germany
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Vogtareuth
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Genova
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Mantova
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Glasgow
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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
This is an international, multicenter, open-label, long-term safety study of ZX008 in
subjects with Dravet syndrome.
Trial website
https://clinicaltrials.gov/show/NCT02823145
Trial related presentations / publications
Public notes

Contacts
Principal investigator
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Contact person for public queries
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Contact person for scientific queries

Summary results
For IPD and results data, please see https://clinicaltrials.gov/show/NCT02823145