A Phase 2 Study to Assess the Efficacy and Safety of Cannabidiol Oral Solution for the Treatment of Refractory Infantile Spasms
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 入组人数
- 9
- 试验地点
- 4
- 主要终点
- Part A: Percentage of Participants Who Are Considered Complete Responders at Day 14
研究概览
简要总结
Infantile Spasms (IS) is a diagnosis described as a fairly rare and terrible form of epilepsy that usually strikes children in the first year of life. There is a great need for safe and effective therapies in the treatment of IS. This need is even more important for infants and toddlers still sick after being treated with medicine that is already available.
This is a multi-center study to evaluate the efficacy and safety of Cannabidiol Oral Solution (CBD) in the treatment of children aged 6 months through 36 months with a diagnosis of infantile spasms who have not responded to first line therapies.
The overall study duration is expected to be 64 weeks for those subjects who respond to CBD treatment. The maximum possible study duration for each patient is approximately 64 weeks, however a subject will be deemed to have completed the study after 58 weeks.
详细描述
A protocol amendment in May 2016 created two parts to this trial: Part A (the extended treatment period) and Part B (the safety treatment period), whose objectives are as follows:
Primary Part A: To evaluate the efficacy of Cannabidiol Oral Solution in treating refractory infantile spasms (IS).
Secondary:
Part A:
- To evaluate the safety of Cannabidiol Oral Solution in treating refractory infantile spasms.
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
- 6 Months 至 36 Months（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Meets protocol-specified criteria for qualification, including infantile spasms
- •Parent(s)/caregiver(s) fully comprehend and sign the informed consent form, understand all study procedures, and can communicate satisfactorily with the Investigator and study coordinator.
排除标准
- •History or current use of over-the-counter medications, dietary supplements, or drugs outside protocol-specified parameters
- •Signs, symptoms or history of any condition that, per protocol or in the opinion of the investigator, might compromise:
- •the safety or well-being of the participant or study staff
- •the analysis of results
- •During the Safety Treatment and Follow-up Periods, subjects are not to receive the following:
- •any cannabinoids (CBD, Δ9-tetrahydrocannabinol (THC), hemp oil, Realm Oil or marijuana)
- •any other investigational drug or investigational device
研究组 & 干预措施
Cannabidiol Oral Solution: 20 or 40 mg/kg/day BID
The dose of Cannabidiol Oral Solution will begin at 20 mg/kg/day [10 mg/kg twice per day (BID)], will be adjusted at any time if the investigator feels the safety or well-being of the participant is at risk, and will be titrated up or down according to protocol-stipulated parameters and at the investigator's discretion after Day 14 to enhance efficacy. Dose will not exceed 40 mg/kg/day.
干预措施: Cannabidiol Oral Solution (Drug)
结局指标
主要结局
Part A: Percentage of Participants Who Are Considered Complete Responders at Day 14
时间窗: Day 14
Complete response was defined as complete resolution of spasms and hypsarrythmia (if present at baseline) confirmed by video-electroencephalogram (EEG) at Day 14.
Part B: Percentage of Participants Experiencing Adverse Events (AEs), Treatment-Emergent AEs (TEAEs), and Serious Adverse Events (SAEs)
时间窗: Up to Week 64
次要结局
- Part A: Percentage of Participants With Absence of Infantile Spasms at Day 14(Day 14)
- Part A: Percentage of Participants With a Partial Response to Treatment(Day 14)
- Part A: Time to Complete Responder Relapse(Day 14)
- Part A: Percentage of Participants With Absence of Hypsarrhythmia at Day 14(Day 14)
- Part A: Median Reduction in Seizure-burden Comparing Video-EEG at Baseline to Repeat Video-EEG at Day 14(Baseline, Day 14)
- Part A: Parent Impression of Efficacy and Tolerability of Study Drug(Visit 3 (Day 14), Visit 4 (Week 4), Visit 5 (Week 8), Visit 6 (Week 10), and end of study.)
- Part A: Percentage of Complete Responders With Relapse(Day 14)
- Part B: Parent Impression of Efficacy and Tolerability of Study Drug as Measured by the Change in Clinical Global Impression of Improvement Assessment (CGI-I), Responses at Every Visit Throughout Part B(Up to Week 64)
- Part B: Investigator Impression of Efficacy and Tolerability of Study Drug as Measured by the Change in CGI-I Responses at Every Visit Throughout Part B(Up to Week 64)
- Part B: Median Reduction in Seizure-burden Comparing Seizure Diaries Throughout Part B.(Up to Week 64)
- Part B: Percentage of Participants Who Have a Relapse of Spasms Based on Video-EEG(Up to Week 64)
- Part B: Time to Relapse as Confirmed by Video-EEG(Up to Week 64)