MedPath

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

Phase 3
Completed
Conditions
Dravet syndrome
Registration Number
JPRN-jRCT2080224792
Lead Sponsor
Zogenix International Limited
Brief Summary

Not available

Detailed Description

Not available

Recruitment & Eligibility

Status
completed
Sex
All
Target Recruitment
12
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

- 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.

Study & Design

Study Type
Interventional
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
efficacy<br>Change from baseline in frequency of convulsive seizures in subjects receiving ZX008 0.8mg/kg/day compared to placebo
Secondary Outcome Measures
NameTimeMethod
safety<br>efficacy<br>- Change from baseline in frequency of convulsive seizures for subjects receiving ZX008 0.2 mg/kg/day compared to placebo<br>- 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<br>- Frequency and severity of seizure activity for subjects receiving ZX008 0.2 mg/kg/day and 0.8 mg/kg/day compared to placebo<br>- Safety and tolerability of ZX008 0.2 and 0.8 mg/kg/day compared to placebo
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