Invasive Fungal InfectionsPhase 4VORIGENPHARMNCT04238884
Effectiveness and Efficiency of a Voriconazole Preemptive Genotyping Strategy
A Phase 4 study testing Voriconazole preemptive genotyping strategy and Voriconazole clinical practice in people with Invasive Fungal Infections, run by Instituto de Investigación Hospital Universitario La Paz at 1 site in Spain.
Trial summary
This is a phase IV pragmatic, multicentre, randomised, simple-blind, parallel arm, centre-stratified clinical trial. The main objective is to compare efficiency of voriconazole preemptive genotyping strategy, compared with routine practice. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Invasive Fungal Infections
- What is being tested?
- Voriconazole preemptive genotyping strategy and Voriconazole clinical practice
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 146 participants
- Where
- 1 siteSpain
- Study dates
- 2 Jan 2020to 31 Dec 2022
- Phase
- Phase 4
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Patient at risk of developing invasive aspergillosis, that will potentially receive treatment or prophylaxis with voriconazole:
- Pediatric population: children who are going to receive a transplant of hematopoietic precursors (HSCT) and acute myeloid leukemias, as well as relapses of it.
- Adult population: patients diagnosed with acute leukemia, and those patients with expected prolonged neutropenia, secondary to hematological process and / or after specific treatment (aplastic anemia and variants, myelodysplastic syndrome, solid organ or bone marrow transplant, etc.), and those whose responsible clinician consider individually that they could present a risk of developing a fungal infection.
- Those who agree to participate in the study by signing informed consent (patients equal or over 18 years old)
- Subjects under 18 years old whose representative / legal guardian has voluntarily signed the informed consent.
- In the case of mature under 18 years subjects (12-17 years of age), in addition to the consent signed by the legal guardian, the consent of the subject will be obtained.
Who cannot join
- Patients who for any reason should not be included in the study according to the criteria of the research team.
- Subjects who are not capable to understand the information sheet and unable to sign the informed consent.
Trial locations and contacts
1 site in 1 country.
Spain1 site, 1 recruiting
- Recruiting
La Paz University Hospital
Madrid, 28046
Alberto M Borobia, PI, +34917277558, alberto.borobia@salud.madrid.org
About this study
Official title: Randomized and Multicenter Clinical Trial to Evaluate the Effectiveness and Efficiency of a Voriconazole Preemptive Genotyping Strategy in Patients With Risk of Aspergillosis
This is a phase IV pragmatic, multicentre, randomised, simple-blind, parallel arm, centre-stratified clinical trial. The main objective is to compare efficiency of voriconazole preemptive genotyping strategy, compared with routine practice.
Read the detailed description
Primary outcome is serum level of voriconazole on fifth day. Secondary outcome is a combined variable of therapeutic failure and adverse events, associated with voriconazole. A total of 146 patients with risk of undergoing invasive aspergillosis who potentially will receive voriconazole will be recruited, and CYP2C19 will be genotyped. If the patient receives voriconazole finally, he will be randomized (1:1 experimental/control). In the experimental arm patients receive dose according to pharmacogenetic algorithm including CYP2C19 genotype and clinical and demographic information. In the control arm patients receive dose according to clinical practice guidelines. In addition, a Spain national health system (NHS) point-of-view cost-effectiveness evaluation is going to be done. Direct costs calculation of each arm will be done.
- Conditions
- Invasive Fungal Infections
- Being tested
- Voriconazole preemptive genotyping strategy, Voriconazole clinical practice
- Medical subject headings
- Invasive Fungal Infections
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Voriconazole preemptive genotyping strategyDrug
The patients who finally receives voriconazole will be randomized to receive the dose according to a pharmacogenetic algorithm including CYP2C19 genotype and clinical and demographic information.
Voriconazole clinical practiceDrug
The patients who finally receives voriconazole will be randomized to receive the dose according to clinical practice
| Study group | Role | What they receive |
|---|---|---|
| Experimental group | Experimental | Based on the genetic study carried out and the patient's characteristics (age, weight, indication), the Pharmacogenetics Unit of the University Hospital La Paz will indicate the dose to be administered based on the therapeutic individualization protocol guided by pharmacogenetics. Receives: Drug: Voriconazole preemptive genotyping strategy |
| Control group | Active comparator | No information will be provided and procedure will be carried out according to normal clinical practice, with clinical monitoring by the doctor in charge. Receives: Drug: Voriconazole clinical practice |
What is being measured
The primary outcome is the main question the study is designed to answer.
Serum voriconazole concentration
Serum voriconazole concentration within the therapeutic range, in μg/mL.
Measured: Day 5 of treatment
Secondary outcomes (4)
Therapeutic failure
% of patients with therapeutic failure. A patient has a therapeutic failure if:
- In patient with suspected or confirmed invasive aspergillosis: drug change or association, because of bad clinical or radiological evolution of the disease.
- In patient who receive prophylactic treatment: the necessity of change because of suspected or confirmed invasive fungal disease.
Measured: Within 3 months
Adverse event
% of patients with a dose-dependent drug adverse event reaction.
It will be considered dose-dependent drug adverse reactions:
- Visual disturbances (photopsias)
- Skin reactions
- Neurotoxicity (confusion and visual hallucinations) and
- Corrected QT interval (QTc) lengthening
Measured: Within 3 months
Costs by adverse event
Quantifying economic burden (in euros) associated with management of severe adverse events.
Measured: Day 90 of treatment
Quality adjusted life years (QALY)
Measure of disease burden, including both the quality and the quantity of life lived.
Measured: Day 90 of treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Jan 2020
- Study start2 Jan 2020
- Primary completion18 Jan 2020
- Study completion31 Dec 2022 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Invasive Fungal InfectionsStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 4
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind
- Who is masked
- Participant
- Enrolment
- 146 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Instituto de Investigación Hospital Universitario La Paz
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Alberto M Borobia, MD, PhD, Principal investigator, La Paz University Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04238884
- Sponsor's protocol ID
- 2019-000376-41
- Other IDs
- 2019
- Acronym
- VORIGENPHARM
- Status last verified
- Jan 2020
- First submitted
- 18 Jan 2020
- Last updated
- 23 Jan 2020
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Monserrat-Villatoro J, Bueno D, Estebanez M, Mozo Y, Rosas-Alonso R, Anton C, Otero MJ, Guerra-Garcia P, Diaz-Villamarin X, Arias P, Perez-Martinez A, Abad-Santos F, Carcas AJ, Garcia-Garcia I, Borobia AM. Pre-emptive CYP2C19 genotyping and voriconazole exposure in pediatric hematology patients at risk of aspergillosis: an exploratory randomized pilot study with a cost-consequence analysis. Front Pharmacol. 2026 Jul 21;17:1830548. doi: 10.3389/fphar.2026.1830548. eCollection 2026.
PubMed 42553256Monserrat Villatoro J, Garcia Garcia I, Bueno D, de la Camara R, Estebanez M, Lopez de la Guia A, Abad-Santos F, Anton C, Mejia G, Otero MJ, Ramirez Garcia E, Frias Iniesta J, Carcas A, Borobia AM. Randomised multicentre clinical trial to evaluate voriconazole pre-emptive genotyping strategy in patients with risk of aspergillosis: vorigenipharm study protocol. BMJ Open. 2020 Oct 1;10(10):e037443. doi: 10.1136/bmjopen-2020-037443.
PubMed 33004392
Study at a glance
From the registry record, last updated by the sponsor on 23 Jan 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Randomized and Multicenter Clinical Trial to Evaluate the Effectiveness and Efficiency of a Voriconazole Preemptive Genotyping Strategy in Patients With Risk of Aspergillosis
- ClinicalTrials.gov ID
- NCT04238884
- Acronym / sponsor ID
- VORIGENPHARM / 2019-000376-41
- Conditions
- Invasive Fungal Infections
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 146 participants (estimated)
- Lead sponsor
- Instituto de Investigación Hospital Universitario La PazUniversity, hospital or other
- Study start
- 2 Jan 2020 (actual)
- Primary completion
- 18 Jan 2020 (actual)
- Study completion
- 31 Dec 2022 (estimated)
- First posted
- 23 Jan 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a phase IV pragmatic, multicentre, randomised, simple-blind, parallel arm, centre-stratified clinical trial. The main objective is to compare efficiency of voriconazole preemptive genotyping strategy, compared with routine practice.
Detailed description
Primary outcome is serum level of voriconazole on fifth day. Secondary outcome is a combined variable of therapeutic failure and adverse events, associated with voriconazole. A total of 146 patients with risk of undergoing invasive aspergillosis who potentially will receive voriconazole will be recruited, and CYP2C19 will be genotyped. If the patient receives voriconazole finally, he will be randomized (1:1 experimental/control). In the experimental arm patients receive dose according to pharmacogenetic algorithm including CYP2C19 genotype and clinical and demographic information. In the control arm patients receive dose according to clinical practice guidelines. In addition, a Spain national health system (NHS) point-of-view cost-effectiveness evaluation is going to be done. Direct costs calculation of each arm will be done.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind (participant)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Experimental group | Experimental | Drug: Voriconazole preemptive genotyping strategyBased on the genetic study carried out and the patient's characteristics (age, weight, indication), the Pharmacogenetics Unit of the University Hospital La Paz will indicate the dose to be administered based on the therapeutic individualization protocol guided by pharmacogenetics. |
| Control group | Active comparator | Drug: Voriconazole clinical practiceNo information will be provided and procedure will be carried out according to normal clinical practice, with clinical monitoring by the doctor in charge. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- Patient at risk of developing invasive aspergillosis, that will potentially receive treatment or prophylaxis with voriconazole:
- Those who agree to participate in the study by signing informed consent (patients equal or over 18 years old)
- Subjects under 18 years old whose representative / legal guardian has voluntarily signed the informed consent.
- In the case of mature under 18 years subjects (12-17 years of age), in addition to the consent signed by the legal guardian, the consent of the subject will be obtained.
Key exclusion criteria (2 listed)
- Patients who for any reason should not be included in the study according to the criteria of the research team.
- Subjects who are not capable to understand the information sheet and unable to sign the informed consent.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Voriconazole preemptive genotyping strategyThe patients who finally receives voriconazole will be randomized to receive the dose according to a pharmacogenetic algorithm including CYP2C19 genotype and clinical and demographic information. | Drug | Experimental group |
| Voriconazole clinical practiceThe patients who finally receives voriconazole will be randomized to receive the dose according to clinical practice | Drug | Control group |
Primary outcome
Serum voriconazole concentration
Time frame: Day 5 of treatment
Serum voriconazole concentration within the therapeutic range, in μg/mL.
4 secondary outcomes
Therapeutic failure
Time frame: Within 3 months
Adverse event
Time frame: Within 3 months
Costs by adverse event
Time frame: Day 90 of treatment
Quality adjusted life years (QALY)
Time frame: Day 90 of treatment
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Jan 2020.
- Derived from this study: PubMed 42553256Monserrat-Villatoro J, Bueno D, Estebanez M, Mozo Y, Rosas-Alonso R, Anton C, Otero MJ, Guerra-Garcia P, Diaz-Villamarin X, Arias P, Perez-Martinez A, Abad-Santos F, Carcas AJ, Garcia-Garcia I, Borobia AM. Pre-emptive CYP2C19 genotyping and voriconazole exposure in pediatric hematology patients at risk of aspergillosis: an exploratory randomized pilot study with a cost-consequence analysis. Front Pharmacol. 2026 Jul 21;17:1830548. doi: 10.3389/fphar.2026.1830548. eCollection 2026.
- Derived from this study: PubMed 33004392Monserrat Villatoro J, Garcia Garcia I, Bueno D, de la Camara R, Estebanez M, Lopez de la Guia A, Abad-Santos F, Anton C, Mejia G, Otero MJ, Ramirez Garcia E, Frias Iniesta J, Carcas A, Borobia AM. Randomised multicentre clinical trial to evaluate voriconazole pre-emptive genotyping strategy in patients with risk of aspergillosis: vorigenipharm study protocol. BMJ Open. 2020 Oct 1;10(10):e037443. doi: 10.1136/bmjopen-2020-037443.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Alberto M Borobia, MD, PhD principal investigator
La Paz University Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Voriconazole preemptive genotyping strategy and Voriconazole clinical practice in people with Invasive Fungal Infections, run by Instituto de Investigación Hospital Universitario La Paz at 1 site in Spain.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 23 Jan 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Spain. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Alberto M Borobia, MD, PhD (+34-917277558, alberto.borobia@salud.madrid.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Serum voriconazole concentration (time frame: Day 5 of treatment). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04238884, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Jan 2020; AzTrials last refreshed its copy on 6 Nov 2026.