A Randomised, Double-blind, Single-centre Study on the Safety, Tolerability and Efficacy of Cannabis Based Medicine Extract (MediCabilis CBD Oil) in Slowing the Disease Progression in Amyotrophic Lateral Sclerosis or Motor Neurone Disease Patients
试验速览
- 阶段
- 3 期
- 状态
- 进行中（未招募）
- 入组人数
- 17
- 试验地点
- 1
- 主要终点
- Difference in mean ALS Functional Rating Scale-Revised (ALSFRS-R) total score between groups at end of treatment (Total score: min 0- max 48) [efficacy]
研究概览
简要总结
This is a randomised, double-blind, placebo controlled study on a cannabis-based medicine extract (MediCabilis CBD Oil), in patients with Amyotrophic Lateral Sclerosis or Motor Neurone Disease. Participants will be randomised in a 1:1 ratio to receive MediCabilis CBD Oil or placebo oil. The treatment duration is 6 months with one-month safety follow up. Participants will be checked every month either face to face or via telephone and will be assessed to collect data for study objectives such as ALSFRS-R, Forced Vital Capacity, pain and spasticity score, and quality of life. Thirty (30) participants will be randomised.
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
- 25 Years 至 80 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Affected by ALS/MND, either of definite or probable according to the El Escorial revised criteria
- •Can provide written informed consent
- •Able and willing to comply with all study requirement
- •Male or female, ages 25-80 years old
- •Onset of first symptom within the last 2 years
- •Forced Vital Capacity (FVC) of at least 60% on baseline
排除标准
- •Participants who are bedridden
- •Have used or taken cannabis or cannabinoid-based medications within 30 days of study entry
- •History of any psychiatric disorder other than depression associated with their underlying condition including immediate family history of schizophrenia
- •Heavy consumption of alcohol or use of illicit drug
- •Hypersensitivity to cannabinoids or any of the excipients
- •Any of the following: eGFR <30 mL/min/1.73m2, ejection fraction <35%, or ASL and ALT >5 X ULN
- •Unwillingness of a female participant of child bearing potential, or their partner, to use effective contraception during the study and 30 days thereafter
- •Pregnant, lactating mother or female participant planning pregnancy during the course of the study and for 30 days thereafter
- •Received any investigational drug or medical device within 30 days prior randomisation
- •Any other significant disease or disorder which, in the opinion of the investigator, may either put the participant at risk because of participation in the study, or may influence the result of the study, or the participant's ability to participate in the study
- •Inability to cooperate with the study procedures
- •Unwilling to stop driving vehicle or operating dangerous machinery whilst on study drug.
- •Close affiliation with the study team, e.g. close relative of the investigator
研究组 & 干预措施
MediCabilis CBD Oil
干预措施: MediCabilis CBD Oil (Drug)
Placebo Oil
干预措施: Placebo Oil (Drug)
结局指标
主要结局
Difference in mean ALS Functional Rating Scale-Revised (ALSFRS-R) total score between groups at end of treatment (Total score: min 0- max 48) [efficacy]
时间窗: Baseline to Day 180
Change from baseline in ALS functional rating total scores on the ALSFRS-R at 24 weeks. Total score ranges from 0 to 48. Higher value represents better outcome.
Difference in mean Forced Vital Capacity (FVC) volume between groups at end of treatment [efficacy]
时间窗: Baseline to Day 180
Change from baseline in Forced Vital Capacity volume on the Lung Function Test at 24 weeks
次要结局
- Difference in mean Percentage of Total Weight Loss score between groups at end of treatment (Percentage score min: 0- max: 100)(Baseline to Day 180)
- Difference in mean ALS Specific Quality of Life- Revised (ALSSQOL-R) total score between groups at end of treatment (Total score min:0- max:460)(Baseline to Day 180)
- Difference in mean Numeric Rating Scale for spasticity total score between groups at end of treatment (Scores 0-100)(Baseline to Day 180)
- Difference in mean Numeric Rating Scale for pain total score between groups at end of treatment (Total score min:1-max:100)(Baseline to Day 180)
- Nature and number of adverse events [safety and tolerability](Baseline to Day 180)