Dravet Syndrome, Lennox Gastaut Syndrome
Conditions
Keywords
Dravet Syndrome, LGS
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).
Interventions
DRUGZX008 0.2 to 0.8 mg/kg/day
ZX008 drug is an oral aqueous solution of fenfluramine hydrochloride. The product is sugar free.
DRUGCannabidiol
Cannabidiol
Sponsors
Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
2 Years to 18 Years
Healthy volunteers
No
Inclusion 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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Adverse Events | Baseline to Day 365 | Number of adverse events including serious adverse events, number of adverse events leading to study withdrawal |
| Change in Heart Rate | Baseline to Day 365 | Change in resting heart rate using standard measures |
| Change in blood pressure | Baseline to Day 365 | Change in resting blood pressure using standard measures |
| Change in temperature | Baseline to Day 365 | Change in resting temperature using standard measures |
| Change in respiratory rate | Baseline to Day 365 | Change in resting respiratory rate using standard measures |
| Changes in heart rhythm | Baseline to Day 365 | Changes in heart beat as measured with 12-lead electrocardiogram |
| Changes in heart valve function | Baseline to Day 365 | Changes in heart valves as measured with standard echocardiogram |
| Changes in treatment-emergent body weight and height | Baseline to Day 365 | Changes in body weight and height by report of BMI in kg/m\^2 |
Countries
United States
Outcome results
None listed