Amyotrophic Lateral Sclerosis, Motor Neuron Disease
Conditions
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.
Interventions
50 mg of CBD: \<2mg of THC in one ml. The cannabis oil consists of CBD extract in MCT oil.
DRUGPlacebo Oil
Placebo will contain only hemp seed oil.
Sponsors
Bod Australia
Gold Coast Hospital and Health Service
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
25 Years to 80 Years
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Difference in mean Forced Vital Capacity (FVC) volume between groups at end of treatment [efficacy] | Baseline to Day 180 | Change from baseline in Forced Vital Capacity volume on the Lung Function Test at 24 weeks |
| 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] | 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. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Difference in mean Numeric Rating Scale for spasticity total score between groups at end of treatment (Scores 0-100) | Baseline to Day 180 | 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. |
| 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 | 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. |
| 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 | 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. |
| 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 | 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. |
| Nature and number of adverse events [safety and tolerability] | Baseline to Day 180 | Number of Participants With Treatment-Related Adverse Events as Assessed by CTCAE v4.0 at 24 weeks |
Other
| Measure | Time frame | Description |
|---|---|---|
| Difference in mean Edinburgh Cognitive and Behavioural ALS Screen (ECAS) total score between groups at end of treatment (Score 0-136) | Baseline to Day 180 | Change from baseline in quality of life total score on the ALS Specific Quality of Life- Revised (ALSSQOL-R) total score at 24 weeks. Total score ranges from 0 to 100. Higher score represent better outcome. |
Countries
Australia
Outcome results
None listed