Minimising Adverse Drug Reactions and Verifying Economic Legitimacy in Children (MARVEL-PIC)
Trial statusRecruiting
Trial phaseNot applicable
Trial typeInterventional
Biological sexAll
AgeUp to 18
SponsorMurdoch Childrens Research Institute
About this trial
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 criteria
This trial does not accept healthy volunteersQualifiers
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.
Disqualifiers
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.
Trial design
Design model
Parallel
Treatments tested in this trial
Release of Extended Pharmacogenomics Report at Week 1
Diagnostic testWhole 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.
Release of Extended Pharmacogenomics Report at Week 13
Diagnostic testWhole 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.
Treatment groups
880 Participants
are divided into 2 treatment groupsGroup A: Standard of CareOther 1 intervention
Group B: Experimental ArmExperimental treatment 2 interventions
Trial outcomes
Primary outcomes
1
Reduction in the number of adverse drug reactions (ADRs)
The primary outcome is a reduction in adverse drug reactions among patients with actionable pharmacogenomic variants. An adverse drug reaction will be considered as any CTCAE grade 2 and above for non-haematological toxicities or CTCAE grade 3 and above for haematological toxicities. CTCAE grades are defined as 1,2,3,4 or 5, with 5 indicating the most severe.
Time frame
12 weeksSecondary outcomes
1
Occurrence of at least one ADR which contributes to primary endpoint
An adverse drug reaction will be considered as any CTCAE grade 2 and above for non-haematological toxicities or CTCAE grade 3 and above for haematological toxicities. This includes an ADR which was caused either by the index drug of inclusion or any subsequent drug. CTCAE grades are defined as 1,2,3,4 or 5, with 5 indicated the most severe.
Time frame
12 weeks2
Occurrence of at least one causal, clinically relevant, drug-genotype specific ADR, attributable to the index drug.
The Liverpool ADR CAT is an assessment tool produced to attribute causality to specific drugs for the grading of ADR symptomatic toxicity. Possible grading of ADRs include either definite, probable or possible.
Time frame
12 weeks3
Number of self-reported ADRs
The Ped-PRO-CTCAE survey is an electronic patient reported outcome measurement survey developed to evaluate symptomatic toxicity in patients who are receiving cancer therapy. Symptoms are evaluated for attributes of frequency, severity, interference, amount, presence or absence. Each symptomatic adverse event (AE) is assessed by 1-3 attributes that can be linked back to a CTCAE grade. The number of self-reported ADRs are measured independent of severity or drug-gene association but caused by one of the chemotherapeutic /targeted agents.
Time frame
12 weeks4
Number of serious self-reported ADRs
The Ped-PRO-CTCAE survey is an electronic patient reported outcome measurement survey developed to evaluate symptomatic toxicity in patients who are receiving cancer therapy. Symptoms are evaluated for attributes of frequency, severity, interference, amount, presence or absence. Each symptomatic adverse event (AE) is assessed by 1-3 attributes that can be linked back to a CTCAE grade. The number of self-reported ADRs are measured independent of severity or drug-gene association but caused by one of the chemotherapeutic /targeted agents. An ADR will be classified as serious if that event led to death, serious deterioration in health, or fetal distress.
Time frame
12 weeksSponsors and contacts
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Source ClinicalTrials.gov