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

March 21, 2024 updated by: Zogenix, Inc.

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

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

Study Overview

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.

Study Type

Interventional

Enrollment (Actual)

412

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

      • Heidelberg, Australia
        • Ep0215 301
      • South Brisbane, Australia
        • Ep0215 302
      • Westmead, Australia
        • Ep0215 303
      • Bruxelles, Belgium
        • Ep0215 803
      • Edegem, Belgium
        • Ep0215 801
      • Jette, Belgium
        • Ep0215 802
      • Montreal, Canada
        • Ep0215 202
      • Toronto, Canada
        • Ep0215 204
      • Vancouver, Canada
        • Ep0215 201
      • Dianalund, Denmark
        • Ep0215 701
      • Bordeaux Cedex, France
        • Ep0215 1004
      • Lille, France
        • Ep0215 1005
      • Marseille Cedex 05, France
        • Ep0215 1007
      • Paris, France
        • Ep0215 1001
      • Paris, France
        • Ep0215 1002
      • Salouel, France
        • Ep0215 1008
      • Bielefeld, Germany
        • Ep0215 902
      • Freiburg Im Breisgau, Germany
        • Ep0215 906
      • Jena, Germany
        • Ep0215 905
      • Kiel, Germany
        • Ep0215 908
      • Radeberg, Germany
        • Ep0215 903
      • Vogtareuth, Germany
        • Ep0215 901
      • Firenze, Italy
        • Ep0215 1201
      • Genova, Italy
        • Ep0215 1204
      • Mantova, Italy
        • Ep0215 1205
      • Milano, Italy
        • Ep0215 1207
      • Roma, Italy
        • Ep0215 1206
      • Roma, Italy
        • Ep0215 1208
      • Verona, Italy
        • Ep0215 1202
      • Guadalajara, Mexico
        • Ep0215 1604
      • Heeze, Netherlands
        • Ep0215 1402
      • Zwolle, Netherlands
        • Ep0215 1401
      • Bydgoszcz, Poland
        • Ep0215 1702
      • Krakow, Poland
        • Ep0215 1701
      • Barcelona, Spain
        • Ep0215 1105
      • Barcelona, Spain
        • Ep0215 1107
      • Esplugues de Llobregat, Spain
        • Ep0215 1103
      • Madrid, Spain
        • Ep0215 1101
      • Pamplona, Spain
        • Ep0215 1102
      • Göteborg, Sweden
        • Ep0215 502
      • Birmingham, United Kingdom
        • Ep0215 605
      • Glasgow, United Kingdom
        • Ep0215 601
      • Liverpool, United Kingdom
        • Ep0215 603
      • London, United Kingdom
        • Ep0215 602
      • London, United Kingdom
        • Ep0215 606
    • Arizona
      • Tucson, Arizona, United States, 85718
        • Ep0215 107
    • California
      • Los Angeles, California, United States, 90095
        • Ep0215 144
      • San Diego, California, United States, 92123
        • Ep0215 108
      • San Francisco, California, United States, 94158
        • Ep0215 101
    • Colorado
      • Aurora, Colorado, United States, 80045
        • Ep0215 103
    • Florida
      • Gulf Breeze, Florida, United States, 32561
        • Ep0215 115
      • Miami, Florida, United States, 33155
        • Ep0215 104
      • Orlando, Florida, United States, 32803
        • Ep0215 141
      • Winter Park, Florida, United States, 32789
        • Ep0215 121
    • Georgia
      • Atlanta, Georgia, United States, 30328
        • Ep0215 117
    • Illinois
      • Chicago, Illinois, United States, 60611
        • Ep0215 110
    • Maryland
      • Bethesda, Maryland, United States, 20817
        • Ep0215 140
    • Massachusetts
      • Boston, Massachusetts, United States, 02114
        • Ep0215 112
    • Minnesota
      • Rochester, Minnesota, United States, 55905
        • Ep0215 109
      • Roseville, Minnesota, United States, 55113
        • Ep0215 132
    • New Jersey
      • Hackensack, New Jersey, United States, 07601
        • Ep0215 105
      • Livingston, New Jersey, United States, 07039
        • Ep0215 118
    • New York
      • Hawthorne, New York, United States, 10532
        • Ep0215 150
      • New York, New York, United States, 10016
        • Ep0215 142
    • Ohio
      • Cleveland, Ohio, United States, 44106
        • Ep0215 131
    • Tennessee
      • Memphis, Tennessee, United States, 38103
        • Ep0215 124
    • Texas
      • Dallas, Texas, United States, 75207
        • Ep0215 146
      • Fort Worth, Texas, United States, 76104
        • Ep0215 126
    • Utah
      • Salt Lake City, Utah, United States, 84108
        • Ep0215 106
    • Washington
      • Seattle, Washington, United States, 98105
        • Ep0215 119
      • Tacoma, Washington, United States, 98405
        • Ep0215 125

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

2 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / 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.
Fenfluramine hydrochloride provided in a concentration of 2.5 mg/mL.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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
Up to 36 months open-label
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
Up to 36 months open-label
Changes in heart rate
Time Frame: Up to 36 months open-label
The analysis will include change in heart rate using standard measure
Up to 36 months open-label
Changes in respiratory rate
Time Frame: Up to 36 months open-label
The analysis will include change in resting respiratory rate using standard measure
Up to 36 months open-label
Changes in blood pressure
Time Frame: Up to 36 months open-label
The analysis will include change in resting blood pressure using standard measure
Up to 36 months open-label
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
Up to 36 months open-label
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
Up to 36 months open-label
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
Up to 36 months open-label

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change in convulsive seizure response
Time Frame: Up to 36 months open-label
The analysis will include percent improvement per investigator rating
Up to 36 months open-label
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".
Up to 36 months open-label
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".
Up to 36 months open-label
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".
Up to 36 months open-label
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".
Up to 36 months open-label
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".
Up to 36 months open-label
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".
Up to 36 months open-label
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"
Up to 36 months open-label
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"
Up to 36 months open-label

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Investigators

  • Study Director: UCB Cares, 001 844 599 2273

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

April 22, 2019

Primary Completion (Estimated)

June 28, 2024

Study Completion (Estimated)

June 28, 2024

Study Registration Dates

First Submitted

April 12, 2019

First Submitted That Met QC Criteria

April 30, 2019

First Posted (Actual)

May 3, 2019

Study Record Updates

Last Update Posted (Actual)

March 22, 2024

Last Update Submitted That Met QC Criteria

March 21, 2024

Last Verified

March 1, 2024

More Information

Terms related to this study

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

Data from this trial may be requested by qualified researchers six months after product approval in the US and/or Europe, or global development is discontinued, and 18 months after trial completion. Investigators may request access to anonymized individual patient-level data and redacted trial documents which may include: analysis-ready datasets, study protocol, annotated case report form, statistical analysis plan, dataset specifications, and clinical study report. Prior to use of the data, proposals need to be approved by an independent review panel at www.Vivli.org and a signed data sharing agreement will need to be executed. All documents are available in English only, for a pre-specified time, typically 12 months, on a password protected portal. This plan may change if the risk of re-identifying trial participants is determined to be too high after the trial is completed; in this case and to protect participants, individual patient-level data would not be made available.

IPD Sharing Time Frame

Data from this trial may be requested by qualified researchers six months after product approval in the US and/or Europe or global development is discontinued, and 18 months after trial completion.

IPD Sharing Access Criteria

Qualified researchers may request access to anonymized IPD and redacted study documents which may include: raw datasets, analysis-ready datasets, study protocol, blank case report form, annotated case report form, statistical analysis plan, dataset specifications, and clinical study report. Prior to use of the data, proposals need to be approved by an independent review panel at www.Vivli.org and a signed data sharing agreement will need to be executed. All documents are available in English only, for a pre-specified time, typically 12 months, on a password protected portal.

IPD Sharing Supporting Information Type

  • STUDY_PROTOCOL
  • SAP
  • CSR

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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