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A Study to Investigate the Long-Term Safety of ZX008 (Fenfluramine Hydrochloride) Oral Solution in Children and Adults With Epileptic Encephalopathy Including Dravet Syndrome and Lennox-Gastaut Syndrome

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ClinicalTrials.gov Identifier: NCT03936777
Recruitment Status : Enrolling by invitation
First Posted : May 3, 2019
Last Update Posted : June 12, 2019
Sponsor:
Collaborator:
Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc.
Information provided by (Responsible Party):
Zogenix, Inc.

Brief Summary:
This is an international, multicenter, open-label, long-term safety study of ZX008 in subjects with Dravet syndrome, Lennox-Gastaut syndrome or epileptic encephalopathy

Condition or disease Intervention/treatment Phase
Dravet Syndrome Lennox Gastaut Syndrome Epileptic Encephalopathy Drug: ZX008 (Fenfluramine Hydrochloride) Phase 3

Detailed Description:
This is an international, multicenter, open-label, long-term safety study of ZX008 in patients with epileptic encephalopathy, including Dravet syndrome or Lennox-Gastaut syndrome. Subjects eligible for participation are those with Dravet syndrome who are currently enrolled in Study ZX008-1503, or those with Lennox-Gastaut syndrome who have successfully completed Study ZX008-1601-Part 2, and are candidates for continued treatment with ZX008 for an extended period of time, or those with Dravet syndrome, Lennox-Gastaut syndrome, or another epileptic encephalopathy who have completed participation in another Zogenix-sponsored study and have been invited to participate in this study.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 650 participants
Intervention Model: Single Group Assignment
Intervention Model Description: Single Group Assisgnment
Masking: None (Open Label)
Masking Description: None (open label)
Primary Purpose: Treatment
Official Title: An Open-Label Extension Trial to Assess the Long-Term Safety of ZX008 (Fenfluramine Hydrochloride) Oral Solution as an Adjunctive Therapy for Seizures in Patients With Rare Seizure Disorders Such as Epileptic Encephalopathies Including Dravet Syndrome and Lennox-Gastaut Syndrome
Actual Study Start Date : April 22, 2019
Estimated Primary Completion Date : April 2023
Estimated Study Completion Date : April 2023


Arm Intervention/treatment
Experimental: ZX008 (Fenfluramine Hydrochloride)
ZX008 is supplied as an open-label oral solution.Doses will include up to 0.8 mg/kg/day divided into 2 daily doses, up to a maximum of 30 mg/day (subjects taking concomitant STP will receive up to 0.5 mg/kg/day, up to a maximum of 20 mg/day) in a concentration of 2.5 mg/mL.
Drug: ZX008 (Fenfluramine Hydrochloride)
Fenfluramine hydrochloride provided in a concentration of 2.5 mg/mL.




Primary Outcome Measures :
  1. Change in Adverse Events and Serious Adverse Events leading to study withdrawal in subjects receiving open label ZX008 [ Time Frame: Up to 36 months open-label ]
    Evaluate the long term safety and tolerability of oral dose administration of ZX008

  2. Changes in laboratory test results [ Time Frame: Up to 36 months open-label ]
    The analysis will include abnormal hematology and chemistry laboratory results outside of reference ranges

  3. Changes in heart rate [ Time Frame: Up to 36 months open-label ]
    The analysis will include change in heart rate using standard measure

  4. Changes in respiratory rate [ Time Frame: Up to 36 months open-label ]
    The analysis will include change in resting respiratory rate using standard measure

  5. Changes in blood pressure [ Time Frame: Up to 36 months open-label ]
    The analysis will include change in resting blood pressure using standard measure

  6. Change in body weight [ Time Frame: Up to 36 months open-label ]
    The analysis will include change in body weight and height by report of BMI in kg/m^2

  7. Changes in heart rhythm [ Time Frame: Up to 36 months open-label ]
    The analysis will include changes in heart beat as measured with 12-lead electrocardiogram

  8. Changes in heart valve function [ Time Frame: Up to 36 months open-label ]
    The analysis will include change in heart valves as measured with standard echocardiogram


Secondary Outcome Measures :
  1. Change in convulsive seizure response [ Time Frame: Up to 36 months open-label ]
    The analysis will include percent improvement per investigator rating

  2. Change in Cognitive CGI by Parent/Caregiver [ Time Frame: Up to 36 months open-label ]
    The rating by parent/caregiver of the patient's change in cognitive symptoms since the beginning of study as "very much improved", "much improved", "minimally improved", "no change", "minimally worse", "much worse", or "very much worse".

  3. Change in Behavioral CGI by Parent/Caregiver [ Time Frame: Up to 36 months open-label ]
    The rating by parent/caregiver of the patient's change in behavioral symptoms since the beginning of study as "very much improved", "much improved", "minimally improved", "no change", "minimally worse", "much worse", or "very much worse".

  4. Change in Motor CGI by Parent/Caregiver [ Time Frame: Up to 36 months open-label ]
    The rating by parent/caregiver of the patient's change in motor symptoms since the beginning of study as "very much improved", "much improved", "minimally improved", "no change", "minimally worse", "much worse", or "very much worse".

  5. Change in Cognitive CGI by Investigator [ Time Frame: Up to 36 months open-label ]
    The rating by investigator of the patient's change in cognitive symptoms since the beginning of study as "very much improved", "much improved", "minimally improved", "no change", "minimally worse", "much worse", or "very much worse".

  6. Change in Behavioral CGI by Investigator [ Time Frame: Up to 36 months open-label ]
    The rating by investigator of the patient's change in behavioral symptoms since the beginning of study as "very much improved", "much improved", "minimally improved", "no change", "minimally worse", "much worse", or "very much worse".

  7. Change in Motor CGI by Investigator [ Time Frame: Up to 36 months open-label ]
    The rating by investigator of the patient's change in motor symptoms since the beginning of study as "very much improved", "much improved", "minimally improved", "no change", "minimally worse", "much worse", or "very much worse".

  8. Change in Clinical Global Impression by Parent/Caregiver [ Time Frame: Up to 36 months open-label ]
    The rating by parent/caregiver of the patient's overall change in symptoms since the beginning of study as "very much improved", "much improved", "minimally improved", "no change", "minimally worse", "much worse", or "very much worse"

  9. Change in Clinical Global Impression by Investigator [ Time Frame: Up to 36 months open-label ]
    The rating by investigator of the patient's overall change in symptoms since the beginning of study as "very much improved", "much improved", "minimally improved", "no change", "minimally worse", "much worse", or "very much worse"



Information from the National Library of Medicine

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Ages Eligible for Study:   2 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Male or nonpregnant, nonlactating female
  • Satisfactory completion of a core study
  • Has a rare seizure disorder, such as epileptic encephalopathy and has successfully completed another Zogenix-sponsored clinical trials with ZX008
  • Subject's caregiver is willing and able to be compliant with study procedures, visit schedule and study drug accountability

Exclusion Criteria:

  • Current cardiac valvulopathy or pulmonary hypertension that is clinically significant
  • Moderate or severe hepatic impairment
  • Receiving monoamine oxidase inhibitors, serotonin agonists, serotonin antagonists, and serotonin reuptake inhibitors within 14 days of receiving ZX008

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): NCT03936777


Locations
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United States, Arizona
Center for Neurosciences
Tucson, Arizona, United States, 85718-6522
United States, Florida
Pediatric Neurology, PA
Orlando, Florida, United States, 32819
United States, Washington
MultiCare Institute for Research & Innovation
Tacoma, Washington, United States, 98405
Sponsors and Collaborators
Zogenix, Inc.
Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc.

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Responsible Party: Zogenix, Inc.
ClinicalTrials.gov Identifier: NCT03936777     History of Changes
Other Study ID Numbers: ZX008-1900
First Posted: May 3, 2019    Key Record Dates
Last Update Posted: June 12, 2019
Last Verified: June 2019

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Brain Diseases
Epilepsy
Lennox Gastaut Syndrome
Epilepsies, Myoclonic
Spasms, Infantile
Syndrome
Disease
Pathologic Processes
Central Nervous System Diseases
Nervous System Diseases
Epileptic Syndromes
Genetic Diseases, Inborn
Epilepsy, Generalized
Fenfluramine
Serotonin Uptake Inhibitors
Neurotransmitter Uptake Inhibitors
Membrane Transport Modulators
Molecular Mechanisms of Pharmacological Action
Neurotransmitter Agents
Serotonin Agents
Physiological Effects of Drugs