Pilot Study of MC in Paediatric Palliative Care

NCT05615389 | Recruiting Phase 1

• 1 other identifier: RCH HREC 88284
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 1

Brief Summary

The goal of this pilot study is to explore the feasibility and acceptability of a medicinal cannabis clinical trial into easing the symptoms of children undergoing palliative care for non-oncological conditions. The trial will evaluate the study design including recruitment strategy, medication tolerability, duration and outcomes to determine acceptability and feasibility for participating families. The data collected will then be used to design a full-scale multi-centre trial. Participants will be randomly allocated to receive one of two medicinal cannabis products. Neither the participants nor researchers will know the study drug allocation until the end of the trial.

Conditions

Palliative Care

Outcome Measures

Primary Outcomes (9)

• Study participant recruitment completion time, calculated as the time required to reach a sample size of 10.

  The time taken to complete recruitment will be calculated as the number of months from the date of commencing recruitment to the date of randomizing the tenth participant.

  From the date of pre-screening the first participant until the tenth participant is randomized, up to 2 years.

• Participant withdrawal rate, calculated as the number of participants who withdraw from the trial as a proportion of the total number of participants randomized.

  The number of participants who withdraw from the trial will be calculated as a proportion of the total number of participants randomized.

  Day 1 to day 43 (date of the end of the maintenance dosing period clinic visit)

• Study medication tolerability, as indicated by the proportion of participants who tolerate the protocol dosing schedule.

  The number of participants who adhere to the protocol dosing schedule without medication related protocol deviations, treatment discontinuations or dose modifications will be calculated as a proportion of the total sample for each arm (C12T12 and C20T5).

  Day 1 to day 43 (date of the end of the maintenance dosing period clinic visit)

• Participant adherence to the study medication dosing schedule, calculated as the proportion of participants who demonstrate acceptable medication compliance.

  Medication compliance will be assessed through pharmacy calculations from returned bottle volumes. Acceptable compliance will fall within the range of 80-120%. The number of participants with acceptable medication compliance will be reported as a proportion of the total sample randomized.

  Day 58 (date of end of treatment)

• Study visit attendance, calculated as the proportion of visits completed across the study sample.

  The number of study visits attended by all participants will be calculated as a proportion of the total possible visits in accordance with the study protocol.

  Screening to day 43 (date of the end of the maintenance dosing period clinic visit)

• Blood test completion, calculated as the proportion of blood tests completed across the study sample.

  The number of study blood tests completed by all participants will be calculated as a proportion of the total possible blood tests in accordance with the study protocol.

  Screening to day 43 (date of the end of the maintenance dosing period clinic visit)

• Parent questionnaire completion, calculated as the proportion of parent-report questionnaires completed across the study sample.

  The number of study questionnaires completed by all parents will be calculated as a proportion of the total possible questionnaires requiring completion in accordance with the study protocol.

  Screening to day 86 (participant trial completion)

• Self-report questionnaire completion, calculated as the proportion of adolescent self-report questionnaires completed across the study sample.

  The number of study self-report questionnaires completed by all participants will be calculated as a proportion of the total possible questionnaires requiring completion in accordance with the study protocol.

  Screening to day 86 (participant trial completion)

• Study design acceptability will be evaluated through a parent-completed study specific evaluation questionnaire completed at the end of the study.

  Study design acceptability will be assessed using an evaluation questionnaire developed specifically for this study, which uses Likert scales to assess satisfaction with recruitment, medication tolerability, frequency of study visits, burden of completing questionnaires, and overall study quality. Parents will complete this questionnaire at the end of their study participation (day 86). Data will be reported for each item individually, as the proportion of parents who responded positively on the Likert scale, where higher scores indicate more favorable responses.

  Day 86 (participant trial completion)

Secondary Outcomes (2)

• 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.

  Day 43 (date of the end of the maintenance dosing period clinic visit)

• The frequency of adverse events as reported throughout the study will be summarized across the two medicinal cannabis treatment arms.

  Day 1 to day 86 (participant trial completion)

Study Arms (2)

Medicinal Cannabis C12T12

EXPERIMENTAL

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

Drug: Medicinal Cannabis - C12T12

Medicinal Cannabis C20T5

EXPERIMENTAL

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

Drug: Medicinal Cannabis - C20T5

Interventions

Medicinal Cannabis - C12T12 DRUG

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.

Medicinal Cannabis C12T12

Medicinal Cannabis - C20T5 DRUG

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.

Medicinal Cannabis C20T5

Eligibility Criteria

• Age: 6 Months - 21 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Males and females aged 6 months to 21 years of age;
• Receiving care in the Victorian Paediatric Palliative Care Program for a non-oncological condition;
• 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:
• Frequency: "Frequently" or "Almost Constantly", AND
• Severity: "Moderate", "Severe", or "Very Severe", AND
• Distress: "Quite a bit", or "Very much";
• No changes in medication or other interventions in the two weeks prior to randomization;
• Participant and family have the ability to comply with the protocol requirements, in the opinion of the investigator;
• Agrees not to drive for the duration of the study.

You may not qualify if:

• Non-English speaking parents.
• Participant history of psychosis, schizophrenia, bipolar disorder, or major depressive disorder, or a first degree family history of psychosis.
• 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.
• Abnormal liver function tests defined as ALT \> 3 x ULN
• Current use of illicit drugs or medicinal cannabis, or use in the 4 weeks prior to screening
• Pregnant or intending to become pregnant during the study, or breastfeeding.
• History of clinically significant suicidal thoughts in the prior 12 months.
• Life expectancy less than 3 months in the opinion of the investigators
• Allergy to any of the components in the investigatory products (eg sunflower oil)
• Diagnosis of a malignant condition

Sponsors & Collaborators

• Murdoch Childrens Research Institute: lead

Study Sites (1)

Royal Children's Hospital / Murdoch Children's Research Institute

Parkville, Victoria, 3052, Australia

RECRUITING

Study Officials

• A/Prof Daryl Efron

  Murdoch Childrens Research Institute

  PRINCIPAL INVESTIGATOR

Central Study Contacts

A/Prof Daryl Efron

CONTACT

+61 (3) 8341 6200; mctrials@mcri.edu.au

Clinical Trial Coordinator

CONTACT

+61 (3) 8341 6200; mctrials@mcri.edu.au

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: SUPPORTIVE CARE
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 7, 2022
• First Posted: November 14, 2022
• Study Start: February 19, 2024
• Primary Completion (Estimated): October 1, 2026
• Study Completion (Estimated): December 1, 2026
• Last Updated: December 24, 2025

Record last verified: 2025-12

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP
• Time Frame: 6 months after publication of primary outcome
• Access Criteria: 1\) Data access agreement; 2) approval by Trial Steering Committee; 3) recognized research institutions.

Locations

AU (1)