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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT06510699
Registration number
NCT06510699
Ethics application status
Date submitted
14/07/2024
Date registered
19/07/2024
Titles & IDs
Public title
Pharmacogenomics for Better Treatment of Fungal Infections in Cancer
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Scientific title
Randomized Clinical Trial to Evaluate the Use of Genotype-based Dosing of Voriconazole in Patients With Haematological Malignancy
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Secondary ID [1]
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PRAGMATIC 01
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Universal Trial Number (UTN)
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Trial acronym
PRAGMATIC
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Fungal Infection
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Haematological Malignancy
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Blood Cancer
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Infectious Disease
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Condition category
Condition code
Infection
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Studies of infection and infectious agents
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Infection
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Other infectious diseases
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Infection
0
0
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Sexually transmitted infections
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Cancer
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0
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0
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Leukaemia - Acute leukaemia
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Cancer
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Leukaemia - Chronic leukaemia
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Other interventions - genotype-directed dosing with dosing software based on therapeutic drug monitoring
Experimental: Precision Care - Voriconazole dosing will be initiated using current standard care dosing. Samples for TDM and genotype testing will be collected. Based on the results of these tests on Day 5, 8, 14, and up to day 30 ( ± 1 day) patients will be evaluated for dose adjustment using dosing software that includes patient data including TDM and genotype data.
No intervention: Standard Care - Current standard of care at trial-site institutions uses weight-based (mg/kg) initial dosing of voriconazole, with dose adjustment based on standard therapeutic drug monitoring (TDM) results of measured voriconazole concentrations and based on clinical judgement.
Other interventions: genotype-directed dosing with dosing software based on therapeutic drug monitoring
Genotype-directed dosing with dosing software based on therapeutic drug monitoring
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Intervention code [1]
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Other interventions
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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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Therapeutic trough voriconazole concentration at Day 8
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Assessment method [1]
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Proportion of patients with measured therapeutic trough voriconazole concentration at Day 9 (+/- 1 day)
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Timepoint [1]
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Day 8
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Secondary outcome [1]
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Simulated therapeutic trough voriconazole exposure at Day 8
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Assessment method [1]
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Proportion of patients with simulated therapeutic trough voriconazole exposure at Day 8
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Timepoint [1]
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Day 8
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Secondary outcome [2]
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Measured therapeutic trough voriconazole exposure at Day 14.
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Assessment method [2]
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Proportion of patients with measured therapeutic trough voriconazole exposure at Day 14.
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Timepoint [2]
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Day 14
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Secondary outcome [3]
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Simulated therapeutic trough voriconazole exposure at Day 14
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Assessment method [3]
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Proportion of patients with simulated therapeutic trough voriconazole exposure at Day 14
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Timepoint [3]
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Day 14
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Secondary outcome [4]
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Measured therapeutic trough voriconazole exposure at both Days 8 and 14
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Assessment method [4]
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Proportion of patients with measured therapeutic trough voriconazole exposure at both Days 8 and 14
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Timepoint [4]
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Days 8 and 14
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Secondary outcome [5]
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Simulated therapeutic trough voriconazole exposure at both Days 8 and 14
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Assessment method [5]
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Proportion of patients with simulated therapeutic trough voriconazole exposure at both Days 8 and 14
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Timepoint [5]
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Days 8 and 14
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Secondary outcome [6]
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Simulated therapeutic trough voriconazole exposure from Day 8 to Day 30
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Assessment method [6]
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Proportion of patients with simulated therapeutic trough voriconazole exposure from Day 8 to Day 30
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Timepoint [6]
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Day 8 to Day 30
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Secondary outcome [7]
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Days to achieve first measured therapeutic trough voriconazole exposure
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Assessment method [7]
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Number of days to achieve first measured therapeutic trough voriconazole exposure
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Timepoint [7]
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Assessed over 30 days
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Secondary outcome [8]
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Doses to achieve first measured therapeutic trough voriconazole exposure.
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Assessment method [8]
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Number of doses to achieve first measured therapeutic trough voriconazole exposure.
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Timepoint [8]
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Assessed over 30 days
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Secondary outcome [9]
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Percent difference in dose when dose adjustment is performed
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Assessment method [9]
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Percent difference in dose when dose adjustment is performed (i) on the first occasion and (ii) on subsequent occasions (set to 0 if no adjustment).
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Timepoint [9]
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Assessed over 30 days
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Secondary outcome [10]
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Number of days of antifungal therapy
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Assessment method [10]
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Number of days of antifungal therapy
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Timepoint [10]
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Assessed over 30 days
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Secondary outcome [11]
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Number of doses of antifungal therapy
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Assessment method [11]
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Number of doses of antifungal therapy
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Timepoint [11]
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Assessed over 30 days
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Secondary outcome [12]
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Clinical cure or stable disease
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Assessment method [12]
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Proportion of patients achieving invasive fungal disease (IFD) clinical cure or stable disease; defined by treating team, if indication is IFD treatment
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Timepoint [12]
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Assessed over 30 days
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Secondary outcome [13]
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Patients with no reported fungal infection during course
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Assessment method [13]
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Proportion of patients with no reported fungal infection during course, if indication is IFD prophylaxis.
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Timepoint [13]
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Assessed over 30 days
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Secondary outcome [14]
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Hospital free days
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Assessment method [14]
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Number of hospital free days at day 30
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Timepoint [14]
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Day 30
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Secondary outcome [15]
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Length of hospital stay post-randomisation
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Assessment method [15]
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Length of hospital stay post-randomisation
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Timepoint [15]
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Assessed over 30 days
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Secondary outcome [16]
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Number of patients experiencing at least one adverse event
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Assessment method [16]
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Number of patients experiencing at least one adverse event
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Timepoint [16]
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Assessed over 30 days
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Secondary outcome [17]
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Number and type of adverse events
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Assessment method [17]
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Number and type of adverse events
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Timepoint [17]
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Assessed over 30 days
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Secondary outcome [18]
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All-cause mortality at 30 days
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Assessment method [18]
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All-cause mortality at 30 days
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Timepoint [18]
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Assessed over 30 days
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Secondary outcome [19]
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Clinical success
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Assessment method [19]
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Proportion of patients achieving clinical success, defined as an absence of a clinical deterioration or event that requires a change of therapy
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Timepoint [19]
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Assessed over 30 days
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Eligibility
Key inclusion criteria
* Age = 2 years.
* Written informed consent obtained.
* Decision to prescribe voriconazole.
* Diagnosed with haematological malignancy or disorder.
* Admitted to a trial site, or sufficient outpatient follow-up appointments are feasible
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Minimum age
2
Years
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Maximum age
No limit
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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
* Post-allogeneic haematopoietic stem cell transplant (HCT) patient, without access to pre HCT DNA
* Death is likely imminent within 7 days.
* Previously randomised to this trial
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Study design
Purpose of the study
Treatment
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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
Phase 2
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Not yet 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
1/11/2024
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Actual
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Date of last participant enrolment
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Actual
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Date of last data collection
Anticipated
2/09/2026
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Actual
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Sample size
Target
104
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Accrual to date
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Final
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Recruitment in Australia
Recruitment state(s)
NSW,QLD,SA,VIC
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Recruitment hospital [1]
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Sydney Children's Hospital Network - Sydney
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Recruitment hospital [2]
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Westmead Hospital - Sydney
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Recruitment hospital [3]
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Royal Brisbane & Women's Hospital - Brisbane
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Recruitment hospital [4]
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Children's Hospital Queensland - South Brisbane
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Recruitment hospital [5]
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Royal Adelaide Hospital - Adelaide
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Recruitment hospital [6]
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Peter MacCallum Cancer Centre - Melbourne
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Recruitment postcode(s) [1]
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21452031 - Sydney
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Recruitment postcode(s) [2]
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2145 - Sydney
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Recruitment postcode(s) [3]
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4029 - Brisbane
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Recruitment postcode(s) [4]
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4029 - South Brisbane
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Recruitment postcode(s) [5]
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5000 - Adelaide
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Recruitment postcode(s) [6]
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3053 - Melbourne
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Recruitment outside Australia
Country [1]
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United States of America
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State/province [1]
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Washington
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Funding & Sponsors
Primary sponsor type
Other
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Name
The University of Queensland
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Address
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Country
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Other collaborator category [1]
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Other
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Name [1]
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Metro North Hospital and Health Service
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Address [1]
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Country [1]
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Other collaborator category [2]
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Other
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Name [2]
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Fred Hutchinson Cancer Research Center/University of Washington Cancer Consortium
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Address [2]
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Country [2]
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Other collaborator category [3]
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Other
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Name [3]
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University of Sydney
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Address [3]
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Country [3]
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Other collaborator category [4]
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Other
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Name [4]
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Western Sydney Local Health District
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Address [4]
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Country [4]
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Other collaborator category [5]
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Other
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Name [5]
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Sydney Children's Hospitals Network
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Address [5]
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Country [5]
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Other collaborator category [6]
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Other
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Name [6]
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Peter MacCallum Cancer Centre, Australia
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Address [6]
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Country [6]
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Other collaborator category [7]
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Other
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Name [7]
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University of Melbourne
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Address [7]
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Country [7]
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Other collaborator category [8]
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Other
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Name [8]
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Queensland Children's Hospital
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Address [8]
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Country [8]
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Other collaborator category [9]
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Other
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Name [9]
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Royal Adelaide Hospital
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Address [9]
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Country [9]
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Other collaborator category [10]
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Other
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Name [10]
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Pathology Queensland
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Address [10]
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Country [10]
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Other collaborator category [11]
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Government body
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Name [11]
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Royal Brisbane and Women's Hospital
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Address [11]
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Country [11]
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Ethics approval
Ethics application status
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Summary
Brief summary
This project aims to address invasive fungal infections in patients with blood cancer, by precision dosing of voriconazole based on CYP2C19 genotype testing with Bayesian dose-forecasting dosing software to develop patient-centric and maximally effective dosing regimens. This study investigates if voriconazole increases the proportion of patients achieving therapeutic exposure at day 8 of dosing compared with standard care; and will assess factors that influence the implementation of genotype testing and dosing software in the healthcare system, including fidelity, feasibility, acceptability and cost-effectiveness. It will recruit at least 104 kids and adults in a parallel-group randomised clinical trial. A hybrid feasibility sub-study will assess the scalability of genotype-directed dosing to ensure sustainable integration of the interventions into the clinical workflow. A health economic sub-study will evaluate the costs, health outcomes and cost-effectiveness of genotype-directed testing compared to standard care.
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Trial website
https://clinicaltrials.gov/study/NCT06510699
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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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Address
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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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Jason A Roberts, PhD
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Address
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Country
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Phone
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+61 7 3346 5032
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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)?
Undecided
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No/undecided IPD sharing reason/comment
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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/NCT06510699