Efficacy of Cannabinoids in Amyotrophic Lateral Sclerosis or Motor Neurone Disease

Study Overview
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Brief Summary
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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.

Official Title
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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

Conditions
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Amyotrophic Lateral Sclerosis, Motor Neuron Disease

Intervention / Treatment
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* Drug: MediCabilis CBD Oil
* Drug: Placebo Oil


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: 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 Exclusion Criteria: 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

Ages Eligible for Study
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Minimum Age: 25 Years
Maximum Age: 80 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Active Comparator: MediCabilis CBD Oil<br> | Drug: MediCabilis CBD Oil<br>* 50 mg of CBD: <2mg of THC in one ml. The cannabis oil consists of CBD extract in MCT oil.<br>|
| Placebo Comparator: Placebo Oil<br> | Drug: Placebo Oil<br>* Placebo will contain only hemp seed oil.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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] | 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. | Baseline to Day 180 | 
| Difference in mean Forced Vital Capacity (FVC) volume between groups at end of treatment [efficacy] | Change from baseline in Forced Vital Capacity volume on the Lung Function Test at 24 weeks | Baseline to Day 180 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Nature and number of adverse events [safety and tolerability] | Number of Participants With Treatment-Related Adverse Events as Assessed by CTCAE v4.0 at 24 weeks | Baseline to Day 180 | 
| Difference in mean Numeric Rating Scale for spasticity total score between groups at end of treatment (Scores 0-100) | Change from baseline in spasticity total score on the Numeric Rating Scale for spasticity at 24 weeks. Total score ranges from 0 to 100. Higher values represent better outcome. | 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) | Change from baseline in pain total score on the Numeric Rating Scale for pain at 24 weeks. Total score ranges from 0 to 100. Higher value represents better outcome. | Baseline to Day 180 | 
| Difference in mean Percentage of Total Weight Loss score between groups at end of treatment (Percentage score min: 0- max: 100) | Change from baseline in weight loss on the Percentage of Total Weight Loss at 24 weeks. Percentage ranges from 0 to 100. Higher value represents better outcome. | 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) | Change from baseline in quality of life total score on the ALS Specific Quality of Life- Revised (ALSSQOL-R) score at 24 weeks. Total score ranges from 0 to 460. Higher score represent better outcome. | Baseline to Day 180 |