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

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

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

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.

Detailed Summary:
Sponsor: Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc.

Current Primary Outcome: 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 ]

Parent/caregiver seizure diary record will be used to assess frequency, type and duration of seizure activity


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • 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 ]
    Parent/caregiver seizure diary record will be used to assess frequency, type and duration of seizure activity
  • 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 ]
    Parent/caregiver seizure diary record will be used to assess frequency, type and duration of seizure activity
  • 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 ]
    Seizure severity evaluated using parent/caregiver seizure diary to record frequency and severity of seizure activity
  • Safety and tolerability of ZX008 0.2 and 0.8 mg/kg/day compared to placebo [ Time Frame: Week 1 through Week 14 ]
    Safety and tolerability will be evaluated by reported adverse events, laboratory parameters, physical and neurological examination, vital signs, electrocardiograms, echocardiograms, and body weight. (Cognitive function will be assessed using age-appropriate versions of the Brief Rating Inventory of Executive Function [BRIEF].)


Original Secondary Outcome:

  • 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 ]
    Parent/caregiver seizure diary record will be used to assess frequency, type and duration of seizure activity
  • 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 ]
    Parent/caregiver seizure diary record will be used to assess frequency, type and duration of seizure activity
  • 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 ]
    Seizure severity evaluated using parent/caregiver seizure diary to record frequency and severity of seizure activity
  • Safety and tolerability of ZX008 0.2 and 0.8 mg/kg/day compared to placebo [ Time Frame: Week 1 through Week 14 ]
    Safety and tolerability evaluated by reported adverse events, laboratory parameters, physical and neurological examination, vital signs, electrocardiograms, echocardiograms, and body weight. (Cognitive function will be assessed using age-appropriate versions of the Brief Rating Inventory of Executive Function [BRIEF].)


Information By: Zogenix, Inc.

Dates:
Date Received: June 13, 2016
Date Started: June 2016
Date Completion: July 2017
Last Updated: May 7, 2017
Last Verified: May 2017