Personalized drug prescribing to reduce side effects in children with cancer
Minimising Adverse Drug Reactions and Verifying Economic Legitimacy - Pharmacogenomics Implementation in Children (MARVEL-PIC)
This study is testing if using personalized genetic information to choose cancer medications can help reduce side effects for children undergoing treatment.
Quick facts
| Phase | Not applicable |
|---|---|
| Study type | Interventional |
| Enrollment | 880 (estimated) |
| Ages | N/A to 18 Years |
| Sex | All |
| Sponsor | Murdoch Childrens Research Institute Academic / other |
| Locations | 4 sites (Randwick, New South Wales and 3 other locations) |
| Trial ID | NCT05667766 on ClinicalTrials.gov |
What this trial studies
This study evaluates the effectiveness of personalized pharmacogenomic testing in reducing adverse drug reactions (ADRs) in pediatric cancer patients. Participants will be randomly assigned to receive either personalized guided prescribing based on pharmacogenomic testing or standard care for a duration of 12 weeks. The study will also assess the economic impact and quality of life through patient-reported outcomes over a follow-up period of up to 12 months. By utilizing genetic testing to inform drug selection and dosing, the study aims to minimize the trial-and-error approach traditionally used in pediatric oncology.
Who should consider this trial
Good fit: Ideal candidates are children under 18 years old with a new cancer diagnosis or those undergoing their first hematopoietic stem cell transplantation.
Not a fit: Patients over 18 years old or those with a life expectancy of less than three months may not benefit from this study.
Why it matters
Potential benefit: If successful, this approach could significantly reduce harmful side effects from cancer treatments, improving patient safety and quality of life.
How similar studies have performed: Previous studies have shown promise in using pharmacogenomic testing to improve treatment outcomes, suggesting this approach may be effective.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * 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 Criteria: * 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.
Where this trial is running
Randwick, New South Wales and 3 other locations
- Sydney Children's Hospital — Randwick, New South Wales, Australia (Recruiting)
- Women's and Children's Hospital — North Adelaide, South Australia, Australia (Recruiting)
- The Royal Children's Hospital — Parkville, Victoria, Australia (Recruiting)
- Perth Children's Hospital — Nedlands, Washington, Australia (Recruiting)
Study contacts
- Principal investigator: A/Prof Rachel Conyers — The Royal Children's Hospital/Murdoch Children's Research Institute
- Study coordinator: Tayla Stenta
- Email: pharmaco.genomics@mcri.edu.au
- Phone: 03 9345 5533
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.