A Study to Assess the Safety and Tolerability of ZX008 in Children and Young Adults With Dravet Syndrome or Lennox Gastaut Syndrome Currently Taking Cannabidiol

Sponsor

Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc. (Industry)

Overall Status

Completed

CT.gov ID

NCT03467113

Collaborator

(none)

Enrollment

Locations

Arm

29.1

Actual Duration (Months)

4.5

Patients Per Site

0.2

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

This is an open label study to evaluate the safety of ZX008 (fenfluramine) in patients with Dravet syndrome (DS) or Lennox Gastaut syndrome (LGS) who are being administered cannabidiol (CBD).

Condition or Disease	Intervention/Treatment	Phase
Dravet Syndrome Lennox Gastaut Syndrome	Drug: ZX008 0.2 to 0.8 mg/kg/day Drug: Cannabidiol	Phase 1

Study Design

Study Type:

Interventional

Actual Enrollment :

9 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

An Open-Label Trial to Assess the Safety of ZX008 (Fenfluramine Hydrochloride) Oral Solution in Combination With Cannabidiol, as an Adjunctive Therapy in Children and Young Adults With Dravet Syndrome or Lennox-Gastaut Syndrome

Actual Study Start Date :

Jan 19, 2018

Actual Primary Completion Date :

Jun 24, 2020

Actual Study Completion Date :

Jun 24, 2020

Arms and Interventions

Arm	Intervention/Treatment
Experimental: ZX008 0.2 to 0.8 mg/kg/day ZX008 is supplied as an oral solution in a concentration of 2.5 mg/mL. Subjects will receive open-label ZX008 (flexible dosing 0.2 mg/kg/day to 0.8 mg/kg/day)	Drug: ZX008 0.2 to 0.8 mg/kg/day ZX008 drug is an oral aqueous solution of fenfluramine hydrochloride. The product is sugar free. Drug: Cannabidiol Cannabidiol

Arm

Intervention/Treatment

Experimental: ZX008 0.2 to 0.8 mg/kg/day

ZX008 is supplied as an oral solution in a concentration of 2.5 mg/mL. Subjects will receive open-label ZX008 (flexible dosing 0.2 mg/kg/day to 0.8 mg/kg/day)

Drug: ZX008 0.2 to 0.8 mg/kg/day

ZX008 drug is an oral aqueous solution of fenfluramine hydrochloride. The product is sugar free.

Drug: Cannabidiol

Cannabidiol

Outcome Measures

Primary Outcome Measures

Adverse Events [Baseline to Day 365]
Number of adverse events including serious adverse events, number of adverse events leading to study withdrawal
Change in Heart Rate [Baseline to Day 365]
Change in resting heart rate using standard measures
Change in blood pressure [Baseline to Day 365]
Change in resting blood pressure using standard measures
Change in temperature [Baseline to Day 365]
Change in resting temperature using standard measures
Change in respiratory rate [Baseline to Day 365]
Change in resting respiratory rate using standard measures
Changes in heart rhythm [Baseline to Day 365]
Changes in heart beat as measured with 12-lead electrocardiogram
Changes in heart valve function [Baseline to Day 365]
Changes in heart valves as measured with standard echocardiogram
Changes in treatment-emergent body weight and height [Baseline to Day 365]
Changes in body weight and height by report of BMI in kg/m^2

Eligibility Criteria

Criteria

Ages Eligible for Study:

2 Years to 18 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Key Inclusion Criteria:

Subject has a clinical diagnosis of DS or LGS, where convulsive seizures (generalized tonic clonic seizures, tonic seizures, atonic seizures, tonic/atonic seizures, focal seizures with clear observable motor signs) are not completely controlled by current AEDs.
All medications or interventions for epilepsy (including ketogenic diet [KD] and vagal nerve stimulation [VNS]) must be stable for at least 4 weeks prior to first ZX008 dose and are expected to remain stable throughout the study.
Subjects must be taking a stable dose of CBD for at least 4 weeks prior to first ZX008 dose.
Subject's source of CBD is expected to be consistent for at least 3 months during study participation.

Key Exclusion Criteria:

Subject has current or past history of cardiovascular or cerebrovascular disease, myocardial infarction or stroke.
Subject with current cardiac valvulopathy or pulmonary hypertension that the Investigator, parent/caregiver, Independent Cardiac Advisory Board, Independent Data and Safety Monitoring Committee, or sponsor deems clinically significant.
Subject has a current or past history of glaucoma.