Amyotrophic Lateral SclerosisPhase 3NCT03690791
Efficacy of Cannabinoids in Amyotrophic Lateral Sclerosis or Motor Neurone Disease
A Phase 3 study testing MediCabilis CBD Oil in people with Amyotrophic Lateral Sclerosis and Motor Neuron Disease, run by Gold Coast Hospital and Health Service at 1 site in Australia.
Trial 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. Read more about this study
- Who is it for?
- Ages 25 to 80 years
- Conditions the study is for
- Amyotrophic Lateral Sclerosis, Motor Neuron Disease
- What is being tested?
- MediCabilis CBD Oil
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 17 participants
- Where
- 1 siteAustralia
- Study dates
- 9 Jan 2019to 30 Jan 2024
- Phase
- Phase 3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 25 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Show all 13Show fewer
- 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
Trial locations and contacts
1 site in 1 country.
Australia1 site
Gold Coast Hospital and Health Service
Gold Coast, Queensland, 4215
About this study
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
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.
- Being tested
- MediCabilis CBD Oil, Placebo Oil
- Medical subject headings
- Amyotrophic Lateral Sclerosis, Motor Neuron Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MediCabilis CBD OilDrug
50 mg of CBD: <2mg of THC in one ml. The cannabis oil consists of CBD extract in MCT oil.
Placebo OilDrug
Placebo will contain only hemp seed oil.
| Study group | Role | What they receive |
|---|---|---|
| MediCabilis CBD Oil | Active comparator | Not described Receives: Drug: MediCabilis CBD Oil |
| Placebo Oil | Placebo comparator | Not described Receives: Drug: Placebo Oil |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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
Measured: Baseline to Day 180
Secondary outcomes (5)
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
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: Baseline to Day 180
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Oct 2018
- Study start9 Jan 2019
- Primary completion30 Dec 2023 (estimated)
- Study completion30 Jan 2024 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Amyotrophic Lateral SclerosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 17 participants (actual)
Sponsor and investigators
- Lead sponsor
- Gold Coast Hospital and Health Service
- Sponsor type
- Other government
- Collaborators
- Bod Australia
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03690791
- Sponsor's protocol ID
- GCMR0001
- Status last verified
- Mar 2023
- First submitted
- 13 Sep 2018
- Last updated
- 3 Apr 2023
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: This study will comply with the Australian Government Data Sharing Policy, the NHMRC Open Access Policy and the Clinical Trials Registration and Results Information Submission rule. Additional data can be requested from the authors. However, the decision to disclose data is solely based from authors' discretion and funding agency.
Publications
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.
PubMed 31719072
Study at a glance
From the registry record, last updated by the sponsor on 3 Apr 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT03690791
- Acronym / sponsor ID
- GCMR0001
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 17 participants (actual)
- Lead sponsor
- Gold Coast Hospital and Health ServiceOther government
- Collaborators
- Bod Australia
- Study start
- 9 Jan 2019 (actual)
- Primary completion
- 30 Dec 2023 (estimated)
- Study completion
- 30 Jan 2024 (estimated)
- First posted
- 1 Oct 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| MediCabilis CBD Oil | Active comparator | Drug: MediCabilis CBD Oil |
| Placebo Oil | Placebo comparator | Drug: Placebo Oil |
Who may qualify
- Ages
- 25 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- 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
Key exclusion criteria (13 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| MediCabilis CBD Oil50 mg of CBD: <2mg of THC in one ml. The cannabis oil consists of CBD extract in MCT oil. | Drug | MediCabilis CBD Oil |
| Placebo OilPlacebo will contain only hemp seed oil. | Drug | Placebo Oil |
Primary outcome
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]
Time frame: 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]
Time frame: Baseline to Day 180
Change from baseline in Forced Vital Capacity volume on the Lung Function Test at 24 weeks
5 secondary outcomes
Nature and number of adverse events [safety and tolerability]
Time frame: Baseline to Day 180
Difference in mean Numeric Rating Scale for spasticity total score between groups at end of treatment (Scores 0-100)
Time frame: 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)
Time frame: 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)
Time frame: 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)
Time frame: Baseline to Day 180
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Apr 2023.
- Derived from this study: PubMed 31719072Urbi 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.
- Individual participant dataSharing planned: Yes. This study will comply with the Australian Government Data Sharing Policy, the NHMRC Open Access Policy and the Clinical Trials Registration and Results Information Submission rule. Additional data can be requested from the authors. However, the decision to disclose data is solely based from authors' discretion and funding agency.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing MediCabilis CBD Oil in people with Amyotrophic Lateral Sclerosis and Motor Neuron Disease, run by Gold Coast Hospital and Health Service at 1 site in Australia.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 3 Apr 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Australia. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome 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] (time frame: Baseline to Day 180). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03690791, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Apr 2023.