Efficacy of Cannabinoids in Amyotrophic Lateral Sclerosis or Motor Neurone Disease

NCT03690791 | Unknown Phase 3 DMC

• 1 other identifier: GCMR0001
• Study Type: interventional
• Target: 17
• Locations: 1 country
• Sites: 1

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.

Conditions

Amyotrophic Lateral Sclerosis; Motor Neuron Disease

Outcome Measures

Primary Outcomes (2)

• 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 Outcomes (5)

• Nature and number of adverse events [safety and tolerability]

  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

• 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

Other Outcomes (1)

• 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

Study Arms (2)

MediCabilis CBD Oil

ACTIVE COMPARATOR

Drug: MediCabilis CBD Oil

Placebo Oil

PLACEBO COMPARATOR

Drug: Placebo Oil

Interventions

MediCabilis CBD Oil DRUG

50 mg of CBD: \<2mg of THC in one ml. The cannabis oil consists of CBD extract in MCT oil.

MediCabilis CBD Oil

Placebo Oil DRUG

Placebo will contain only hemp seed oil.

Placebo Oil

Eligibility Criteria

• Age: 25 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Gold Coast Hospital and Health Service: lead
• Bod Australia: collaborator

Study Sites (1)

Gold Coast Hospital and Health Service

Gold Coast, Queensland, 4215, Australia

Location

Related Publications (1)

• 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.

  PMID: 31719072 DERIVED

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis; Motor Neuron Disease

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Neurodegenerative Diseases; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER GOV
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 13, 2018
• First Posted: October 1, 2018
• Study Start: January 9, 2019
• Primary Completion: December 30, 2023
• Study Completion: January 30, 2024
• Last Updated: April 3, 2023

Record last verified: 2023-03

Data Sharing

• IPD Sharing: Will share

Locations

AU (1)