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

• Register: ClinicalTrials.gov
• Last refreshed on: 11 December 2023
• Main ID: NCT03467113
• Date of registration: 30/01/2018
• Prospective Registration: No
• Primary sponsor: Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc.
• Public title: 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
• Scientific 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
• Date of first enrolment: January 19, 2018
• Target sample size: 9
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/ct2/show/NCT03467113
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 1

Countries of recruitment

United States

Key inclusion & exclusion criteria

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.



Age minimum: 2 Years
Age maximum: 18 Years
Gender: All

Health Condition(s) or Problem(s) studied

Dravet Syndrome

Lennox Gastaut Syndrome

Intervention(s)

Drug: Cannabidiol

Drug: ZX008 0.2 to 0.8 mg/kg/day

Primary Outcome(s)

Change in temperature [Time Frame: Baseline to Day 365]

Adverse Events [Time Frame: Baseline to Day 365]

Changes in treatment-emergent body weight and height [Time Frame: Baseline to Day 365]

Change in Heart Rate [Time Frame: Baseline to Day 365]

Changes in heart valve function [Time Frame: Baseline to Day 365]

Change in blood pressure [Time Frame: Baseline to Day 365]

Change in respiratory rate [Time Frame: Baseline to Day 365]

Changes in heart rhythm [Time Frame: Baseline to Day 365]

Secondary ID(s)

ZX008-1602

Source(s) of Monetary Support

Please refer to primary and secondary sponsors