ZX008 in CDKL5 Deficiency Disorder

• Research type

  Research Study

• Full title

  A PHASE 3, RANDOMIZED, DOUBLE-BLIND, PLACEBOCONTROLLED, FIXED-DOSE, MULTICENTER STUDY TO EXAMINE THE EFFICACY AND SAFETY OF ZX008 IN SUBJECTS WITH CDKL5 DEFICIENCY DISORDER FOLLOWED BY AN OPEN-LABEL EXTENSION

• IRAS ID

  1004438

• Contact name

  Gavin Barker

• Contact email

  gbarker@zogenix.com

• Sponsor organisation

  Zogenix International Limited

• Eudract number

  2021-003222-76

• Clinicaltrials.gov Identifier

  NCT05064878

• Research summary

  The purpose of this phase 3 study is to learn about the efficacy and safety of the medication, fenfluramine hydrochloride, otherwise known as ZX008, for the treatment of uncontrolled seizures in children and adults with cyclin-dependent kinase like-5 (CDKL5) deficiency disorder (CDD). This study will enrol participants meeting the CDD diagnostic criteria. This is a two part, multicentre trial. Part 1 is a 20 week, randomised, double bind, placebo controlled study, to examine the efficacy and safety of ZX008 as an adjunctive therapy. Here, two cohorts will be randomised to receive either the ZX008 medication or placebo. Part 2 is a 54 week, open label, flexible dose, long term extension for participants who complete part 1.

  The objectives for these parts of the study are as follows:
  - Part 1: To determine if ZX008 is superior to placebo as an adjunctive therapy for CDD, by measuring seizure frequency in participants. This part will also aim to characterise the safety and tolerability of ZX008 in subjects with CDD, that is, the ability of the participant’s body to accept ZX008, and how their body may react to it.
  - Part 2: To assess the long-term effectiveness of ZX008 in patients with CDD by measuring seizure frequency, and to characterise the long-term safety of ZX008 in patients with CDD.

  Participants may receive study treatments for up to two years, depending on their ability to tolerate treatment, and their doctor’s decision to continue treatment. Tests and procedures involved in this study include: physical and neurological examinations, vital signs (blood pressure, breathing rate, heart rate), and blood and biological sample collection, amongst other possible tests.
  This study is conducted in North America, Europe, UK and Japan. It is sponsored by the pharmaceutical company Zogenix, Inc.

• REC name

  East Midlands - Derby Research Ethics Committee

• REC reference

  22/EM/0089

• Date of REC Opinion

  7 Jun 2022

• REC opinion

  Further Information Favourable Opinion