A 2-part Study to Investigate the Dose-ranging Pharmacokinetics and Tolerability, Followed by the Efficacy and Safety of Cannabidiol (GWP42003-P) in Children and Young Adults With
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 26
- 试验地点
- 1
- 主要终点
- Seizure Frequency
研究概览
简要总结
Part A: To evaluate the safety and tolerability of multiple ascending doses of GWP42003-P compared with placebo with respect to:
- Incidence, type and severity of adverse events (AEs)
- Effect on vital signs, including weight
- Effect on 12-lead electrocardiogram (ECG) findings
- Effect on laboratory parameters Part B: To make an assessment of the anti-epileptic efficacy of GWP42003-P compared with placebo with respect to the incidence in convulsive seizures
- To determine the plasma concentration time curves for GWP42003-P and its major human metabolite, following escalating multiple doses of GWP42003-P.
- To investigate the effect of GWP42003-P on the pharmacokinetics of concomitant anti-epileptic drugs (AEDs).
- To evaluate cognitive function, sleep quality and daytime sleepiness, in patients taking GWP42003-P in combination with AEDs.
详细描述
This multi-center study will consist of two parts: Part A and Part B. Part A will investigate the dose-ranging pharmacokinetics and tolerability of GWP42003-P.
Part B is a 1:1 randomized, placebo-controlled 60 day comparison of GWP42003-P against placebo, at the dose which has been identified by Part A as the maximum tolerated dose. Part B will investigate the efficacy and safety of GWP42003-P.
Following completion of Part B, all patients will be invited to continue to receive GWP42003-P in an open label extension study (which is separate to this protocol).
Part A Following establishment of initial eligibility and baseline measurements, patients will enter Part A at Visit 1 and will begin the two month baseline observation period. Patients will record their number of convulsive seizures, and of other seizures, using an IVRS telephone diary system (recorded twice each day). Each call will take approximately five minutes to complete. Patients will return to the clinic at Visit 2 and the investigator will assess the patient's daily number of convulsive seizures from the patient's IVRS data. Patients who have experienced at least four convulsive seizures (tonic-clonic seizures and/or drop attacks) per month for each of the previous two months) and who meet all of the other inclusion/ exclusion criteria will be eligible to continue in the study. Patients will be asked for information regarding AEs, concomitant medications and/or changes to their medication.
Eligible patients will enter into the pharmacokinetics part of the study at Visit 2 where blood samples will be taken using a sparse sampling strategy to minimize the blood volumes required for analysis of the plasma concentration/time curve for concomitant AEDs and routine safety bloods. This is conducted prior to the administration of Study medication at Dose Level 1.
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
open label
入排标准
- 年龄范围
- 2 Years 至 25 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients meeting the following criteria will be considered eligible for this study:
- •Documentation of a diagnosis of drug resistant epilepsy as evidenced by failure to control siezures despite appropriate trial of four or more Anti-Epileptic Drugs at therapeutic doses. Documentation must include the diagnosis of epilepsy type or epilepsy syndrome (if possible), as well as the underlying case, when known.
- •Between 1-3 baseline anti-epileptic drugs at stable doses for a minimum of 4 weeks prior to enrollment. Vagus nerve stimulator, ketogenic diet and modified Atkins diet do not count toward this limit.
- •Vagus nerve stimulator must be on stable settings for a minimum of 3 months.
- •Written informed consent obtained from the patient or the patient's legal representative must be obtained prior to beginning treatment.
排除标准
- •Exclusion: The patient may not enter the study if ANY of the following apply:
- •Treatment with any artisanal preparation containing or possible containing CBD during the month before initiation of the study drug.
研究组 & 干预措施
Cannabidiol
open label administration
干预措施: Cannabidiol (Drug)
结局指标
主要结局
Seizure Frequency
时间窗: Baseline to 1 year
Number of Seizures
次要结局
- Drug Plasma Levels of Cannabidiol(Baseline, 8 weeks, 6 months)
研究者
Francis Filloux
Francis M. Filloux, M.D. Division Chief, Pediatric Neurology University of Utah School of Medicine
University of Utah