NCT07670130Recruiting
Venetoclax TDM in Newly Diagnosed AML: Exposure-Response and Prognosis
Exposure-Response and Prognostic Analysis of Venetoclax Therapeutic Drug Monitoring in Newly Diagnosed AML
Source: ClinicalTrials.gov · Last updated 6 Aug 2026
Brief summary (registry, in English)
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.
- Status (registry)
- Recruiting
- Study type
- Observational
- Enrollment
- 50 (estimated)
- First submitted
- 19 Jun 2026
- Start date
- 1 Jun 2026
- Primary completion date
- 1 Jun 2027
- Lead sponsor
- The First Affiliated Hospital of Soochow University
- Diseases
- Acute myeloid leukemia, Childhood cancers
- NCT number
- NCT07670130
What you commit to
- Study type: Observational
- Planned participants: 50
- From 1 Jun 2026 to the primary completion date, 1 Jun 2027
- 1 site in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- 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.
Who the study is looking for
- 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.
Age in the registry: 16 years to - · Sex: All · Does not accept healthy volunteers
Sites (1)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| The First Affiliated Hospital of Soochow University Coordinator of this site? Confirm its status | Suzhou | China | Recruiting |
This page is information, not medical advice. Discuss any trial with your doctor.