How venetoclax blood levels relate to treatment response in acute myeloid leukemia
Prospective, Multicenter, Clinical-biological Cohort Study to Assess Pharmacokinetics and Pharmacodynamics (PK-PD) of Venetoclax (VEN) in Patients With Acute Myeloid Leukemia (AML)
This project will test how blood levels of venetoclax relate to treatment effects in adults with AML who are starting venetoclax plus azacitidine.
Quick facts
| Phase | Not applicable |
|---|---|
| Study type | Interventional |
| Enrollment | 100 (estimated) |
| Ages | 18 Years and up |
| Sex | All |
| Sponsor | Centre Leon Berard Academic / other |
| Drugs / interventions | chemotherapy |
| Locations | 5 sites (Bourgoin, France and 4 other locations) |
| Trial ID | NCT07243483 on ClinicalTrials.gov |
What this trial studies
This is a prospective, multicenter clinical-biological cohort that will follow 100 adult patients with AML over a 12-month inclusion period while they receive standard venetoclax‑azacitidine therapy. The study will collect serial blood samples (up to 21 additional samplings, maximum 12 mL per sampling day) to measure venetoclax pharmacokinetics and link those levels to pharmacodynamic measures and cytologic response. Therapeutic management is not altered by participation and the protocol excludes patients previously treated with venetoclax or azacitidine, those eligible for intensive chemotherapy, and patients with acute promyelocytic leukemia. The design focuses on real-world treatment settings at several French centers to clarify how drug levels and interactions (for example with azole antifungals) affect outcomes and safety.
Who should consider this trial
Good fit: Adults (≥18) with histologically confirmed AML who are about to start first-line venetoclax plus azacitidine, not previously treated with venetoclax or azacitidine, and not eligible for intensive chemotherapy.
Not a fit: Patients with acute promyelocytic leukemia, those eligible for intensive chemotherapy, those previously treated with venetoclax or azacitidine, or those unable/unwilling to undergo the required blood sampling are unlikely to benefit from joining this cohort.
Why it matters
Potential benefit: If successful, the findings could help personalize venetoclax dosing to improve effectiveness and reduce side effects.
How similar studies have performed: Venetoclax combined with hypomethylating agents has shown clinical benefit in AML and known drug‑drug interactions (notably with azole antifungals) exist, but dedicated prospective PK‑PD cohort data are relatively limited.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Male or female patient aged of at least 18 years on day of signing informed consent * Patient with histologically-confirmed diagnosis of Acute Myeloblactic Leukaemia according to classification ELN 2022 (European Leukemia Net 2022) * Patient who has to initiate treatment venetoclax-azacitidine as first line. Note : triple associations with targeted therapy are not authorized * Patient should understand, sign, and date the written voluntary informed consent form prior to any protocol-specific procedures performed. * Patient must be affiliated or benificiary of a social security system. Exclusion Criteria: * Patient with acute promyelocytic leukemia (APL, AML3) * Patient with AML eligible to intensive chemotherapy * Patient previously treaed with venetoclax and/or azacitidine * Patient participating to another clinical trial with a medicinal product * Any condition that contraindicates blood sampling procedures required by the protocol * Any psychological, family, geographical, or social situation that, according to investigator's judgment, could potentially prevent the signing of an informed consent form and/or woulld likely interfere compliance with study procedures. * Patient under curatorship, guardianship or judicial protection * Pregnant or breast-feeding female patient.
Where this trial is running
Bourgoin, France and 4 other locations
- Centre Hospitalier Pierre Oudot — Bourgoin, France, France (Recruiting)
- CHU de Grenoble — Grenoble, France (Recruiting)
- Centre Leon Berard — Lyon, France (Recruiting)
- CHU de Saint-Étienne — Saint-Etienne, France (Recruiting)
- Hôpitaux Nord-Ouest - Villefranche-sur-Saône — Villefranche-sur-Saône, France (Recruiting)
Study contacts
- Study coordinator: Michaël Philippe
- Email: michael.philippe@lyon.unicancer.fr
- Phone: +33478782666
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.