Clinical Trial: A Two-Part Study to Investigate the Dose-Ranging Safety and Pharmacokinetics, Followed by the Efficacy and Safety of ZX008 (Fenfluramine Hydrochloride) Oral Solution as an Adjunctive Therapy in Children ≥2 Years Old and Young Adults With Dravet Syndrome

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

Official Title: A Multicenter, 2-Cohort Trial to First Assess the Pharmacokinetic and Safety Profile of a Single Dose of ZX008 (Fenfluramine Hydrochloride) Oral Solution When Added to Standard of Care , Followed by a

Brief Summary: The primary purpose of this study is to evaluate the safety, tolerability and efficacy of a single dose of ZX008 (Fenfluramine Hydrochloride) when added to standard of care and when added to Adjunctive Antiepleptic therapy to Stiripentol treatment in children and young adults with Dravet Syndrome

Detailed Summary: This is a multicenter, two-cohort trial to assess the pharmacokinetic and safety profile of a single dose of ZX008 (fenfluramine hydrochloride) oral solution when added to Dravet syndrome treatment regimen containing VPA and CLB, with or without STP (Cohort 1), followed by a randomized, double-blind, placebo-controlled parallel group evaluation of the efficacy, safety, and tolerability of ZX008 as adjunctive therapy for seizures in children and young adults with Dravet syndrome (Cohort 2). Cohort 2 will not be dosed until the PK and safety data from Cohort 1 have been collected and evaluated. The PK and safety data from Cohort 1 will determine the dose of ZX008 to be used in Cohort 2.
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 0.2 mg/kg/day as adjunctive therapy compared to placebo [ Time Frame: Time between 6-week baseline assessment period and 12 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 in subjects receiving ZX008 0.4 mg/kg/day as adjunctive therapy compared to placebo [ Time Frame: Time between 6-week baseline assessment period and 12 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 0.4 mg/kg/day as adjunctive therapy compared to placebo [ Time Frame: Time between 6-week baseline assessment period and combined 12 week treatment and maintenance period ]
    Parent/caregiver seizure diary record will be used to assess frequency, type and duration of seizure activity


Original Secondary Outcome: Same as current

Information By: Zogenix, Inc.

Dates:
Date Received: August 10, 2016
Date Started: September 2016
Date Completion: August 2017
Last Updated: May 1, 2017
Last Verified: May 2017