NCT05064878

A Study to Investigate the Efficacy and Safety of ZX008 in Subjects With CDKL5 Deficiency Disorder

A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Fixed-Dose, Multicenter Study To Examine The Efficacy And Safety Of ZX008 In Subjects With CDKL5 Deficiency Disorder Followed By An Open-Label Extension

• Status: Active Not Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 87 (actual)
• Sponsor: Zogenix, Inc. · Industry
• Sex: All
• Age: 1 Year – 35 Years
• Healthy volunteers: Not accepted

Summary

This is a multicenter, double-blind, parallel-group, placebo controlled, 2-part study to evaluate the efficacy and safety of ZX008 when used as adjunctive therapy for the treatment of uncontrolled seizures in children and adults with cyclin-dependent kinase like-5 (CDKL5) deficiency disorder (CDD).

Detailed description

This is a 2-part multicenter trial. Part 1 is a 20-week randomized, double-blind, placebo-controlled, fixed-dose, parallel-group study to examine the efficacy and safety of ZX008 as an adjunctive therapy (to existing concomitant treatment with antiepileptic treatments \[AETs\]) in children and adults with a CDD diagnosis and uncontrolled seizures. Part 1 of the study is 20 weeks in duration and will consist of the following stages: Baseline Period (ie, Baseline \[BL\]; 4 weeks including the Screening Visit and baseline observation), Titration Period (ie, Titration; 2 weeks), Maintenance Period (ie, Maintenance; 12 weeks), and a 2-week Transition Period (ie, Transition; 2 weeks) to the open-label starting dose. Part 2 is a 54-week, open-label, flexible-dose, long-term extension for subjects who complete Part 1. Part 2 includes an Open-Label Extension (OLE) Treatment Period (52 weeks) with a Taper Period (ie, Taper; 2 weeks). The primary study analysis to evaluate the efficacy and safety of ZX008 in children and adults with CDD will be based on Part 1 data in all randomized subjects.

Conditions

• CDKL5 Deficiency Disorder
• Generalized Tonic Clonic Seizure
• Epileptic Spasm
• Refractory Seizures

Interventions

Type	Name	Description
DRUG	ZX008 (Fenfluramine Hydrochloride)	ZX008 is supplied as an oral aqueous solution of Fenfluramine Hydrochloride.
DRUG	Matching ZX008 Placebo	Matching ZX008 placebo is supplied as an oral solution.

Timeline

• Start date: 2022-03-08
• Primary completion: 2027-11-08
• Completion: 2027-11-08
• First posted: 2021-10-01
• Last updated: 2026-01-05

Locations

46 sites across 14 countries: United States, Austria, Belgium, Georgia, Germany, Ireland, Israel, Italy, Japan, Netherlands, Portugal, Spain, United Arab Emirates, United Kingdom

Regulatory

• FDA-regulated drug study