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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)
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Scientific title
Minimising Adverse Drug Reactions and Verifying Economic Legitimacy - Pharmacogenomics Implementation in Children (MARVEL-PIC)
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Secondary ID [1]
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89083
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Universal Trial Number (UTN)
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Trial acronym
MARVEL-PIC
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Neoplasms
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0
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Bone Marrow Transplantation
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0
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Condition category
Condition code
Other
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Research that is not of generic health relevance and not applicable to specific health categories listed above
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Intervention/exposure
Study type
Interventional
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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.
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Intervention code [1]
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Diagnosis / Prognosis
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Reduction in the number of adverse drug reactions (ADRs)
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Assessment method [1]
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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.
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Timepoint [1]
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12 weeks
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Secondary outcome [1]
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Occurrence of at least one ADR which contributes to primary endpoint
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Assessment method [1]
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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.
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Timepoint [1]
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12 weeks
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Secondary outcome [2]
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Occurrence of at least one causal, clinically relevant, drug-genotype specific ADR, attributable to the index drug.
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Assessment method [2]
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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.
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Timepoint [2]
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12 weeks
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Secondary outcome [3]
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Number of self-reported ADRs
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Assessment method [3]
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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.
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Timepoint [3]
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12 weeks
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Secondary outcome [4]
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Number of serious self-reported ADRs
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Assessment method [4]
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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.
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Timepoint [4]
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12 weeks
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Secondary outcome [5]
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Number of dose adjustments
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Assessment method [5]
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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.
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Timepoint [5]
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12 weeks
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Secondary outcome [6]
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Incidence of drug cessation due to ADR
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Assessment method [6]
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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.
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Timepoint [6]
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12 months
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Secondary outcome [7]
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Incidence of drug cessation due to lack of efficacy
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Assessment method [7]
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Incidence of drug cessation due to lack of efficacy by review of the participants electronic medical record and medication reconciliation.
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Timepoint [7]
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12 months
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Secondary outcome [8]
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Therapeutic drug monitoring
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Assessment method [8]
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Therapeutic drug monitoring (routine drug levels) performed during entire study follow up period by review of the participants electronic medical record and medication reconciliation.
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Timepoint [8]
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12 months
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Secondary outcome [9]
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Physician and Pharmacist adherence to the CPIC guidelines
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Assessment method [9]
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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.
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Timepoint [9]
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12 months
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Secondary outcome [10]
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Health care expenditure related to adverse events using MBS/PBS data
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Assessment method [10]
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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).
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Timepoint [10]
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12 months
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Secondary outcome [11]
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Change in quality of life outcomes using CHU9D
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Assessment method [11]
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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
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Timepoint [11]
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Baseline, Week 12, 12 months
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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.
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Minimum age
No limit
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Maximum age
18
Years
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Sex
Both males and females
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Can healthy volunteers participate?
No
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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.
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Study design
Purpose of the study
Other
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Allocation to intervention
Randomised controlled trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Parallel
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Other design features
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Phase
NA
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Recruiting
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
22/03/2023
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
1/08/2026
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Actual
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Sample size
Target
880
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Accrual to date
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Final
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Recruitment in Australia
Recruitment state(s)
NSW,SA,VIC,WA
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Recruitment hospital [1]
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Sydney Children's Hospital - Randwick
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Recruitment hospital [2]
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Women's and Children's Hospital - North Adelaide
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Recruitment hospital [3]
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The Royal Children's Hospital - Parkville
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Recruitment hospital [4]
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Perth Children's Hospital - Nedlands
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Recruitment postcode(s) [1]
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2031 - Randwick
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Recruitment postcode(s) [2]
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5006 - North Adelaide
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Recruitment postcode(s) [3]
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3052 - Parkville
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Recruitment postcode(s) [4]
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6009 - Nedlands
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Funding & Sponsors
Primary sponsor type
Other
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Name
Murdoch Childrens Research Institute
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Address
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Country
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Ethics approval
Ethics application status
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Summary
Brief 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.
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Trial website
https://clinicaltrials.gov/study/NCT05667766
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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A/Prof Rachel Conyers
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Address
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The Royal Children's Hospital/Murdoch Children's Research Institute
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Country
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Phone
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Fax
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Email
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Contact person for public queries
Name
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Tayla Stenta
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Address
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Country
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Phone
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03 9345 5533
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Fax
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Email
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[email protected]
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Contact person for scientific queries
Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
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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
[email protected]
.
Supporting document/s available: Study protocol, Statistical analysis plan (SAP), Informed consent form (ICF)
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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.
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Available to whom?
The data may be obtained from the Melbourne Children's Trials Centre (MCTC) at Murdoch Children's Research Institute by emailing
[email protected]
.
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Available for what types of analyses?
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How or where can data be obtained?
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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