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

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

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).

Study Overview

• Status: Completed
• Conditions: Dravet Syndrome; Lennox Gastaut Syndrome
• Intervention / Treatment: Drug: Cannabidiol; Drug: ZX008 0.2 to 0.8 mg/kg/day

• Study Type: Interventional
• Enrollment (Actual): 9
• Phase: Phase 1

Contacts and Locations

Study Locations

• United States
  • Georgia
    • Atlanta, Georgia, United States, 30328
      • PANDA Neurology/CIRCA
  • Washington
    • Tacoma, Washington, United States, 98405
      • MultiCare Institute for Research & Innovation

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 1 year to 14 years (Child, Adult)
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / 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: Cannabidiol; Cannabidiol; 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.

What is the study measuring?

Primary Outcome Measures

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

Collaborators and Investigators

• Sponsor: Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc.

Study record dates

Study Major Dates

• Study Start (Actual): January 19, 2018
• Primary Completion (Actual): June 24, 2020
• Study Completion (Actual): June 24, 2020

Study Registration Dates

• First Submitted: January 30, 2018
• First Submitted That Met QC Criteria: March 14, 2018
• First Posted (Actual): March 15, 2018

Study Record Updates

• Last Update Posted (Actual): December 1, 2023
• Last Update Submitted That Met QC Criteria: November 30, 2023
• Last Verified: November 1, 2023

More Information

Terms related to this study

• Keywords: LGS; Dravet Syndrome
• Additional Relevant MeSH Terms: Pathologic Processes; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Epilepsy, Generalized; Epileptic Syndromes; Disease; Genetic Diseases, Inborn; Epilepsy; Epilepsies, Myoclonic; Syndrome; Lennox Gastaut Syndrome; Anticonvulsants; Cannabidiol
• Other Study ID Numbers: ZX008-1602

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No