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

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT02826863
• Date of registration: 13/06/2016
• Prospective Registration: Yes
• Primary sponsor: Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc.
• Public title: A Trial of Two Fixed Doses of ZX008 (Fenfluramine HCl) as an Adjunctive Therapy in Children and Young Adults With Dravet Syndrome
• Scientific title: A Multicenter, Randomized, Double-blind, Parallel Group, Placebo-controlled Trial of Two Fixed Doses of ZX008 (Fenfluramine Hydrochloride) Oral Solution as an Adjunctive Therapy in Children and Young Adults With Dravet Syndrome
• Date of first enrolment: July 15, 2016
• Target sample size: 130
• Recruitment status: Recruiting
• URL: https://clinicaltrials.gov/show/NCT02826863
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 3

Countries of recruitment

Australia, Belgium, Denmark, France, Germany, Italy, Japan, Norway, Spain, Sweden, United Kingdom

Contacts

• Name: ZX008 Clinical Trials Information Desk

Key inclusion & exclusion criteria

Key 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.

Key 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, and/or 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.

- 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.



Age minimum: 2 Years
Age maximum: 18 Years
Gender: All

Health Condition(s) or Problem(s) studied

Dravet Syndrome

Intervention(s)

Drug: Placebo

Drug: ZX008 - 0.2 mg/kg/day

Drug: ZX008 - 0.8 mg/kg/day

Primary Outcome(s)

Change from baseline in frequency of convulsive seizures in subjects receiving ZX008 0.8mg/kg/day compared to placebo [Time Frame: Time between 6-week baseline assessment period and 14 week treatment and maintenance period]

Secondary Outcome(s)

Change from baseline in frequency of convulsive seizures for subjects receiving ZX008 0.2mg/kg/day compared to placebo [Time Frame: Time between 6-week baseline assessment period and 14 week treatment and maintenance period]

Proportion of subjects achieving a =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 [Time Frame: Time between 6-week baseline assessment period and combined 14 week treatment and maintenance period]

Frequency and severity of seizure activity for subjects receiving ZX008 0.2mg/kg/day and 0.8mg/kg/day compared to placebo [Time Frame: Time between 6-week baseline assessment period and 14 week treatment and maintenance period]

Safety and tolerability of ZX008 0.2 and 0.8 mg/kg/day compared to placebo [Time Frame: Week 1 through Week 14]

Secondary ID(s)

ZX008-1502

Source(s) of Monetary Support

Please refer to primary and secondary sponsors