Neoplasms Clinical Trial
— MARVEL-PICOfficial title:
Minimising Adverse Drug Reactions and Verifying Economic Legitimacy - Pharmacogenomics Implementation in Children (MARVEL-PIC)
NCT number | NCT05667766 |
Other study ID # | 89083 |
Secondary ID | |
Status | Recruiting |
Phase | N/A |
First received | |
Last updated | |
Start date | March 22, 2023 |
Est. completion date | August 2026 |
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.
Status | Recruiting |
Enrollment | 880 |
Est. completion date | August 2026 |
Est. primary completion date | August 2025 |
Accepts healthy volunteers | No |
Gender | All |
Age group | N/A to 18 Years |
Eligibility | Inclusion Criteria: - Age < 18 years - New cancer diagnosis or patient receiving HSCT. - Must receive a first prescription for one or more of the drugs for which the CPIC guideline is available, which is prescribed to them in routine care. - 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 < 40 kgs, 12 mL > 40kgs) - 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. |
Country | Name | City | State |
---|---|---|---|
Australia | Perth Children's Hospital | Nedlands | Western Australia |
Australia | Women's and Children's Hospital | North Adelaide | South Australia |
Australia | The Royal Children's Hospital | Parkville | Victoria |
Australia | Sydney Children's Hospital | Randwick | New South Wales |
Lead Sponsor | Collaborator |
---|---|
Murdoch Childrens Research Institute |
Australia,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | 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. | 12 weeks | |
Secondary | 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. | 12 weeks | |
Secondary | 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. | 12 weeks | |
Secondary | 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. | 12 weeks | |
Secondary | 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. | 12 weeks | |
Secondary | Number of dose adjustments | Number of dose adjustments made to drug-gene associated therapy during the entire study follow up by review of the participants electronic medical record and medication reconciliation. | 12 weeks | |
Secondary | Incidence of drug cessation due to ADR | Incidence of drug cessation due to ADR during the entire study follow up. Relates to inclusion criteria drug by review of the participants electronic medical record and medication reconciliation. | 12 months | |
Secondary | Incidence of drug cessation due to lack of efficacy | Incidence of drug cessation due to lack of efficacy by review of the participants electronic medical record and medication reconciliation. | 12 months | |
Secondary | Therapeutic drug monitoring | Therapeutic drug monitoring (routine drug levels) performed during entire study follow up period by review of the participants electronic medical record and medication reconciliation. | 12 months | |
Secondary | Physician and Pharmacist adherence to the CPIC guidelines | After discussion and consensus of a pharmacogenetic variant. A clinical report will be generated with the results and therapeutic recommendations according to Clinical Pharmacogenetics Implementation Consortium (CPIC) guidelines. Adherence to recommendations will be reported with reasoning, during the 12 week period. | 12 months | |
Secondary | Health care expenditure related to adverse events using MBS/PBS data | Data will be collected on the costs of management comparing the standard of care arm versus the study arm. The actual costs assigned to each inpatient or outpatient episode will be collected. This information is readily accessible through Services Australia and The Department of Health via The Centre for Victorian Data Linkage (CVDL). | 12 months | |
Secondary | Change in quality of life outcomes using CHU9D | The University of Sheffield CHU9D Quality of Life Survey will be used to monitor changes in patient QoL. The CHU9D consists of a descriptive system and set of preference weights, for 9 questions that assess participants daily functioning across the following domains: worry, sadness, pain, tiredness, annoyance, school, sleep, daily routine and activities. The tool allows for calculation of quality adjusted life years for use in cost utility analysis | Baseline, Week 12, 12 months |
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