Dravet syndrome
Conditions
Interventions
investigational material(s)
Generic name etc : ZX008
INN of investigational material : fenfluramine hydrochloride
Therapeutic category code : 113 Antiepileptics
Dosage and Administration for Investiga
Sponsors
Zogenix International Limited
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: - Male or non-pregnant, non-lactating female, age 2 to 18 years, inclusive as of the day of the Screening Visit. - Clinical diagnosis of Dravet syndrome, where convulsive seizures are not completely controlled by current antiepileptic drugs. - Must have a minimum # of convulsive seizures per 4-week period for past 12 weeks prior to screening. - All medications or interventions for epilepsy (including KD and VNS) must be stable for at least 4 weeks prior to screening and are expected to remain stable throughout the study. - Parent/caregiver is willing and able to be compliant with diary completion, visit schedule and study drug accountability.
Exclusion criteria
Exclusion criteria: - Pulmonary arterial hypertension. - Current or past history of cardiovascular or cerebrovascular disease, such as cardiac valvulopathy, myocardial infarction or stroke. - Current or past history of glaucoma. - Moderate or severe hepatic impairment - Receiving concomitant therapy with: centrally-acting anorectic agents; monoamine-oxidase inhibitors; medications that act via serotonin including serotonin reuptake inhibitors; atomoxetine, or other centrally-acting noradrenergic agonist; cyproheptadine, - Currently taking carbamazepine, oxcarbamazepine, eslicarbazepine, phenobarbital, or phenytoin, or has taken any of these within the past 30 days. - Subject is unwilling to refrain from large or daily servings of grapefruits and/or Seville oranges, and their juices beginning with the Baseline Period and throughout the study. - 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. - Currently receiving an investigational product.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| efficacy Change from baseline in frequency of convulsive seizures in subjects receiving ZX008 0.8mg/kg/day compared to placebo | — |
Secondary
| Measure | Time frame |
|---|---|
| safety efficacy - Change from baseline in frequency of convulsive seizures for subjects receiving ZX008 0.2 mg/kg/day compared to placebo - Proportion of subjects achieving >=40% or >=50% reduction from baseline in convulsive seizure frequency and longest seizure-free interval in subjects receiving ZX008 0.2 and 0.8 mg/kg/day compared to placebo - Frequency and severity of seizure activity for subjects receiving ZX008 0.2 mg/kg/day and 0.8 mg/kg/day compared to placebo - Safety and tolerability of ZX008 0.2 and 0.8 mg/kg/day compared to placebo | — |
Countries
Australia, Europe, Japan
Outcome results
None listed