A Trial of Two Fixed Doses of ZX008 (Fenfluramine HCl) as an Adjunctive Therapy in Children and Young Adults With Dravet Syndrome

JRCT ID: jRCT2080224792

Registered date:19/07/2019

Basic Information

• Recruitment status: completed
• Health condition(s) or Problem(s) studied: Dravet syndrome
• Date of first enrollment: 31/07/2019
• Target sample size: 12
• Countries of recruitment: Europe,Japan,Australia
• Study type: Interventional

Intervention(s)

investigational material(s)
Generic name etc : ZX008
INN of investigational material : fenfluramine hydrochloride
Therapeutic category code : 113 Antiepileptics
Dosage and Administration for Investigational material : Oral administration

control material(s)
Generic name etc : Placebo
INN of investigational material : -
Therapeutic category code : --- Other
Dosage and Administration for Investigational material : -

Outcome(s)

Primary Outcome

efficacy
Change from baseline in frequency of convulsive seizures in subjects receiving ZX008 0.8mg/kg/day compared to placebo

Secondary Outcome

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

Key inclusion & exclusion criteria

• Age minimum: >= 2age old
• Age maximum: <= 18age old
• Gender: Both

Include 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.

Exclude 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.

Related Information

• Primary Sponsor: Zogenix International Limited
• Secondary Sponsor: -
• Source(s) of Monetary Support: -
• Secondary ID(s): JapicCTI-194872