AML (Acute Myelogenous LeukemiaVEN-AMLNCT07254312
Correlation of Venetoclax Plasma Concentrations With Toxicity of Hypometilating Agents and Venetoclax Combination for Acute Myeloid Leukemia Patients in Remission.
An observational study of AML (Acute Myelogenous Leukemia, run by Fondazione IRCCS Policlinico San Matteo di Pavia at 1 site in Italy.
Trial summary
Venetoclax (VEN) is a potent and selective oral inhibitor of the BCL-2 gene and has shown anti-leukemic activity when used in combination with hypomethylating agents (HMA) in patients with Acute Myeloid Leukemia (AML), both newly diagnosed and in relapse or refractory (R/R) stages. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- AML (Acute Myelogenous Leukemia
- How many people
- 14 participants
- Where
- 1 siteItaly
- Study dates
- 21 Oct 2024to Apr 2026
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
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
- Child, Adult, Older adult
Study population: Study population: patients with newly diagnosed Acute Myeloid Leukemia treated with HMA-VEN in disease remission (from the first post-remission cycle). Patients will be excluded if they are undergoing treatment with moderate or strong Cytochrome 3A4 inhibitors or inducers, or if they are unwilling to receive treatment as previously specified and to be tested for VEN plasma levels at the previously specified timepoint.
Who may be able to join
- patients with newly diagnosed Acute Myeloid Leukemia treated with HMA-VEN in disease remission (from the first post-remission cycle).
Who cannot join
- undergoing treatment with moderate or strong Cytochrome 3A4 inhibitors or inducers,
- unwilling to receive treatment as previously specified and to be tested for VEN plasma levels at the previously specified timepoint.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Italy1 site, 1 recruiting
- Recruiting
Fondazione IRCCS Policlinico San Matteo, SC Oncologia
Pavia, Pavia, 27100
Patrizia Zappasodi, MD, +390382503070, p.zappasodi@smatteo.pv.it
About this study
Venetoclax (VEN) is a potent and selective oral inhibitor of the BCL-2 gene and has shown anti-leukemic activity when used in combination with hypomethylating agents (HMA) in patients with Acute Myeloid Leukemia (AML), both newly diagnosed and in relapse or refractory (R/R) stages. A daily dose of 400 mg has shown the best results in terms of efficacy, toxicity, and low early mortality rates (DiNardo et al., Blood 2019). The HMA-VEN combination has been approved for the treatment of newly diagnosed AML patients who are not candidates for intensive therapy. However, although this treatment is considered low-intensity, it causes a non-negligible toxicity profile, especially hematological toxicity, even in patients who have already achieved remission. As a result, treatment often needs to be interrupted, and VEN dosage adjusted in subsequent cycles.
An analysis by Pratz et al. (Pratz et al., Am J Hematol 2022) following the publication of the pivotal trial reported grade IV cytopenias lasting at least 7 days in the cycles following remission in 161 (87%) patients in the VEN+Azacitidine arm. Furthermore, plasma concentrations of VEN were analyzed in patients who developed grade IV cytopenias for at least 7 days, and no correlation was found between VEN plasma levels and the number of observed cytopenias.
Read more
In the routine management of these patients, when hematologic toxicity occurs, the approach varies greatly from center to center and is based on the individual experience and assessment of the referring clinician. As a result, there is no standardized approach. Plasma concentrations of VEN are not routinely measured during treatment.
A better understanding of the factors determining the variable toxicity observed in patients in remission could optimize treatment to improve patient tolerability and allow for the regular administration of therapy, which is essential for maintaining leukemia remission status.
- Conditions
- AML (Acute Myelogenous Leukemia
- Medical subject headings
- Leukemia, Myeloid, Acute
What is being measured
The primary outcome is the main question the study is designed to answer.
Analysis of the association between VEN plasma levels and the incidence of grade IV neutropenia.
Venetoclax plasma concentrations are determined by liquid chromatography coupled to mass spectrometry. The method was developed and validated according to the European Medicines Agency (EMA) guidelines. The drug is separated from the matrix by protein precipitation obtained by adding an acidified solution (0.1% HCOOH: 0.1%) of acetonitrile/methanol in a 1:1 ratio (200 µL) to the test samples (50 µL). The deuterated analogue, Venetoclax-D7, was chosen as the internal standard (IS). The analyte and IS are ionized with an ESI (Electrospray Ionization) source in positive mode. The observed mass transitions are m/z 868.1 > 320.7; 635.5 and m/z 875.2 > 320.8; 642.9 for Venetoclax and IS, respectively.
Measured: 18 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Nov 2025
- Study start21 Oct 2024
- Primary completionApr 2026 (estimated)
- Study completionApr 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for AML (Acute Myelogenous LeukemiaStudies of the same condition, open and under way.
- More trials run by Fondazione IRCCS Policlinico San Matteo di PaviaThe sponsor's other registered studies.
- 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
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 14 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Fondazione IRCCS Policlinico San Matteo di Pavia
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Patrizia Zappasodi, Fondazione IRCCS Policlinico San Matteo di Pavia
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07254312
- Sponsor's protocol ID
- VEN-AML
- Acronym
- VEN-AML
- Status last verified
- Mar 2025
- First submitted
- 17 Nov 2025
- Last updated
- 28 Nov 2025
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 28 Nov 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Correlation of Venetoclax Plasma Concentrations With Toxicity of Hypometilating Agents and Venetoclax Combination for Acute Myeloid Leukemia Patients in Remission.
- ClinicalTrials.gov ID
- NCT07254312
- Acronym / sponsor ID
- VEN-AML / VEN-AML
- Conditions
- AML (Acute Myelogenous Leukemia
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 14 participants (estimated)
- Lead sponsor
- Fondazione IRCCS Policlinico San Matteo di PaviaUniversity, hospital or other
- Study start
- 21 Oct 2024 (actual)
- Primary completion
- Apr 2026 (estimated)
- Study completion
- Apr 2026 (estimated)
- First posted
- 28 Nov 2025 (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.
Venetoclax (VEN) is a potent and selective oral inhibitor of the BCL-2 gene and has shown anti-leukemic activity when used in combination with hypomethylating agents (HMA) in patients with Acute Myeloid Leukemia (AML), both newly diagnosed and in relapse or refractory (R/R) stages. A daily dose of 400 mg has shown the best results in terms of efficacy, toxicity, and low early mortality rates (DiNardo et al., Blood 2019). The HMA-VEN combination has been approved for the treatment of newly diagnosed AML patients who are not candidates for intensive therapy. However, although this treatment is considered low-intensity, it causes a non-negligible toxicity profile, especially hematological toxicity, even in patients who have already achieved remission. As a result, treatment often needs to be interrupted, and VEN dosage adjusted in subsequent cycles.
An analysis by Pratz et al. (Pratz et al., Am J Hematol 2022) following the publication of the pivotal trial reported grade IV cytopenias lasting at least 7 days in the cycles following remission in 161 (87%) patients in the VEN+Azacitidine arm. Furthermore, plasma concentrations of VEN were analyzed in patients who developed grade IV cytopenias for at least 7 days, and no correlation was found between VEN plasma levels and the number of observed cytopenias.
In the routine management of these patients, when hematologic toxicity occurs, the approach varies greatly from center to center and is based on the individual experience and assessment of the referring clinician. As a result, there is no standardized approach. Plasma concentrations of VEN are not routinely measured during treatment.
A better understanding of the factors determining the variable toxicity observed in patients in remission could optimize treatment to improve patient tolerability and allow for the regular administration of therapy, which is essential for maintaining leukemia remission status.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Sex
- All
- Age groups
- Child, Adult, Older adult
- Study population
- Study population: patients with newly diagnosed Acute Myeloid Leukemia treated with HMA-VEN in disease remission (from the first post-remission cycle). Patients will be excluded if they are undergoing treatment with moderate or strong Cytochrome 3A4 inhibitors or inducers, or if they are unwilling to receive treatment as previously specified and to be tested for VEN plasma levels at the previously specified timepoint.
Key inclusion criteria (1 listed)
- patients with newly diagnosed Acute Myeloid Leukemia treated with HMA-VEN in disease remission (from the first post-remission cycle).
Key exclusion criteria (2 listed)
- undergoing treatment with moderate or strong Cytochrome 3A4 inhibitors or inducers,
- unwilling to receive treatment as previously specified and to be tested for VEN plasma levels at the previously specified timepoint.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Analysis of the association between VEN plasma levels and the incidence of grade IV neutropenia.
Time frame: 18 months
Venetoclax plasma concentrations are determined by liquid chromatography coupled to mass spectrometry. The method was developed and validated according to the European Medicines Agency (EMA) guidelines. The drug is separated from the matrix by protein precipitation obtained by adding an acidified solution (0.1% HCOOH: 0.1%) of acetonitrile/methanol in a 1:1 ratio (200 µL) to the test samples (50 µL). The deuterated analogue, Venetoclax-D7, was chosen as the internal standard (IS). The analyte and IS are ionized with an ESI (Electrospray Ionization) source in positive mode. The observed mass transitions are m/z 868.1 > 320.7; 635.5 and m/z 875.2 > 320.8; 642.9 for Venetoclax and IS, respectively.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Nov 2025.
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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of AML (Acute Myelogenous Leukemia, run by Fondazione IRCCS Policlinico San Matteo di Pavia at 1 site in Italy.
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 1 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: looking for participants now, as of the sponsor's last update on 28 Nov 2025. 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 Italy. 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.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Patrizia Zappasodi (+390382503070, p.zappasodi@smatteo.pv.it), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Analysis of the association between VEN plasma levels and the incidence of grade IV neutropenia. (time frame: 18 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 28 Nov 2025.