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

Dravet Syndrome Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03467113
• Other study ID #: ZX008-1602
• Status: Completed
• Phase: Phase 1
• Start date: January 19, 2018
• Est. completion date: June 24, 2020

Study information

• Verified date: November 2023
• Source: UCB Pharma
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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).

Recruitment information / eligibility

• Status: Completed
• Enrollment: 9
• Est. completion date: June 24, 2020
• Est. primary completion date: June 24, 2020
• Accepts healthy volunteers: No
• Gender: All
• Age group: 2 Years to 18 Years

Eligibility

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.

Related Conditions & MeSH terms

• Dravet Syndrome
• Epilepsies, Myoclonic
• Lennox Gastaut Syndrome
• Syndrome

Intervention

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.
• Cannabidiol
Cannabidiol

Locations

Country	Name	City	State
United States	PANDA Neurology/CIRCA	Atlanta	Georgia
United States	MultiCare Institute for Research & Innovation	Tacoma	Washington

Sponsors (1)

Lead Sponsor	Collaborator
Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Adverse Events	Number of adverse events including serious adverse events, number of adverse events leading to study withdrawal	Baseline to Day 365
Primary	Change in Heart Rate	Change in resting heart rate using standard measures	Baseline to Day 365
Primary	Change in blood pressure	Change in resting blood pressure using standard measures	Baseline to Day 365
Primary	Change in temperature	Change in resting temperature using standard measures	Baseline to Day 365
Primary	Change in respiratory rate	Change in resting respiratory rate using standard measures	Baseline to Day 365
Primary	Changes in heart rhythm	Changes in heart beat as measured with 12-lead electrocardiogram	Baseline to Day 365
Primary	Changes in heart valve function	Changes in heart valves as measured with standard echocardiogram	Baseline to Day 365
Primary	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