RecruitingNot ApplicableNCT05667766

Minimising Adverse Drug Reactions and Verifying Economic Legitimacy in Children (MARVEL-PIC)

Minimising Adverse Drug Reactions and Verifying Economic Legitimacy - Pharmacogenomics Implementation in Children (MARVEL-PIC)


Sponsor

Murdoch Childrens Research Institute

Enrollment

880 participants

Start Date

Mar 22, 2023

Study Type

INTERVENTIONAL

Conditions

Summary

A prospective, open, randomised implementation study in paediatric cancer patients. The study aims to determine whether a personalised approach will result in an overall reduction in clinically relevant adverse drug reactions (ADRs) and to evaluate the economic and quality of life impacts. Participants will be randomised to receive personalised guided prescribing of supportive care therapy (study arm) or standard of care (control arm) for a period of 12 weeks. The follow up period includes prospective patient reporting of symptoms and quality of life through electronically delivered surveys, for a maximum of 12 months.


Eligibility

Max Age: 18 Years

Inclusion Criteria9

  • Age < 18 years
  • New cancer diagnosis or patient receiving HSCT or patient has a relapsed cancer diagnosis and is starting treatment after more than 6 months without.
  • Starting treatment with a chemotherapeutic agent that is not single agent oral targeted therapy.
  • Must also be taking a medication for which there is an established CPIC guideline available.
  • Parent or patient is able and willing to give consent for patient to take part and be followed up for at least 12 weeks.
  • Patient is amenable to venepuncture and blood draw (5mL ideally with an absolute minimum requirement of 2.5 mL) or has Whole Genome Sequencing available (WGS).
  • Patient and/or parent is able and willing to sign an informed consent form.
  • Patient and/or parent is able to complete Ped-PRO-CTCAE survey in English, Italian or Chinese.
  • Study enrolment limit has not been reached.

Exclusion Criteria9

  • Age > 18 years.
  • Patient has a life expectancy estimated to be less than three months by the treating clinical team.
  • Duration of the drug of inclusion total treatment length is planned to be less than one week.
  • Patient and/or parent is unable to consent to the study.
  • Patient and/or parent is unwilling to take part in the study.
  • Patient and/or parent is able unable to complete Ped-PRO-CTCAE survey in English, Italian or Chinese.
  • Patient has existing impaired hepatic or renal function for which a lower dose or alternate drug selection are already part of current routine care.
  • Patient has a glomerular filtration rate of less than 15 mL/min per 1.73m2.
  • Patient has advanced liver failure.

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Interventions

DIAGNOSTIC_TESTRelease of Extended Pharmacogenomics Report at Week 1

Whole genome sequencing with reporting on current standard of care (SoC) pharmacogenomic variants (TPMT, NUD15) where applicable (i.e, for patients with diagnosis of acute lymphoblastic leukaemia) by Week 1. Whole genome sequencing on a broader number of actionable variants as per international guidelines for cancer supportive care (identical to study arm) will be reported on at Week 13.

DIAGNOSTIC_TESTRelease of Extended Pharmacogenomics Report at Week 13

Whole genome sequencing with reporting on current standard of care (SoC) pharmacogenomic variants (TPMT, NUD15) where applicable (i.e, for patients with diagnosis of acute lymphoblastic leukaemia) and reporting of a broader number of actionable pharmacogenomic variants as per international guidelines for cancer supportive care reported on by Week 1.


Locations(4)

Sydney Children's Hospital

Randwick, New South Wales, Australia

Women's and Children's Hospital

North Adelaide, South Australia, Australia

The Royal Children's Hospital

Parkville, Victoria, Australia

Perth Children's Hospital

Nedlands, Washington, Australia

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NCT05667766


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