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
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 412
- 试验地点
- 71
- 主要终点
- Percentage of Participants With Treatment-emergent Adverse Events (TEAEs)
研究概览
简要总结
This is an international, multicenter, open-label, long-term safety study of ZX008 in subjects with Dravet syndrome, Lennox-Gastaut syndrome or epileptic encephalopathy
详细描述
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.
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
盲法说明
None (open label)
入排标准
- 年龄范围
- 2 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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
排除标准
- •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
研究组 & 干预措施
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.
干预措施: ZX008 (Fenfluramine Hydrochloride) (Drug)
结局指标
主要结局
Percentage of Participants With Treatment-emergent Adverse Events (TEAEs)
时间窗: From First dose of ZX008 (in study EP0215 [ZX008-1900]) to Cardiac Follow-up after ZX008 treatment (Up to 6 Years, 17 Days)
An adverse event (AE) is any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product and which does not necessarily have a causal relationship with this treatment. An AE can, therefore, be any unfavorable and unintended sign (including an abnormal, clinically significant laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not considered related to the medicinal (investigational) product. TEAEs were defined as AE that began on or after the first day of treatment with ZX008 in Study EP0215 (ZX008-1900) (NCT03936777) or that occurred prior to first ZX008 treatment in Study EP0215 (ZX008-1900) but increased in severity after treatment in Study EP0215 (ZX008-1900) began. Events recorded at Follow-up/Cardiac Follow-up were considered treatment-emergent. The percentage of participants was rounded to one decimal place.
次要结局
- Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Parent/Caregiver(Months 6, 12, 18, 24, 30, 36, Last On-Treatment Visit (Up to Month 49))
- Clinical Global Impression-Improvement in Participants, Subscale Global: Assessment by Investigator(Months 6, 12, 18, 24, 30, 36, Last On-Treatment Visit (Up to Month 49))
- Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Parent/Caregiver(Months 6, 12, 18, 24, 30, 36, Last On-Treatment Visit (Up to Month 49))
- Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Parent/Caregiver(Months 6, 12, 18, 24, 30, 36, Last On-Treatment Visit (Up to Month 49))
- Number of Participants With Improvement in Seizure Burden as Assessed by the Investigator(Baseline (Day 1), Months 6, 12, 18, 24, 30, 36, Last On-Treatment Visit (Up to Month 49))
- Clinical Global Impression-Improvement in Participants, Subscale Motor Abilities: Assessment by Parent/Caregiver(Months 6, 12, 18, 24, 30, 36, Last On-Treatment Visit (Up to Month 49))
- Clinical Global Impression-Improvement in Participants, Subscale Cognition: Assessment by Investigator(Months 6, 12, 18, 24, 30, 36, Last On-Treatment Visit (Up to Month 49))
- Clinical Global Impression-Improvement in Participants, Subscale Behavior: Assessment by Investigator(Months 6, 12, 18, 24, 30, 36, Last On-Treatment Visit (Up to Month 49))
- Clinical Global Impression Improvement in Participants, Subscale Motor Abilities: Assessment by Investigator(Months 6, 12, 18, 24, 30, 36, Last On-Treatment Visit (Up to Month 49))