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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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03467113
Recruitment Status : Completed
First Posted : March 15, 2018
Last Update Posted : May 24, 2022
Sponsor:
Information provided by (Responsible Party):
Zogenix, Inc. ( Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc. )

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

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Study Type : Interventional  (Clinical Trial)
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 : January 19, 2018
Actual Primary Completion Date : June 24, 2020
Actual Study Completion Date : June 24, 2020


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




Primary Outcome Measures :
  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



Information from the National Library of Medicine

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Ages Eligible for Study:   2 Years to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03467113


Locations
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United States, Georgia
PANDA Neurology/CIRCA
Atlanta, Georgia, United States, 30328
United States, Washington
MultiCare Institute for Research & Innovation
Tacoma, Washington, United States, 98405
Sponsors and Collaborators
Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc.
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Responsible Party: Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc.
ClinicalTrials.gov Identifier: NCT03467113    
Other Study ID Numbers: ZX008-1602
First Posted: March 15, 2018    Key Record Dates
Last Update Posted: May 24, 2022
Last Verified: May 2022

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Zogenix, Inc. ( Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc. ):
Dravet Syndrome
LGS
Additional relevant MeSH terms:
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Epilepsies, Myoclonic
Lennox Gastaut Syndrome
Syndrome
Disease
Pathologic Processes
Epilepsy, Generalized
Epilepsy
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Epileptic Syndromes
Genetic Diseases, Inborn
Cannabidiol
Anticonvulsants