Pharmacogenomics to improve voriconazole dosing for fungal infections
Randomized Clinical Trial to Evaluate the Use of Genotype-based Dosing of Voriconazole
This trial will test whether using a patient's CYP2C19 genotype with Bayesian dosing software helps get voriconazole levels into the therapeutic range by Day 8 for people with fungal infections, including those with blood cancers.
Quick facts
| Phase | Phase 2 |
|---|---|
| Study type | Interventional |
| Enrollment | 104 (estimated) |
| Ages | 2 Years and up |
| Sex | All |
| Sponsor | The University of Queensland Academic / other |
| Locations | 7 sites (Seattle, Washington and 6 other locations) |
| Trial ID | NCT06510699 on ClinicalTrials.gov |
What this trial studies
This randomized, parallel-group Phase 2 trial will compare standard care dosing and therapeutic drug monitoring of voriconazole with a precision-care approach that adds CYP2C19 genotype results and Bayesian dose-forecasting software to guide dose adjustments. Participants aged 2 years and older who are starting voriconazole will be randomized to standard or genotype-directed dosing and will have blood sampling for therapeutic drug monitoring and genotype testing. Dose adjustments for the precision group will be guided by the dosing software on Days 5, 9, 15 and 22, while the standard group will follow usual clinical practice. The trial includes a hybrid feasibility sub-study to assess implementation and a health economic sub-study to estimate costs and cost-effectiveness, aiming to recruit at least 104 children and adults.
Who should consider this trial
Good fit: Ideal candidates are people aged 2 years or older for whom clinicians have decided to prescribe voriconazole and who can be managed at or followed up by one of the participating hospitals or outpatient clinics.
Not a fit: Patients unlikely to benefit include those post-allogeneic hematopoietic stem cell transplant without access to pre-transplant DNA, those not expected to survive seven days, or those already stably controlled on voriconazole outside the dosing window evaluated.
Why it matters
Potential benefit: If successful, the approach could help patients reach safe and effective voriconazole concentrations more quickly and consistently, potentially reducing treatment failure and drug-related toxicity.
How similar studies have performed: Prior observational studies and small interventional reports show CYP2C19 genotype influences voriconazole levels and suggest genotype-guided dosing can help, but large randomized data are limited.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Age ≥ 2 years. * Written informed consent obtained. * Decision to prescribe voriconazole. * Admitted to a trial site, or sufficient outpatient follow-up appointments are feasible 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
Where this trial is running
Seattle, Washington and 6 other locations
- Fred Hutchinson Cancer Centre — Seattle, Washington, United States (Recruiting)
- Sydney Children's Hospital Network — Sydney, New South Wales, Australia (Recruiting)
- Westmead Hospital — Sydney, New South Wales, Australia (Recruiting)
- Royal Brisbane & Women's Hospital — Brisbane, Queensland, Australia (Recruiting)
- Children's Hospital Queensland — South Brisbane, Queensland, Australia (Recruiting)
- Royal Adelaide Hospital — Adelaide, South Australia, Australia (Recruiting)
- Peter MacCallum Cancer Centre — Melbourne, Victoria, Australia (Recruiting)
Study contacts
- Principal investigator: Jason A Roberts, PhD — The University of Queensland
- Study coordinator: Jason A Roberts, PhD
- Email: j.roberts2@uq.edu.au
- Phone: +61 7 3346 5032
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.