A Triple-Blind, Placebo-Controlled, Randomized Clinical Trial of CANnabinoids for Drug Resistant Epilepsy in Adults and Children
试验速览
- 阶段
- 2 期
- 状态
- 尚未招募
- 入组人数
- 90
- 主要终点
- Efficacy in reducing seizure frequency
研究概览
简要总结
Epilepsy is a neurological disorder affecting more than 50 million people globally, including more than 260,000 Canadians. Cannabidiol (CBD) reduces seizure frequency and improves quality of life for adults and children with Drug Resistant Epilepsy (DRE). Several uncontrolled, small, open label studies reported that CBD-enriched Cannabis Herbal Extract (CHE) resulted in a reduction of seizure frequency, but we lack critical information on efficacy, comparative effectiveness and dosing of CBD and ∆9-tetrahydrocannabinol (THC) in children and adults with DRE. CAN-DRE is an early phase, triple-blind, placebo-controlled, randomized clinical trial to answer the questions of if cannabinoids work to reduce seizures in children and adults (24 months to 55 years) with DRE and if CBD works better in an isolate or in a CBD-enriched Cannabis Herbal Extract. The primary outcome of CAN-DRE is reported monthly seizure count from baseline to maintenance phase.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 24 Months 至 55 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Ages 24 months to 55 years old at the time of enrollment
- •Diagnosed with DRE: not achieved seizure freedom, with adequate trials of 2 antiseizure medications 29
- •Medical history of 4 + clinically recognizable seizures (any type with clusters counted as a single event) per month
- •Have a negative pregnancy test at screening for patients who have experienced menarche
- •Agree to abstain from driving and recreational cannabis use throughout the study
排除标准
- •Diagnosis of psychogenic non-epileptic seizure
- •Recent (<30 days) change in anticonvulsant therapies including anticonvulsant medications, or settings on vagal nerve stimulator
- •Ketogenic diet started within 6 months (participants stable on the ketogenic diet for more than 6 months are eligible to participate)
- •Vagal nerve stimulator implanted and activated within 12 months
- •Concomitant regular use of narcotics (except in emergencies and physician supervised)
- •Initiation or dosage change of oral or injected steroids within 3 months
- •Allergy or intolerance to compounds in trial preparations
- •DRE secondary to progressive neurological disease
- •Clinically significant cardiac, renal or hepatic disease (as assessed by site investigator); elevated liver enzymes (GGT and/or AST and/or ALT) or lipase >3 times upper limit, adjusted for age
- •History of psychotic disorders
- •Uncontrolled (in the perspective of the qualified investigator) medical conditions including substance use disorders
- •History or concurrent cannabis use disorder
- •Unwilling or unable to use highly effective methods of contraception throughout the study period and three months post-trial, where applicable
研究组 & 干预措施
Arm 1 - Placebo Arm (MPL-012)
Placebo arm -> MPL-012 oil, each mL contains 0mg CBD and 0mg THC.
干预措施: Placebo (Drug)
Arm 2 - CBD Isolate (MPL-015)
CBD-Isolate arm -> MPL-015 oil, each ml contains 100mg CBD and 0mg THC.
干预措施: CBD Isolate (Drug)
Arm 3 - CBD-CHE (MPL-016)
CBD-CHE arm -> MPL-016 oil, a CBD-enriched cannabis herbal extract, each ml contains 100mg CBD and 3mg THC.
干预措施: CBD CHE (Drug)
结局指标
主要结局
Efficacy in reducing seizure frequency
时间窗: 116 days
reported monthly seizure count from baseline to maintenance
Cannabinoid-related AEs and DLTs
时间窗: 116 + 60 days
The frequency and type of adverse events and dose limiting toxicities (DLTs) reported by caregivers and participants throughout the trial participation
次要结局
- participant/family acceptability of this trial design(116 days)
- Quality of Life reported by adult participant/family(116 days)
- health resource utilization and changes(176 days)
- changes in work and activity impairment affected by seizure(116 days)
- Quality of Life reported by pediatric participants and family(116 days)
研究者
Lauren Kelly
PhD, MSc, BMedSci, CCRP, Associate Professor, Dept. of Pediatrics & Child Health, University of Manitoba Scientific Director, the Canadian Collaborative for Childhood Cannabinoid Therapeutics (https://www.medcannkids.ca/)
University of Manitoba