Pilot Study of MC in Paediatric Palliative Care

Condition(s) studied

Investigational drug(s) / intervention(s)

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.

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.

Study 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.

Eligibility

Primary outcome measure(s)

• Study participant recruitment completion time, calculated as the time required to reach a sample size of 10. — From the date of pre-screening the first participant until the tenth participant is randomized, up to 2 years.
  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.
• Participant withdrawal rate, calculated as the number of participants who withdraw from the trial 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)
  The number of participants who withdraw from the trial will be calculated as a proportion of the total number of participants randomized.
• Study medication tolerability, as indicated by the proportion of participants who tolerate the protocol dosing schedule. — Day 1 to day 43 (date of the end of the maintenance dosing period clinic visit)
  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).
• Participant adherence to the study medication dosing schedule, calculated as the proportion of participants who demonstrate acceptable medication compliance. — Day 58 (date of end of treatment)
  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.
• Study visit attendance, calculated as the proportion of visits completed across the study sample. — Screening to day 43 (date of the end of the maintenance dosing period clinic visit)
  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.
• Blood test completion, calculated as the proportion of blood tests completed across the study sample. — Screening to day 43 (date of the end of the maintenance dosing period clinic visit)
  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.
• Parent questionnaire completion, calculated as the proportion of parent-report questionnaires completed across the study sample. — Screening to day 86 (participant trial completion)
  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.
• Self-report questionnaire completion, calculated as the proportion of adolescent self-report questionnaires completed across the study sample. — Screening to day 86 (participant trial completion)
  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.
• Study design acceptability will be evaluated through a parent-completed study specific evaluation questionnaire completed at the end of the study. — Day 86 (participant trial completion)
  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.

Trial sites (1)

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