Back to resultsA Study to Assess the Safety and Tolerability of ZX008 in Children and Young Adults With Dravet Syndrome or Lennox Gastaut Syndrome Currently Taking Cannabidiol
Primary Purpose
Dravet Syndrome, Lennox Gastaut Syndrome
Status
Completed
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
ZX008 0.2 to 0.8 mg/kg/day
Cannabidiol
Sponsored by
About this trial
This is an interventional treatment trial for Dravet Syndrome focused on measuring Dravet Syndrome, LGS
Eligibility 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.
Sites / Locations
- PANDA Neurology/CIRCA
- MultiCare Institute for Research & Innovation
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
ZX008 0.2 to 0.8 mg/kg/day
Arm Description
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)
Outcomes
Primary Outcome Measures
Adverse Events
Number of adverse events including serious adverse events, number of adverse events leading to study withdrawal
Change in Heart Rate
Change in resting heart rate using standard measures
Change in blood pressure
Change in resting blood pressure using standard measures
Change in temperature
Change in resting temperature using standard measures
Change in respiratory rate
Change in resting respiratory rate using standard measures
Changes in heart rhythm
Changes in heart beat as measured with 12-lead electrocardiogram
Changes in heart valve function
Changes in heart valves as measured with standard echocardiogram
Changes in treatment-emergent body weight and height
Changes in body weight and height by report of BMI in kg/m^2
Secondary Outcome Measures
Full Information
NCT ID
NCT03467113
First Posted
January 30, 2018
Last Updated
October 31, 2022
Sponsor
Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc.
1. Study Identification
Unique Protocol Identification Number
NCT03467113
Brief Title
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
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
Study Type
Interventional
2. Study Status
Record Verification Date
October 2022
Overall Recruitment Status
Completed
Study Start Date
January 19, 2018 (Actual)
Primary Completion Date
June 24, 2020 (Actual)
Study Completion Date
June 24, 2020 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc.
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
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).
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Dravet Syndrome, Lennox Gastaut Syndrome
Keywords
Dravet Syndrome, LGS
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
9 (Actual)
8. Arms, Groups, and Interventions
Arm Title
ZX008 0.2 to 0.8 mg/kg/day
Arm Type
Experimental
Arm Description
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)
Intervention Type
Drug
Intervention Name(s)
ZX008 0.2 to 0.8 mg/kg/day
Intervention Description
ZX008 drug is an oral aqueous solution of fenfluramine hydrochloride. The product is sugar free.
Intervention Type
Drug
Intervention Name(s)
Cannabidiol
Intervention Description
Cannabidiol
Primary Outcome Measure Information:
Title
Adverse Events
Description
Number of adverse events including serious adverse events, number of adverse events leading to study withdrawal
Time Frame
Baseline to Day 365
Title
Change in Heart Rate
Description
Change in resting heart rate using standard measures
Time Frame
Baseline to Day 365
Title
Change in blood pressure
Description
Change in resting blood pressure using standard measures
Time Frame
Baseline to Day 365
Title
Change in temperature
Description
Change in resting temperature using standard measures
Time Frame
Baseline to Day 365
Title
Change in respiratory rate
Description
Change in resting respiratory rate using standard measures
Time Frame
Baseline to Day 365
Title
Changes in heart rhythm
Description
Changes in heart beat as measured with 12-lead electrocardiogram
Time Frame
Baseline to Day 365
Title
Changes in heart valve function
Description
Changes in heart valves as measured with standard echocardiogram
Time Frame
Baseline to Day 365
Title
Changes in treatment-emergent body weight and height
Description
Changes in body weight and height by report of BMI in kg/m^2
Time Frame
Baseline to Day 365
10. Eligibility
Sex
All
Minimum Age & Unit of Time
2 Years
Maximum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility 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.
Facility Information:
Facility Name
PANDA Neurology/CIRCA
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30328
Country
United States
Facility Name
MultiCare Institute for Research & Innovation
City
Tacoma
State/Province
Washington
ZIP/Postal Code
98405
Country
United States
12. IPD Sharing Statement
Learn more about this trial
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
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