Clinical trial · Observational
Influence of CYP2C19 Genotype on Safety and Efficacy of Voriconazole in Pediatric Patients With Hematologic Malignancy
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
Voriconazole is a drug used to treat invasive fungal infections. The amount of voriconazole in a person's blood helps to determine how effectively it treats an infection, and how safe it is. Patients respond differently when receiving the same dose - some clearly benefit, other patients experience side effects, and others see limited improvement in their infection. Voriconazole is broken down in the liver mainly by an enzyme called CYP2C19, before being excreted from the body. The activity of CY2C19 differs between people because of variation in the DNA that encodes the body's instructions to make CYP2C19. If CYP2C19 activity is very high, voriconazole blood levels may remain below the target range when a patient receives a standard dose of voriconazole, which may be insufficient to treat their infection. Besides, children tend to have faster voriconazole metabolism regardless of the genetic makeup, mainly because of higher liver mass/body proportion. That's why, younger patients needs higher doses and it is harder for them to reach target range. Having a high CYP2C19 activity and being young combined may cause to consider choosing an alternative drug. By contrast, decreased CYP2C19 activity due to genetic variation may result in excessively high voriconazole blood levels, predisposing to serious side effects. Therefore, knowing a patient's CYP2C19 genetic makeup is very important for predicting their response to voriconazole. Thus, we aim to determine the influence of genetic variation in CYP2C19 on the frequency and severity of side effects related to voriconazole, and on the effectiveness of voriconazole for treating serious fungal infections. The findings from this study will contribute to determining the optimal dose of voriconazole that patients with different genetic variants in CYP2C19 should be started on, and will take us one step closer to both understanding the genetic structure of CYP2C19 in the Turkish population, and to 'personalised medicine'.
Conditions
Conditions (1)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Adverse Drug Reactions | — | UNRESOLVED | — |
Interventions
Interventions (1)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| Voriconazole | Drug | — | UNRESOLVED |
Design
Arms and outcomes
Arms (1)
- label
- Voriconazole
- description
- All eligible patients will be followed up for voriconazole related adverse drug events and efficacy
- interventionNames
- Drug: Voriconazole
Primary outcomes (1)
- measure
- Frequency and severity of voriconazole related adverse drug reactions
- timeFrame
- Follow up period for primary outcome is 30 days for each participant
- description
- Voriconazole related adverse drug reactions of grade 3-5 systematically evaluated using Liverpool Causality Assessment Tool identified during study follow up. Severity of all adverse events will be graded according to the National Cancer Institute's Common Terminology Criteria for Adverse Events (NCI-CTCAE) version 5.0.
Secondary outcomes (3)
- measure
- Non-response to voriconazole
- timeFrame
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 2 Years
- Maximum age
- 18 Years
Show eligibility criteria text
Inclusion Criteria: * Aged between ≥2 years and \<18 years * Receiving voriconazole for the treatment of an invasive fungal infection (IFI) and are within the first five days of the treatment course, OR are receiving voriconazole for the secondary prophylaxis against IFI during or after chemotherapy * Agree to give one additional blood sample twice during the study for the purposes of pharmacogenetic analysis and determination of the serum trough voriconazole concentration, whilst their blood is being collected during normal clinical follow up without the requirement for any additional intravenous intervention * Are willing and able to give informed consent and sign the informed consent form (by the patients and the parents OR the legal guardians) Exclusion Criteria: * Have previously had CYP2C19 PGx testing performed * Pregnant/breastfeeding * Have cognitive impairment and/or psychiatric disorders and/or any other condition that will draw into question their capacity to provide informed consent * Have severe hepatic insufficiency (Child-Pugh Class C) and have renal failure (estimated GFR \<15ml/min /1,73m2)
References
Publications (0)
Data not yet available