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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT05667766




Registration number
NCT05667766
Ethics application status
Date submitted
19/12/2022
Date registered
29/12/2022

Titles & IDs
Public title
Minimising Adverse Drug Reactions and Verifying Economic Legitimacy in Children (MARVEL-PIC)
Scientific title
Minimising Adverse Drug Reactions and Verifying Economic Legitimacy - Pharmacogenomics Implementation in Children (MARVEL-PIC)
Secondary ID [1] 0 0
89083
Universal Trial Number (UTN)
Trial acronym
MARVEL-PIC
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Neoplasms 0 0
Bone Marrow Transplantation 0 0
Condition category
Condition code
Other 0 0 0 0
Research that is not of generic health relevance and not applicable to specific health categories listed above

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Diagnosis / Prognosis - Release of Extended Pharmacogenomics Report at Week 1
Diagnosis / Prognosis - Release of Extended Pharmacogenomics Report at Week 13

Other: Standard of Care - Standard of Care prescribing for period of 12 months. Participants will receive pharmacogenomic test results according to the current standard of care. The participants will be followed up for a minimum of 12 weeks, with maximal time-period being 12 months depending on time of enrolment.

Experimental: Experimental Arm - Extended Pharmacogenomic prescribing for a period of 12 months. Participants will receive pharmacogenomic testing across a range of clinically relevant variants, to guide the dose and drug selection of 27 drugs commonly used in supportive care. The participants will be followed up for a minimum of 12 weeks, with maximal time-period being 12 months depending on time of enrolment.


Diagnosis / Prognosis: Release 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.

Diagnosis / Prognosis: Release 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.
Intervention code [1] 0 0
Diagnosis / Prognosis
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Reduction in the number of adverse drug reactions (ADRs)
Timepoint [1] 0 0
12 weeks
Secondary outcome [1] 0 0
Occurrence of at least one ADR which contributes to primary endpoint
Timepoint [1] 0 0
12 weeks
Secondary outcome [2] 0 0
Occurrence of at least one causal, clinically relevant, drug-genotype specific ADR, attributable to the index drug.
Timepoint [2] 0 0
12 weeks
Secondary outcome [3] 0 0
Number of self-reported ADRs
Timepoint [3] 0 0
12 weeks
Secondary outcome [4] 0 0
Number of serious self-reported ADRs
Timepoint [4] 0 0
12 weeks
Secondary outcome [5] 0 0
Number of dose adjustments
Timepoint [5] 0 0
12 weeks
Secondary outcome [6] 0 0
Incidence of drug cessation due to ADR
Timepoint [6] 0 0
12 months
Secondary outcome [7] 0 0
Incidence of drug cessation due to lack of efficacy
Timepoint [7] 0 0
12 months
Secondary outcome [8] 0 0
Therapeutic drug monitoring
Timepoint [8] 0 0
12 months
Secondary outcome [9] 0 0
Physician and Pharmacist adherence to the CPIC guidelines
Timepoint [9] 0 0
12 months
Secondary outcome [10] 0 0
Health care expenditure related to adverse events using MBS/PBS data
Timepoint [10] 0 0
12 months
Secondary outcome [11] 0 0
Change in quality of life outcomes using CHU9D
Timepoint [11] 0 0
Baseline, Week 12, 12 months

Eligibility
Key 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.
Minimum age
No limit
Maximum age
18 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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.

Study design
Purpose of the study
Other
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Parallel
Other design features
Phase
Not applicable
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
22/03/2023
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/08/2026
Actual
Sample size
Target
880
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,SA,VIC,WA
Recruitment hospital [1] 0 0
Sydney Children's Hospital - Randwick
Recruitment hospital [2] 0 0
Women's and Children's Hospital - North Adelaide
Recruitment hospital [3] 0 0
The Royal Children's Hospital - Parkville
Recruitment hospital [4] 0 0
Perth Children's Hospital - Nedlands
Recruitment postcode(s) [1] 0 0
2031 - Randwick
Recruitment postcode(s) [2] 0 0
5006 - North Adelaide
Recruitment postcode(s) [3] 0 0
3052 - Parkville
Recruitment postcode(s) [4] 0 0
6009 - Nedlands

Funding & Sponsors
Primary sponsor type
Other
Name
Murdoch Childrens Research Institute
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
A/Prof Rachel Conyers
Address 0 0
The Royal Children's Hospital/Murdoch Children's Research Institute
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Tayla Stenta
Address 0 0
Country 0 0
Phone 0 0
03 9345 5533
Fax 0 0
Email 0 0
pharmaco.genomics@mcri.edu.au
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
What data in particular will be shared?
The de-identified data set collected for this analysis of the study will be available from 6 months after publication of the primary outcome. Only de-identified data will published. The study protocol, analysis plan and consent forms will also be available. The data may be obtained from the Murdoch Children's Research Institute by emailing MCTC@mcri.edu.au.

Supporting document/s available: Study protocol, Statistical analysis plan (SAP), Informed consent form (ICF)
When will data be available (start and end dates)?
The de-identified data set collected for the analysis of the trial will be available from six months after publication of the primary outcome. The study protocol can be obtained from Murdoch Children's Research Institute. Prior to access to any data the following would be required: a data access agreement must be signed by all relevant parties, the investigators of the study must see and approve the analysis plan describing how the data will be analysed. There must be also an agreement around appropriate acknowledgement in any future publications.
Available to whom?
The data may be obtained from the Melbourne Children's Trials Centre (MCTC) at Murdoch Children's Research Institute by emailing MCTC@mcri.edu.au.
Available for what types of analyses?
How or where can data be obtained?


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT05667766