Clinical trial NCT05615389: Pilot Study of MC in Paediatric Palliative Care
Official title: A Pilot Study of Medicinal Cannabis in Paediatric Patients Undergoing Palliative Care for Non-oncological Conditions
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE1
PHASE2
Conditions: Palliative Care
Lead sponsor: Murdoch Childrens Research Institute
Sponsor class: OTHER
First posted: 2022-11-14
Enrollment: 10
Sex: ALL
Minimum age (years): 6
Maximum age (years): 21
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: SUPPORTIVE_CARE

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.

Detailed description: 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.

Eligibility criteria: 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

Primary outcomes:
measure: Study participant recruitment completion time, calculated as the time required to reach a sample size of 10.
timeFrame: From the date of pre-screening the first participant until the tenth participant is randomized, up to 2 years.
measure: Participant withdrawal rate, calculated as the number of participants who withdraw from the trial as a proportion of the total number of participants randomized.
timeFrame: Day 1 to day 43 (date of the end of the maintenance dosing period clinic visit)
measure: Study medication tolerability, as indicated by the proportion of participants who tolerate the protocol dosing schedule.
timeFrame: Day 1 to day 43 (date of the end of the maintenance dosing period clinic visit)
measure: Participant adherence to the study medication dosing schedule, calculated as the proportion of participants who demonstrate acceptable medication compliance.
timeFrame: Day 58 (date of end of treatment)
measure: Study visit attendance, calculated as the proportion of visits completed across the study sample.
timeFrame: Screening to day 43 (date of the end of the maintenance dosing period clinic visit)
measure: Blood test completion, calculated as the proportion of blood tests completed across the study sample.
timeFrame: Screening to day 43 (date of the end of the maintenance dosing period clinic visit)
measure: Parent questionnaire completion, calculated as the proportion of parent-report questionnaires completed across the study sample.
timeFrame: Screening to day 86 (participant trial completion)
measure: Self-report questionnaire completion, calculated as the proportion of adolescent self-report questionnaires completed across the study sample.
timeFrame: Screening to day 86 (participant trial completion)
measure: Study design acceptability will be evaluated through a parent-completed study specific evaluation questionnaire completed at the end of the study.
timeFrame: Day 86 (participant trial completion)
Secondary outcomes:
measure: 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.
timeFrame: Day 43 (date of the end of the maintenance dosing period clinic visit)
measure: The frequency of adverse events as reported throughout the study will be summarized across the two medicinal cannabis treatment arms.
timeFrame: Day 1 to day 86 (participant trial completion)
Other outcomes:
Locations:
city: Parkville
country: Australia
facility: Royal Children's Hospital / Murdoch Children's Research Institute
state: Victoria
Study officials:
affiliation: Murdoch Childrens Research Institute
name: A/Prof Daryl Efron
role: PRINCIPAL_INVESTIGATOR
References: