NCT02682927

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

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

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 262 (actual)
• Sponsor: Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc. · Industry
• Sex: All
• Age: 2 Years – 18 Years
• Healthy volunteers: Not accepted

Summary

Study 1 and Study 3 are the prospective, merged analyses of 2 identical double-blind, placebo-controlled studies, ZX008-1501 and ZX008-1502, to assess the efficacy, safety, and pharmacokinetics of ZX008 when used as adjunctive therapy in pediatric and young adult subjects with Dravet syndrome. Study 1501 and Study 1502 were conducted in parallel; Study 1501 was conducted at approximately 30 study sites in North America; Study 1502 was conducted at approximately 30 study sites in Europe, Asia and Australia. Upon completion of the Baseline Period after initial Screening and Baseline charting of seizure frequency, subjects who qualified for the studies were randomized (1:1:1) in a double-blind manner to receive either 1 of 2 doses of ZX008 (0.2 mg/kg/day or 0.8 mg/kg/day; maximum dose: 30 mg/day) or placebo. Randomization was stratified by age group (\< 6 years, ≥6 to 18 years) to achieve balance across treatment arms, with the target of 25% of subjects in each age group. All subjects were titrated to their randomized dose over a 14-day Titration Period. Following titration, subjects continued treatment at their randomly assigned dose over a 12-week Maintenance Period. Subjects exiting the study underwent a 2-week taper, unless they enrolled in a follow-on study. Subjects were followed for post-study safety monitoring.

Conditions

• Dravet Syndrome
• Seizure Disorder

Interventions

Type	Name	Description
DRUG	ZX008 (Fenfluramine Hydrochloride)	ZX008 drug product is an oral aqueous solution of fenfluramine hydrochloride buffered to pH 5. The product is sugar free and is intended to be compatible with a ketogenic diet.
DRUG	Matching Placebo	Placebo solution for ZX008. The product is sugar free and is intended to be compatible with a ketogenic diet.

Timeline

• Start date: 2016-01-15
• Primary completion: 2020-07-29
• Completion: 2020-07-29
• First posted: 2016-02-17
• Last updated: 2023-09-28
• Results posted: 2022-10-19

Locations

55 sites across 11 countries: United States, Australia, Belgium, Canada, Denmark, France, Germany, Italy, Japan, Spain, United Kingdom

Regulatory

• FDA-regulated drug study