Acute Myeloid Leukemia (AML)ND-AMLNCT07670130
Venetoclax TDM in Newly Diagnosed AML: Exposure-Response and Prognosis
An observational study of Acute Myeloid Leukemia (AML), run by The First Affiliated Hospital of Soochow University at 1 site in China.
Trial summary
Venetoclax combined with azacitidine (VEN-AZA) is the current first-line standard of care for newly diagnosed acute myeloid leukemia (AML) patients unfit for intensive chemotherapy. Read more about this study
- Who is it for?
- Ages 16 years and older
- Conditions the study is for
- Acute Myeloid Leukemia (AML)
- How many people
- 50 participants
- Where
- 1 siteChina
- Study dates
- 1 Jun 2026to 1 Jun 2027
- 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 16 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: This prospective study included newly diagnosed AML patients who received the venetoclax plus azacitidine as induction. Venetoclax concentration data were collected on days 8, 15, and 22, and the steady-state trough concentration (Cmin), peak concentration (Cmax), and area under the curve (AUC) were calculated. The relationships between venetoclax exposure levels (Cmax, Cmin, and AUC) and efficacy, safety, survival, and individual patient characteristics were analyzed.
Who may be able to join
- Diagnosis: Newly diagnosed acute myeloid leukemia (AML) confirmed according to the WHO 2022 or International Consensus Classification (ICC) criteria, based on bone marrow morphology, flow cytometry, and molecular genetics. Acute promyelocytic leukemia (APL) is excluded.
- Treatment regimen: Planned or already initiated first-line therapy with venetoclax plus azacitidine (VEN-AZA), with dosing determined by the treating physician according to routine clinical practice (no protocol-mandated dose restrictions).
- Age: ≥ 16 years.
- Informed consent: Willingness and ability to provide written informed consent for participation in this observational study.
- Follow-up: Agreement to attend scheduled follow-up visits and to permit clinical data collection at the time points specified in the study protocol.
Who cannot join
- Prior AML therapy: Prior treatment for AML, with the exception of leukapheresis, hydroxyurea, low-dose cytarabine, or corticosteroids.
- Concurrent interventional trials: Current participation in any interventional clinical trial, including those involving investigational agents.
- Extremely short life expectancy: Judged by the investigator to be unable to complete at least one full cycle of therapy and the associated follow-up.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
The first Affiliated Hospital of Soochow University
Suzhou, 21500
Jia Chen, 86+052167976801, drchenjia@163.com
About this study
Official title: Exposure-Response and Prognostic Analysis of Venetoclax Therapeutic Drug Monitoring in Newly Diagnosed AML
Venetoclax combined with azacitidine (VEN-AZA) is the current first-line standard of care for newly diagnosed acute myeloid leukemia (AML) patients unfit for intensive chemotherapy. Although this regimen substantially improves remission rates, marked inter-individual variability is observed in clinical practice-ranging from severe myelosuppression or tumor lysis syndrome in some patients to poor response or early relapse in others. Venetoclax is primarily metabolized by CYP3A4, and its systemic exposure is modulated by multiple factors, including hepatic and renal function, concomitant medications (particularly azole antifungals), and UGT1A1 polymorphisms, leading to a 50%-70% inter-individual variability in blood drug concentrations.
Despite this variability, the current VEN-AZA regimen employs a fixed-dose strategy (400 mg/day) without incorporating therapeutic drug monitoring (TDM) to guide individual dosing. Critical knowledge gaps remain: (1) whether a clear exposure-response relationship exists between venetoclax exposure and composite remission rate (CR+CRi); (2) what blood concentration range optimizes efficacy while minimizing toxicity; (3) which covariates significantly influence venetoclax clearance; and (4) whether early concentration sampling can reliably predict subsequent exposure and clinical outcomes.*
To address these questions, investigators designed a prospective study enrolling newly diagnosed AML patients receiving VEN-AZA therapy. Investigators aim to systematically characterize the exposure-response relationship, establish an optimal therapeutic concentration window, identify key covariates contributing to inter-individual pharmacokinetic variability, and evaluate early-sampling prediction strategies. The findings are expected to provide direct evidence for TDM-guided individualized dosing and to support a paradigm shift from a "fixed-dose" to a "concentration-guided" approach in precision AML therapy.
- Conditions
- Acute Myeloid Leukemia (AML)
- Medical subject headings
- Leukemia, Myeloid, Acute
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Venetoclax combined with Azacitidine | ND-AML patients received the venetoclax plus azacitidine as induction. Dosage is determined by clinicians based on routine practice (not mandatory in the study protocol). Venetoclax concentration data were collected on days 8, 15, and 22, and the steady-state trough concentration (Cmin), peak concentration (Cmax), and area under the curve (AUC) were calculated. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Complete remission rate
Percentage of subjects with complete remission (CR) and incomplete hematologic recovery (CRi)
Measured: At the end of induction treatment (28 days ± 7days)
Secondary outcomes (5)
Hematological toxicity
Number of subjects with hematological adverse events
Measured: Start of treatment to 2 weeks after end of treatment
Non-hematological toxicity
Number of subjects with non-hematological adverse events
Measured: Start of treatment to 2 weeks after end of treatment
Overall Survival
From start of induction treatment to time of death due to any cause, or until last follow-up
Measured: 24 months
Event-free Survival
From start of induction treatment to time of disease relapse/progression or death due to any cause, whichever occurs earlier; or until last follow-up
Measured: 24 months
Measurable residual disease response rate
Percentage of subjects with MRD negative
Measured: At the end of induction treatment (28 days ± 7days)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Jun 2026
- Study start1 Jun 2026
- Primary completion1 Jun 2027 (estimated)
- Study completion1 Jun 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acute Myeloid Leukemia (AML)Studies of the same condition, open and under way.
- More trials run by The First Affiliated Hospital of Soochow UniversityThe 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
- 50 participants (estimated)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, CHEN Jia, The First Affiliated Hospital of Soochow University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07670130
- Sponsor's protocol ID
- SZ3203
- Acronym
- ND-AML
- Status last verified
- Jun 2026
- First submitted
- 19 Jun 2026
- Last updated
- 6 Aug 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 6 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Exposure-Response and Prognostic Analysis of Venetoclax Therapeutic Drug Monitoring in Newly Diagnosed AML
- ClinicalTrials.gov ID
- NCT07670130
- Acronym / sponsor ID
- ND-AML / SZ3203
- Conditions
- Acute Myeloid Leukemia (AML)
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 50 participants (estimated)
- Lead sponsor
- The First Affiliated Hospital of Soochow UniversityUniversity, hospital or other
- Study start
- 1 Jun 2026 (actual)
- Primary completion
- 1 Jun 2027 (estimated)
- Study completion
- 1 Jun 2027 (estimated)
- First posted
- 26 Jun 2026 (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 combined with azacitidine (VEN-AZA) is the current first-line standard of care for newly diagnosed acute myeloid leukemia (AML) patients unfit for intensive chemotherapy. Although this regimen substantially improves remission rates, marked inter-individual variability is observed in clinical practice-ranging from severe myelosuppression or tumor lysis syndrome in some patients to poor response or early relapse in others. Venetoclax is primarily metabolized by CYP3A4, and its systemic exposure is modulated by multiple factors, including hepatic and renal function, concomitant medications (particularly azole antifungals), and UGT1A1 polymorphisms, leading to a 50%-70% inter-individual variability in blood drug concentrations.
Despite this variability, the current VEN-AZA regimen employs a fixed-dose strategy (400 mg/day) without incorporating therapeutic drug monitoring (TDM) to guide individual dosing. Critical knowledge gaps remain: (1) whether a clear exposure-response relationship exists between venetoclax exposure and composite remission rate (CR+CRi); (2) what blood concentration range optimizes efficacy while minimizing toxicity; (3) which covariates significantly influence venetoclax clearance; and (4) whether early concentration sampling can reliably predict subsequent exposure and clinical outcomes.*
To address these questions, investigators designed a prospective study enrolling newly diagnosed AML patients receiving VEN-AZA therapy. Investigators aim to systematically characterize the exposure-response relationship, establish an optimal therapeutic concentration window, identify key covariates contributing to inter-individual pharmacokinetic variability, and evaluate early-sampling prediction strategies. The findings are expected to provide direct evidence for TDM-guided individualized dosing and to support a paradigm shift from a "fixed-dose" to a "concentration-guided" approach in precision AML therapy.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Venetoclax combined with Azacitidine | – | –ND-AML patients received the venetoclax plus azacitidine as induction. Dosage is determined by clinicians based on routine practice (not mandatory in the study protocol). Venetoclax concentration data were collected on days 8, 15, and 22, and the steady-state trough concentration (Cmin), peak concentration (Cmax), and area under the curve (AUC) were calculated. |
Who may qualify
- Ages
- 16 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- This prospective study included newly diagnosed AML patients who received the venetoclax plus azacitidine as induction. Venetoclax concentration data were collected on days 8, 15, and 22, and the steady-state trough concentration (Cmin), peak concentration (Cmax), and area under the curve (AUC) were calculated. The relationships between venetoclax exposure levels (Cmax, Cmin, and AUC) and efficacy, safety, survival, and individual patient characteristics were analyzed.
Key inclusion criteria (5 listed)
- Diagnosis: Newly diagnosed acute myeloid leukemia (AML) confirmed according to the WHO 2022 or International Consensus Classification (ICC) criteria, based on bone marrow morphology, flow cytometry, and molecular genetics. Acute promyelocytic leukemia (APL) is excluded.
- Treatment regimen: Planned or already initiated first-line therapy with venetoclax plus azacitidine (VEN-AZA), with dosing determined by the treating physician according to routine clinical practice (no protocol-mandated dose restrictions).
- Age: ≥ 16 years.
- Informed consent: Willingness and ability to provide written informed consent for participation in this observational study.
- Follow-up: Agreement to attend scheduled follow-up visits and to permit clinical data collection at the time points specified in the study protocol.
Key exclusion criteria (3 listed)
- Prior AML therapy: Prior treatment for AML, with the exception of leukapheresis, hydroxyurea, low-dose cytarabine, or corticosteroids.
- Concurrent interventional trials: Current participation in any interventional clinical trial, including those involving investigational agents.
- Extremely short life expectancy: Judged by the investigator to be unable to complete at least one full cycle of therapy and the associated follow-up.
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
Complete remission rate
Time frame: At the end of induction treatment (28 days ± 7days)
Percentage of subjects with complete remission (CR) and incomplete hematologic recovery (CRi)
5 secondary outcomes
Hematological toxicity
Time frame: Start of treatment to 2 weeks after end of treatment
Non-hematological toxicity
Time frame: Start of treatment to 2 weeks after end of treatment
Overall Survival
Time frame: 24 months
Event-free Survival
Time frame: 24 months
Measurable residual disease response rate
Time frame: At the end of induction treatment (28 days ± 7days)
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 6 Aug 2026.
- 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.
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 Acute Myeloid Leukemia (AML), run by The First Affiliated Hospital of Soochow University at 1 site in China.
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 5 inclusion and 3 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 6 Aug 2026. 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 China. 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 Jia Chen (86+052167976801, drchenjia@163.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Complete remission rate (time frame: At the end of induction treatment (28 days ± 7days)). 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 6 Aug 2026.