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出境医 / 临床实验 / 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
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
Study Description
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.
Study Design
| Study Type : | Interventional (Clinical Trial) |
| Estimated Enrollment : | 650 participants |
| Allocation: | N/A |
| 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 |
Arms and Interventions
| 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.
|
Outcome Measures
Primary Outcome Measures :
- 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
- 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
- Changes in heart rate [ Time Frame: Up to 36 months open-label ]The analysis will include change in heart rate using standard measure
- Changes in respiratory rate [ Time Frame: Up to 36 months open-label ]The analysis will include change in resting respiratory rate using standard measure
- Changes in blood pressure [ Time Frame: Up to 36 months open-label ]The analysis will include change in resting blood pressure using standard measure
- 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
- 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
- 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 :
- Change in convulsive seizure response [ Time Frame: Up to 36 months open-label ]The analysis will include percent improvement per investigator rating
- 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".
- 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".
- 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".
- 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".
- 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".
- 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".
- 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"
- 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"
Eligibility Criteria
| 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
Contacts and Locations
Locations
Show 28 study locations
Sponsors and Collaborators
Zogenix, Inc.
Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc.
| Tracking Information | |||||
|---|---|---|---|---|---|
| First Submitted Date ICMJE | April 12, 2019 | ||||
| First Posted Date ICMJE | May 3, 2019 | ||||
| Last Update Posted Date | September 17, 2020 | ||||
| Actual Study Start Date ICMJE | April 22, 2019 | ||||
| Estimated Primary Completion Date | April 2023 (Final data collection date for primary outcome measure) | ||||
| Current Primary Outcome Measures ICMJE |
|
||||
| Original Primary Outcome Measures ICMJE | Same as current | ||||
| Change History | |||||
| Current Secondary Outcome Measures ICMJE |
|
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| Original Secondary Outcome Measures ICMJE | Same as current | ||||
| Current Other Pre-specified Outcome Measures | Not Provided | ||||
| Original Other Pre-specified Outcome Measures | Not Provided | ||||
| Descriptive Information | |||||
| Brief Title ICMJE | 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 | ||||
| Official Title ICMJE | 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 | ||||
| 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 | ||||
| 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 ICMJE | Interventional | ||||
| Study Phase ICMJE | Phase 3 | ||||
| Study Design ICMJE | Allocation: N/A Intervention Model: Single Group Assignment Intervention Model Description: Single Group Assisgnment Masking: None (Open Label)Masking Description: None (open label) Primary Purpose: Treatment
|
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| Condition ICMJE |
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| Intervention ICMJE | Drug: ZX008 (Fenfluramine Hydrochloride)
Fenfluramine hydrochloride provided in a concentration of 2.5 mg/mL.
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| Study Arms ICMJE | 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.
Intervention: Drug: ZX008 (Fenfluramine Hydrochloride)
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| Publications * | Not Provided | ||||
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* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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| Recruitment Information | |||||
| Recruitment Status ICMJE | Enrolling by invitation | ||||
| Estimated Enrollment ICMJE |
650 | ||||
| Original Estimated Enrollment ICMJE | Same as current | ||||
| Estimated Study Completion Date ICMJE | April 2023 | ||||
| Estimated Primary Completion Date | April 2023 (Final data collection date for primary outcome measure) | ||||
| Eligibility Criteria ICMJE |
Inclusion Criteria:
Exclusion Criteria:
|
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| Sex/Gender ICMJE | Not Provided | ||||
| Ages ICMJE | 2 Years and older (Child, Adult, Older Adult) | ||||
| Accepts Healthy Volunteers ICMJE | No | ||||
| Contacts ICMJE | Contact information is only displayed when the study is recruiting subjects | ||||
| Listed Location Countries ICMJE | Australia, Belgium, Canada, Denmark, Spain, United Kingdom, United States | ||||
| Removed Location Countries | |||||
| Administrative Information | |||||
| NCT Number ICMJE | NCT03936777 | ||||
| Other Study ID Numbers ICMJE | ZX008-1900 | ||||
| Has Data Monitoring Committee | Yes | ||||
| U.S. FDA-regulated Product |
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| IPD Sharing Statement ICMJE | Not Provided | ||||
| Responsible Party | Zogenix, Inc. | ||||
| Study Sponsor ICMJE | Zogenix, Inc. | ||||
| Collaborators ICMJE | Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc. | ||||
| Investigators ICMJE | Not Provided | ||||
| PRS Account | Zogenix, Inc. | ||||
| Verification Date | September 2020 | ||||
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ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |
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