Clinical trial NCT03690791: Efficacy of Cannabinoids in Amyotrophic Lateral Sclerosis or Motor Neurone Disease
Official title: 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
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE3
Conditions: Amyotrophic Lateral Sclerosis
Motor Neuron Disease
Lead sponsor: Gold Coast Hospital and Health Service
Sponsor class: OTHER_GOV
Collaborators: name: Bod Australia
First posted: 2018-10-01
Enrollment: 17
Sex: ALL
Minimum age (years): 25
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: 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.


Eligibility criteria: Inclusion Criteria:

1. Affected by ALS/MND, either of definite or probable according to the El Escorial revised criteria
2. Can provide written informed consent
3. Able and willing to comply with all study requirement
4. Male or female, ages 25-80 years old
5. Onset of first symptom within the last 2 years
6. Forced Vital Capacity (FVC) of at least 60% on baseline

Exclusion Criteria:

1. Participants who are bedridden
2. Have used or taken cannabis or cannabinoid-based medications within 30 days of study entry
3. History of any psychiatric disorder other than depression associated with their underlying condition including immediate family history of schizophrenia
4. Heavy consumption of alcohol or use of illicit drug
5. Hypersensitivity to cannabinoids or any of the excipients
6. Any of the following: eGFR \<30 mL/min/1.73m2, ejection fraction \<35%, or ASL and ALT \>5 X ULN
7. Unwillingness of a female participant of child bearing potential, or their partner, to use effective contraception during the study and 30 days thereafter
8. Pregnant, lactating mother or female participant planning pregnancy during the course of the study and for 30 days thereafter
9. Received any investigational drug or medical device within 30 days prior randomisation
10. 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
11. Inability to cooperate with the study procedures
12. Unwilling to stop driving vehicle or operating dangerous machinery whilst on study drug.
13. Close affiliation with the study team, e.g. close relative of the investigator

Primary outcomes:
measure: 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]
timeFrame: Baseline to Day 180
measure: Difference in mean Forced Vital Capacity (FVC) volume between groups at end of treatment [efficacy]
timeFrame: Baseline to Day 180
Secondary outcomes:
measure: Nature and number of adverse events [safety and tolerability]
timeFrame: Baseline to Day 180
measure: Difference in mean Numeric Rating Scale for spasticity total score between groups at end of treatment (Scores 0-100)
timeFrame: Baseline to Day 180
measure: Difference in mean Numeric Rating Scale for pain total score between groups at end of treatment (Total score min:1-max:100)
timeFrame: Baseline to Day 180
measure: Difference in mean Percentage of Total Weight Loss score between groups at end of treatment (Percentage score min: 0- max: 100)
timeFrame: Baseline to Day 180
measure: 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)
timeFrame: Baseline to Day 180
Other outcomes:
measure: Difference in mean Edinburgh Cognitive and Behavioural ALS Screen (ECAS) total score between groups at end of treatment (Score 0-136)
timeFrame: Baseline to Day 180
Locations:
city: Gold Coast
country: Australia
facility: Gold Coast Hospital and Health Service
state: Queensland
Study officials:
References:
citation: Urbi B, Broadley S, Bedlack R, Russo E, Sabet A. Study protocol for a randomised, double-blind, placebo-controlled study evaluating the Efficacy of cannabis-based Medicine Extract in slowing the disease pRogression of Amyotrophic Lateral sclerosis or motor neurone Disease: the EMERALD trial. BMJ Open. 2019 Nov 11;9(11):e029449. doi: 10.1136/bmjopen-2019-029449.