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Completed

NCT Number: NCT03467113

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

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).

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Key information

Conditions

Dravet Syndrome Brain Diseases Central Nervous System Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Epilepsies, Myoclonic Epilepsy Epilepsy, Generalized Epileptic Syndromes Genetic Diseases, Inborn Lennox Gastaut Syndrome Nervous System Diseases

Age range

2 year–18 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

PANDA Neurology/CIRCA, Atlanta, Georgia, United States

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

ZX008 0.2 to 0.8 mg/kg/day

Drug

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

Cannabidiol

Drug

Cannabidiol

Primary outcomes

  1. Adverse Events

    Time frame: Baseline to Day 365

    Number of adverse events including serious adverse events, number of adverse events leading to study withdrawal

  2. Change in Heart Rate

    Time frame: Baseline to Day 365

    Change in resting heart rate using standard measures

  3. Change in blood pressure

    Time frame: Baseline to Day 365

    Change in resting blood pressure using standard measures

  4. Change in temperature

    Time frame: Baseline to Day 365

    Change in resting temperature using standard measures

  5. Change in respiratory rate

    Time frame: Baseline to Day 365

    Change in resting respiratory rate using standard measures

  6. Changes in heart rhythm

    Time frame: Baseline to Day 365

    Changes in heart beat as measured with 12-lead electrocardiogram

  7. Changes in heart valve function

    Time frame: Baseline to Day 365

    Changes in heart valves as measured with standard echocardiogram

  8. Changes in treatment-emergent body weight and height

    Time frame: Baseline to Day 365

    Changes in body weight and height by report of BMI in kg/m^2

Sponsors and collaborators

Lead sponsor

Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc.

Industry

Registry information

Official study 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

Important dates

Study start
2018
Primary completion
2020
Study completion
2020
First posted
Mar 15, 2018
Registry last updated
Dec 1, 2023

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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