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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT05615389




Registration number
NCT05615389
Ethics application status
Date submitted
7/11/2022
Date registered
14/11/2022

Titles & IDs
Public title
Pilot Study of MC in Paediatric Palliative Care
Scientific title
A Pilot Study of Medicinal Cannabis in Paediatric Patients Undergoing Palliative Care for Non-oncological Conditions
Secondary ID [1] 0 0
RCH HREC 88284
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Palliative Care 0 0
Condition category
Condition code

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Medicinal Cannabis - C12T12
Treatment: Drugs - Medicinal Cannabis - C20T5

Experimental: Medicinal Cannabis C12T12 - C12T12 Ruby Balanced Oil (Cannatrek Ltd, Australia). 12.5mg/ml CBD and 12.5mg/ml THC in sunflower oil.

Experimental: Medicinal Cannabis C20T5 - C20T5 Ruby CBD Oil (Cannatrek Ltd, Australia). 20mg/ml CBD and 5mg/ml THC in sunflower oil.


Treatment: Drugs: Medicinal Cannabis - C12T12
Participants randomised to receive C12T12 Ruby Balanced Oil will commence with 0.008ml/kg/day (0.1 mg/kg/day THC) in two divided doses, and titrate up in four steps (increase by 0.008ml/kg/day every four days) over 16 days up to a maintenance dose of 0.04ml/kg/day (0.5mg/kg/day THC) in two divided doses, with a ceiling dose of 2ml/day (25mg/day THC) for participants weighing 50kg or more. The maintenance dose will then be continued from day 17 until day 42.

Down-titration will occur for 16 days after the end of study visit, in the reverse of the up-titration period.

Treatment: Drugs: Medicinal Cannabis - C20T5
Participants randomised to the C20T5 Ruby CBD Oil will receive a matched volume to the C12T12 arm during the up-titration, maintenance, and down-titration phases.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Study participant recruitment completion time, calculated as the time required to reach a sample size of 10.
Timepoint [1] 0 0
From the date of pre-screening the first participant until the tenth participant is randomized, up to 2 years.
Primary outcome [2] 0 0
Participant withdrawal rate, calculated as the number of participants who withdraw from the trial as a proportion of the total number of participants randomized.
Timepoint [2] 0 0
Day 1 to day 43 (date of the end of the maintenance dosing period clinic visit)
Primary outcome [3] 0 0
Study medication tolerability, as indicated by the proportion of participants who tolerate the protocol dosing schedule.
Timepoint [3] 0 0
Day 1 to day 43 (date of the end of the maintenance dosing period clinic visit)
Primary outcome [4] 0 0
Participant adherence to the study medication dosing schedule, calculated as the proportion of participants who demonstrate acceptable medication compliance.
Timepoint [4] 0 0
Day 58 (date of end of treatment)
Primary outcome [5] 0 0
Study visit attendance, calculated as the proportion of visits completed across the study sample.
Timepoint [5] 0 0
Screening to day 43 (date of the end of the maintenance dosing period clinic visit)
Primary outcome [6] 0 0
Blood test completion, calculated as the proportion of blood tests completed across the study sample.
Timepoint [6] 0 0
Screening to day 43 (date of the end of the maintenance dosing period clinic visit)
Primary outcome [7] 0 0
Parent questionnaire completion, calculated as the proportion of parent-report questionnaires completed across the study sample.
Timepoint [7] 0 0
Screening to day 86 (participant trial completion)
Primary outcome [8] 0 0
Self-report questionnaire completion, calculated as the proportion of adolescent self-report questionnaires completed across the study sample.
Timepoint [8] 0 0
Screening to day 86 (participant trial completion)
Primary outcome [9] 0 0
Study design acceptability will be evaluated through a parent-completed study specific evaluation questionnaire completed at the end of the study.
Timepoint [9] 0 0
Day 86 (participant trial completion)
Secondary outcome [1] 0 0
The frequency of adverse events as reported on the modified version of the Liverpool Adverse Event Profile (LAEP) at day 43 will be summarized across the two medicinal cannabis treatment arms.
Timepoint [1] 0 0
Day 43 (date of the end of the maintenance dosing period clinic visit)
Secondary outcome [2] 0 0
The frequency of adverse events as reported throughout the study will be summarized across the two medicinal cannabis treatment arms.
Timepoint [2] 0 0
Day 1 to day 86 (participant trial completion)

Eligibility
Key inclusion criteria
1. Males and females aged 6 months to 21 years of age;
2. Receiving care in the Victorian Paediatric Palliative Care Program for a non-oncological condition;
3. Pain, dystonia and/or gut dysfunction parent-rated symptom score above threshold, defined by rating on the relevant revised Memorial Symptom Assessment Scale (MSAS) question(s) of:

1. Frequency: "Frequently" or "Almost Constantly", AND
2. Severity: "Moderate", "Severe", or "Very Severe", AND
3. Distress: "Quite a bit", or "Very much";
4. No changes in medication or other interventions in the two weeks prior to randomization;
5. Participant and family have the ability to comply with the protocol requirements, in the opinion of the investigator;
6. Agrees not to drive for the duration of the study.
Minimum age
6 Months
Maximum age
21 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1. Non-English speaking parents.
2. Participant history of psychosis, schizophrenia, bipolar disorder, or major depressive disorder, or a first degree family history of psychosis.
3. Taking medications which are known to interact with medicinal cannabis: warfarin, mTOR inhibitors (e.g sirolimus, tacrolimus), anti-cancer agents, citalopram >20mg/day, escitalopram >10mg/day.
4. Abnormal liver function tests defined as ALT > 3 x ULN
5. Current use of illicit drugs or medicinal cannabis, or use in the 4 weeks prior to screening
6. Pregnant or intending to become pregnant during the study, or breastfeeding.
7. History of clinically significant suicidal thoughts in the prior 12 months.
8. Life expectancy less than 3 months in the opinion of the investigators
9. Allergy to any of the components in the investigatory products (eg sunflower oil)
10. Diagnosis of a malignant condition

Study design
Purpose of the study
Other
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 1
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
19/02/2024
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/02/2025
Actual
Sample size
Target
10
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 0 0
Royal Children's Hospital / Murdoch Children's Research Institute - Parkville
Recruitment postcode(s) [1] 0 0
3052 - Parkville

Funding & Sponsors
Primary sponsor type
Other
Name
Murdoch Childrens Research Institute
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
A/Prof Daryl Efron
Address 0 0
Murdoch Childrens Research Institute
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
A/Prof Daryl Efron
Address 0 0
Country 0 0
Phone 0 0
+61 (3) 8341 6200
Fax 0 0
Email 0 0
[email protected]
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
What data in particular will be shared?
The de-identified data set collected for this analysis of the Pilot Study of MC in Paediatric Palliative Care will be available six months after publication of the primary outcome. The study protocol and analysis plan will also be available. The data must be obtained from the Murdoch Children's Research Institute. Prior to releasing any data the following are required: a data access agreement must be signed between relevant parties, the 'Pilot Study of MC in Paediatric Palliative Care' Study Management Group must see and approve the data analysis plan describing how the data will be analysed, there must be an agreement around appropriate acknowledgment and any additional costs involved must be covered. Should the Study Management Group be unavailable, this role is delegated to the Murdoch Children's Research Institute. Data will only be shared with a recognized research organisation which has approved the proposed analysis plan.

Supporting document/s available: Study protocol, Statistical analysis plan (SAP)
When will data be available (start and end dates)?
6 months after publication of primary outcome
Available to whom?
1) Data access agreement; 2) approval by Trial Steering Committee; 3) recognized research institutions.
Available for what types of analyses?
How or where can data be obtained?


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT05615389