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
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 发起方
- 入组人数
- 9
- 试验地点
- 2
- 主要终点
- Change in Heart Rate
研究概览
简要总结
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).
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 2 Years 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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.
排除标准
- •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.
研究组 & 干预措施
ZX008 0.2 to 0.8 mg/kg/day
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)
干预措施: ZX008 0.2 to 0.8 mg/kg/day (Drug)
ZX008 0.2 to 0.8 mg/kg/day
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)
干预措施: Cannabidiol (Drug)
结局指标
主要结局
Change in Heart Rate
时间窗: Baseline to Day 365
Change in resting heart rate using standard measures
Adverse Events
时间窗: Baseline to Day 365
Number of adverse events including serious adverse events, number of adverse events leading to study withdrawal
Changes in heart rhythm
时间窗: Baseline to Day 365
Changes in heart beat as measured with 12-lead electrocardiogram
Changes in heart valve function
时间窗: Baseline to Day 365
Changes in heart valves as measured with standard echocardiogram
Changes in treatment-emergent body weight and height
时间窗: Baseline to Day 365
Changes in body weight and height by report of BMI in kg/m\^2
Change in blood pressure
时间窗: Baseline to Day 365
Change in resting blood pressure using standard measures
Change in respiratory rate
时间窗: Baseline to Day 365
Change in resting respiratory rate using standard measures
Change in temperature
时间窗: Baseline to Day 365
Change in resting temperature using standard measures
次要结局
未报告次要终点