Understanding resistance to azacitidine-based therapies in acute myeloid leukemia

Dynamics of Resistance Emergence to Azacitidine-based Therapies in Acute Myeloid

Observational Assistance Publique - Hôpitaux de Paris · NCT06225128

This study is trying to understand why some people with acute myeloid leukemia don't respond to the standard treatment with azacitidine and venetoclax, by testing new ways to predict who might have resistance to the drugs.

Quick facts

Study typeObservational
Enrollment120 (estimated)
Ages18 Years to 100 Years
SexAll
SponsorAssistance Publique - Hôpitaux de Paris Academic / other
Drugs / interventionschemotherapy
Locations1 site (Paris)
Trial IDNCT06225128 on ClinicalTrials.gov

What this trial studies

This observational study focuses on acute myeloid leukemia (AML) and aims to investigate the mechanisms behind resistance to the first-line treatment combining azacitidine and venetoclax. It will prospectively validate a drug sensitivity testing assay to predict primary resistance in 100 unfit AML patients. The study will also explore newer assays that better mimic the tumor environment to improve prediction accuracy. By analyzing the response of leukemic and immune cells to treatment, the research seeks to uncover both cell-intrinsic and immune-mediated resistance mechanisms.

Who should consider this trial

Good fit: Ideal candidates are adults aged 18 and older with newly diagnosed acute myeloid leukemia who are planned to receive azacitidine and venetoclax or azacitidine and ivosidenib as frontline therapy.

Not a fit: Patients who have previously received hypomethylating agents for myelodysplastic syndrome may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could lead to improved treatment strategies for patients with acute myeloid leukemia by identifying resistance mechanisms.

How similar studies have performed: Other studies have shown success in understanding resistance mechanisms in AML, but this specific approach is novel.

Eligibility criteria

Show full inclusion / exclusion criteria
Inclusion Criteria:

* be ≥18 years old,
* have a newly diagnosed AML according to ICC 2022 criteria,

  * patients with AML related to prior chemotherapy or radiotherapy for another cancer will be eligible,
  * patients with MDS/AML per ICC 2022 criteria will be eligible,
* have signed the informed consent form of the eTHEMA observatory trial
* have ≥10% blasts on the bone marrow smear at screening,
* have not received any treatment for AML except for hydroxyurea and/or steroids,

  * Patients having previously received hypomethylating agents for an antecedent myelodysplastic syndrome are ineligible,
* be eligible to AZA/VEN or AZA/IVO therapy, due to general health status,
* have an ECOG performance status ≤ 2,
* be planned to receive azacitidine and venetoclax (AZA/VEN) or azacitidine and ivosidenib (AZA/IVO) as frontline therapy,
* weigh ≥ 40 kg (compliance to Loi Jardé for PB sampling),
* have provided written informed consent obtained prior to any screening procedures

Exclusion Criteria:

At screening, patients must NOT:

* have suspected or proven acute promyelocytic leukemia based on morphology, karyotype or molecular assay, including APL with non-PML::RARA rearrangements,
* have suspected or proven AML with t(9;22)(q34.1;q11.2)/BCR::ABL1 based on karyotype or molecular assay,
* have myeloid sarcoma,
* have failed to perform bone marrow aspiration at screening,
* have received previous therapy for AML with any investigational agent or cytotoxic drug, within 28 days before starting treatment. Only hydroxyurea is permitted for the control of blood counts. Aside from hypomethylating agents, other treatments for an antecedent myeloid neoplasm (MDS or MPN) are not considered as exclusion criteria,
* be pregnant or breastfeeding (for women),
* present any of concurrent severe and/or uncontrolled medical condition, which could compromise participation in the study,
* be enrolled in a clinical trial which could compromise participation in the study.

Where this trial is running

Paris

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
Conditions Acute Myeloid Leukemia
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.