Efficacy & Safety of ZX008 as Adjunctive therapy in Dravet Syndrome
Research type
Research Study
Full 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 (Cohort 1), Followed by a Randomized, Double-blind, Placebo-controlled Parallel Group Evaluation of the Efficacy, Safety, and Tolerability of ZX008 as Adjunctive Antiepileptic Therapy to Stiripentol Treatment in Children and Young Adults with Dravet Syndrome (Cohort 2)
IRAS ID
213673
Contact name
Sameer Zuberi
Contact email
Sponsor organisation
Zogenix International Limited, A wholly owned subsidiary of Zogenix, Inc.
Eudract number
2016-000474-38
Duration of Study in the UK
0 years, 8 months, 1 days
Research summary
This is a randomised, double-blind, placebo-controlled parallel group study to test the efficacy, safety and tolerability of ZX008 as add-on therapy in children and young adults with Dravet syndrome also known as severe myoclonic epilepsy of infancy (SMEI), a type of epilepsy with seizures.
The current study is cohort 2 following on from cohort 1 that assed the pharmacokinetics (the action of drugs in the body) and then the safety profile of a single dose of ZX008, the study drug, an oral solution added to the standard treatment for Dravet syndrome.
The 6 week Baseline Period of Cohort 2 will establish eligibility. Upon completion of the Baseline Period, patients will be randomised (1:1) in a double-blind (neither patients nor investigators will know which drug the patient is receiving) manner to receive ZX008. The drug dose is determined from Cohort 1 of the study.
Approximately 85 patients aged 2 to 18 years will be screened to obtain 80 patients who enter the Baseline Period. Of these 80, it is estimated that at least 70 patients will be randomised into the study. Patients who discontinue from the clinical investigation for any reason will not be replaced.
All patients will receive ZX008 or placebo (drug with no active ingredient) for up to approximately 16 weeks. After completion of the Maintenance Period, eligible patients may enroll in a separate open-label extension study. Patients who will continue into the open-label extension trial will participate in a transition period of 14 days. Patients who do not participate in the open-label extension trial will undergo a taper period of 14 days, after which they will be off study medication. For patients who do not enroll in the separate long-term extension, there will be a follow-up visit for cardiovascular safety monitoring (including ECG and ECHO) 3-6 months after the last dose of study medication.REC name
Scotland A REC
REC reference
17/SS/0021
Date of REC Opinion
6 Apr 2017
REC opinion
Favourable Opinion