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

Brief Title

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

Official 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

Brief Summary

      This is a multicenter, double-blind, parallel-group, placebo-controlled, study to assess the
      efficacy, safety, and pharmacokinetics of ZX008 when used as adjunctive therapy in pediatric
      and young adult subjects with Dravet syndrome. Subjects who qualify for the study will be
      randomized (1:1:1) in a double-blind manner to receive 1 of 2 doses of ZX008 or placebo. All
      subjects will be titrated to their randomized dose over a 14-day Titration Period. Following
      titration, subjects will continue treatment at their randomly assigned dose over a 12-week
      Maintenance Period. Total treatment time from the beginning of the Titration Period through
      the end of the Maintenance Period is 14 weeks.
    

Study Phase

Phase 3

Study Type

Interventional

Primary Outcome

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

Secondary Outcome

 Change from baseline in frequency of convulsive seizures for subjects receiving ZX008 0.2mg/kg/day compared to placebo

Condition

Dravet Syndrome

Intervention

ZX008 - 0.8 mg/kg/day

Study Arms / Comparison Groups

 Experimental: ZX008 - 0.8 mg/kg/day

Description:  ZX008 is supplied as an oral solution in concentrations of 1.25, 2.5, and 5 mg/mL. ZX008 will be administered twice a day (BID) in equally divided doses with food.

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Status

Drug

Estimated Enrollment

130

Start Date

July 15, 2016

Completion Date

July 2020

Primary Completion Date

July 2020

Eligibility 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.
      

Gender

All

Ages

2 Years - 18 Years

Accepts Healthy Volunteers

No

Contacts

, 510-388-9968, [email protected]

Location Countries

Australia

Location Countries

Australia

NCT ID

NCT02826863

Organization ID

ZX008-1502

Responsible Party

Sponsor

Study Sponsor

Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc.

Study Sponsor

, , 

Verification Date

January 2020