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A Study to Assess the Safety and Tolerability of ZX008 in Children and Young Adults With DS or LGS Currently Taking CBD

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 : Active, not recruiting
First Posted : March 15, 2018
Last Update Posted : August 14, 2018
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 DS or LGS who are being administered CBD.

Condition or disease Intervention/treatment Phase
Dravet Syndrome Lennox Gastaut Syndrome Drug: ZX008 02 and 0.8 mg/kg/day Phase 1 Phase 2

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 25 participants
Intervention Model: Single Group Assignment
Intervention Model Description: 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
Estimated Primary Completion Date : February 2019
Estimated Study Completion Date : February 2019


Arm Intervention/treatment
Experimental: ZX008 0.2 and 0.8 mg/kg/day
FZX008 is supplied as an oral solution in a concentration of 2.5 mg/mL. Subjects will receive open-label ZX008 (0.2 mg/kg/day titrated to 0.8 mg/kg/day)
Drug: ZX008 02 and 0.8 mg/kg/day
ZX008 drug is an oral aqueous solution of fenfluramine hydrochloride. The product is sugar free.




Primary Outcome Measures :
  1. Adverse events [ Time Frame: Baseline to Day 365 ]
    Number of AEs including SAEs, number of AEs leading to study withdrawal

  2. Change in heart rate [ Time Frame: Baseline to Day 365 ]
    Change in resting HR using standard measures

  3. Change in blood pressure [ Time Frame: Baseline to Day 365 ]
    Change in resting BP 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


Secondary Outcome Measures :
  1. Maximum Plasma Concentration (Cmax) will be assessed [ Time Frame: Day 12-15 and, 3 months to 12 months ]
    PK analyses will be performed on the concentration measurement of fenfluramine and its metabolite (norfenfluramine) and the effect on the concentration of CBD by measuring the Cmax

  2. Area Under the Curve (AUC) will be assessed [ Time Frame: Day 12-15 and, 3 months to 12 months ]
    PK analyses will be performed on the concentration measurement of fenfluramine and its metabolite (norfenfluramine) and the effect on the concentration of CBD by measuring the AUC

  3. Time to Maximum Concentration (Tmax) will be assessed [ Time Frame: Day 12-15 and, 3 months to 12 months ]
    PK analyses will be performed on the concentration measurement of fenfluramine and its metabolite (norfenfluramine) and the effect on the concentration of CBD by measuring the Tmax



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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, IPCAB, IDSMC, 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     History of Changes
Other Study ID Numbers: ZX008-1602
First Posted: March 15, 2018    Key Record Dates
Last Update Posted: August 14, 2018
Last Verified: March 2018

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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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Syndrome
Lennox Gastaut Syndrome
Epilepsies, Myoclonic
Spasms, Infantile
Rett Syndrome
Disease
Pathologic Processes
Epileptic Syndromes
Epilepsy
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Epilepsy, Generalized
Mental Retardation, X-Linked
Intellectual Disability
Neurobehavioral Manifestations
Neurologic Manifestations
Genetic Diseases, X-Linked
Heredodegenerative Disorders, Nervous System
Fenfluramine
Serotonin Uptake Inhibitors
Neurotransmitter Uptake Inhibitors
Membrane Transport Modulators
Molecular Mechanisms of Pharmacological Action
Neurotransmitter Agents
Serotonin Agents
Physiological Effects of Drugs