A Phase 2 interventional study of genotype-directed dosing with dosing software based on therapeutic drug monitoring in Fungal Infection, Haematological Malignancy and Blood Cancer, sponsored by The University of Queensland. Recruiting at 7 sites in 2 countries. Open to participants aged 2 Years and older, including healthy volunteers. Per ClinicalTrials.gov, last updated 2026-04-16.
Sponsored by The University of Queensland · Phase 2, Interventional, and Treatment
This project aims to address invasive fungal infections in patients, 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.
Participants will be randomly assigned to standard care or precision 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 based on clinical judgement. In precision care, voriconazole dosing will be initiated using current standard dosing. Samples for the TDM and genotype testing will be collected. Based on results of these tests on Day 5 (+/- 1 day) patients will be evaluated for dose adjustment using dosing software that includes patient data, TDM and genotype data.
Trial procedures: following baseline data collection and randomisation genotype testing will be performed on Day 1. The precision care group have dose adjustment performed on Days 5, 9, 15, and 22 using genotype and/or TDM results in dosing software. The standard care group will have TDM performed, and dose adjustments in accordance with usual clinical practice. Blood sampling for TDM will be performed 24-hours prior to dose adjustment, with additional blood samples collected on Days 1 and 2 in both standard care and precision care groups. All blood sampling, genotype testing and dose adjustments will be performed +/- day to support feasibility.
The primary objective is to compare the proportion of patients achieving therapeutic voriconazole exposure at Day 8 when using precision care compared to standard care.
Secondary objectives are:
The implementation feasibility sub-study will assess the scalability of precision care to support optimal voriconazole dosing by tailoring the intervention to each trial setting and measuring outcomes with the involvement of key stakeholders (end-users, health administrators, consumers, community members).
Data will be collected to ascertain Fidelity including: 1) assess barriers and enablers; 2) identify prioritise the factors influencing delivery and tailor these to fit local settings; 3) assess intended fidelity to the precision care intervention.
Data will be collected to ascertain Feasibility or the extent to which precision care can be successfully used in each study setting via completion of the feasibility implementation measure (FIM) at baselines and quarterly thereafter, including qualitative interviews at the end of the study.
Data will be collected to ascertain Acceptability or whether end-users perceive precision care as agreeable or satisfactory by survey and qualitative interviews with pharmacists, physicians and health administrators.
Data will be collected to undertake an economic evaluation to determine the cost-effectiveness of precision versus standard care, exploring individual level data, incremental cost effectiveness ratios (ICERs) at day 14 and 30; and cost-utility analysis to demonstrate if precision care offers value for money at Day 30 in the Australian setting. The health economic evaluation will include use of surveys to capture: 1) healthcare usage of trial participants; 2) implementation feasibility measures; and 3) implementation costs.
Data will be collected to ascertain scalability or the ability to expand the efficacy of precision care on a small scale in controlled conditions to real world conditions to a greater proportion of the population.
Therapeutic trough voriconazole exposures (concentrations) In this trial, serum concentrations > 1 mg/L (minimum effective concentration) and \< 5 mg/L (maximum safe concentration) are defined as the therapeutic range. Treating clinicians may nominate an individualised patient target within this range prior to randomisation and that range will be applied during the trial. Where dosing software is being applied, the software will be programmed to target 2.5 mg/L.
260 studies on the registry are indexed under Mycoses; 37 are open to participants now.
This study's planned enrollment of 104 is above the median of 68 across 164 interventional studies indexed under Mycoses.
Browse Mycoses studies →The University of Queensland is the lead sponsor of 91 studies on the registry; 24 are open to participants now.
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Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Stem cell (bone marrow) transplants. Allogeneic uses cells from a donor; autologous uses your own cells, collected earlier and given back.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Allogeneic and autologous transplant
Stem cell (bone marrow) transplants. Allogeneic uses cells from a donor; autologous uses your own cells, collected earlier and given back.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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.
Other: genotype-directed dosing with dosing software based on therapeutic drug monitoring
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.
Genotype-directed dosing with dosing software based on therapeutic drug monitoring
Also known as: genotype directed with dosing software
Therapeutic trough voriconazole concentration at Day 8
Proportion of patients with measured therapeutic trough voriconazole concentration at Day 9 (+/- 1 day)
Time frame: Day 8
Simulated therapeutic trough voriconazole exposure at Day 8
Proportion of patients with simulated therapeutic trough voriconazole exposure at Day 8
Time frame: Day 8
Measured therapeutic trough voriconazole exposure at Day 14.
Proportion of patients with measured therapeutic trough voriconazole exposure at Day 14.
Time frame: Day 14
Simulated therapeutic trough voriconazole exposure at Day 14
Proportion of patients with simulated therapeutic trough voriconazole exposure at Day 14
Time frame: Day 14
Measured therapeutic trough voriconazole exposure at both Days 8 and 14
Proportion of patients with measured therapeutic trough voriconazole exposure at both Days 8 and 14
Time frame: Days 8 and 14
Simulated therapeutic trough voriconazole exposure at both Days 8 and 14
Proportion of patients with simulated therapeutic trough voriconazole exposure at both Days 8 and 14
Time frame: Days 8 and 14
Simulated therapeutic trough voriconazole exposure from Day 8 to Day 30
Proportion of patients with simulated therapeutic trough voriconazole exposure from Day 8 to Day 30
Time frame: Day 8 to Day 30
Days to achieve first measured therapeutic trough voriconazole exposure
Number of days to achieve first measured therapeutic trough voriconazole exposure
Time frame: Assessed over 30 days
Doses to achieve first measured therapeutic trough voriconazole exposure.
Number of doses to achieve first measured therapeutic trough voriconazole exposure.
Time frame: Assessed over 30 days
Percent difference in dose when dose adjustment is performed
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).
Time frame: Assessed over 30 days
Number of days of antifungal therapy
Number of days of antifungal therapy
Time frame: Assessed over 30 days
Number of doses of antifungal therapy
Number of doses of antifungal therapy
Time frame: Assessed over 30 days
Clinical cure or stable disease
Proportion of patients achieving invasive fungal disease (IFD) clinical cure or stable disease; defined by treating team, if indication is IFD treatment
Time frame: Assessed over 30 days
Patients with no reported fungal infection during course
Proportion of patients with no reported fungal infection during course, if indication is IFD prophylaxis.
Time frame: Assessed over 30 days
Hospital free days
Number of hospital free days at day 30
Time frame: Day 30
Length of hospital stay post-randomisation
Length of hospital stay post-randomisation
Time frame: Assessed over 30 days
Number of patients experiencing at least one adverse event
Number of patients experiencing at least one adverse event
Time frame: Assessed over 30 days
Number and type of adverse events
Number and type of adverse events
Time frame: Assessed over 30 days
All-cause mortality at 30 days
All-cause mortality at 30 days
Time frame: Assessed over 30 days
Clinical success
Proportion of patients achieving clinical success, defined as an absence of a clinical deterioration or event that requires a change of therapy
Time frame: Assessed over 30 days
Plan to share: Undecided
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Started Apr 2025 (actual). Primary completion is estimated for Sep 2026, 16 months after the start.
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The University of Queensland