Pilot Study of MC in Paediatric Palliative Care

NCT ID: NCT05615389

Last Updated: 2025-12-24

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE1/PHASE2

Total Enrollment

10 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-02-19

Study Completion Date

2026-12-31

Brief Summary

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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.

Detailed Description

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This is a single site, double-blind, randomized, pilot study of 10 participants investigating two medicinal cannabis (MC) products in children aged 6 months to 21 years who are undergoing palliative care for non-oncological conditions. Eligible participants will be randomized 1:1 to receive one of two medicinal cannabis products (C12T12 or C20T5).

The primary objective of this pilot study is to evaluate all elements of the study design (recruitment strategy, study duration, study procedures, study medication tolerance and outcome measures) to assess if they are acceptable and feasible for the conduct of a full-scale randomised trial of MC to reduce symptom burden in paediatric patients undergoing palliative care for non-oncological conditions.

The secondary objective of this study is to collect preliminary data on the safety of two different oral MC oil formulations in this population.

Each participants will be involved in the trial for up to 100 days:

* Screening period: Up to 14 days
* Up-titration: day 1 to day 16
* Maintenance treatment period: day 17 to day 42
* Down-titration: day 43 (end of maintenance period clinic visit) to day 58
* Post-treatment follow-up: day 59 to day 86.

Conditions

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Palliative Care

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

SUPPORTIVE_CARE

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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Medicinal Cannabis C12T12

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

Group Type EXPERIMENTAL

Medicinal Cannabis - C12T12

Intervention Type 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 C20T5

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

Group Type EXPERIMENTAL

Medicinal Cannabis - C20T5

Intervention Type 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.

Interventions

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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.

Intervention Type DRUG

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 Type DRUG

Eligibility Criteria

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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.

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
Minimum Eligible Age

6 Months

Maximum Eligible Age

21 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Murdoch Childrens Research Institute

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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A/Prof Daryl Efron

Role: PRINCIPAL_INVESTIGATOR

Murdoch Childrens Research Institute

Locations

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

Parkville, Victoria, Australia

Site Status RECRUITING

Countries

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Australia

Central Contacts

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A/Prof Daryl Efron

Role: CONTACT

Phone: +61 (3) 8341 6200

Email: [email protected]

Clinical Trial Coordinator

Role: CONTACT

Phone: +61 (3) 8341 6200

Email: [email protected]

Facility Contacts

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Clinical Trial Coordinator

Role: primary

Other Identifiers

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RCH HREC 88284

Identifier Type: -

Identifier Source: org_study_id