Genetic factors affecting treatment response in acute myeloid leukemia

A Prospective Pilot Study of the Genetic Determinants of Toxicity and Response to Azacitidine and Venetoclax in Patients With Newly Diagnosed Acute Myeloid Leukemia Through Evaluation of Polymorphisms in Pharmacokinetic Genes and Venetoclax Levels

Observational Wake Forest University Health Sciences · NCT06580106

This study is testing how certain genetic differences affect the side effects and success of a treatment for people newly diagnosed with acute myeloid leukemia who are receiving azacitidine and venetoclax.

Quick facts

Study typeObservational
Enrollment50 (estimated)
Ages18 Years and up
SexAll
SponsorWake Forest University Health Sciences Academic / other
Locations2 sites (Charlotte, North Carolina and 1 other locations)
Trial IDNCT06580106 on ClinicalTrials.gov

What this trial studies

This research investigates how specific genetic variations influence the side effects and treatment outcomes for patients with newly diagnosed acute myeloid leukemia (AML) receiving azacitidine and venetoclax. It is a prospective pilot study that will collect buccal swabs for genetic analysis and measure venetoclax levels during treatment. The study aims to identify associations between genetic markers and the toxicity or efficacy of the treatment regimen. Participants will be recruited from designated cancer centers in North Carolina.

Who should consider this trial

Good fit: Ideal candidates are adults over 18 years old with a confirmed diagnosis of AML who are starting treatment with azacitidine and venetoclax.

Not a fit: Patients who are not newly diagnosed with AML or those who are fit for intensive induction therapy may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could lead to personalized treatment strategies that minimize side effects and improve outcomes for AML patients.

How similar studies have performed: While this study explores genetic determinants in AML treatment, similar approaches in pharmacogenomics have shown promise in other cancer treatments, indicating potential for success.

Eligibility criteria

Show full inclusion / exclusion criteria
Inclusion Criteria:

* Written informed consent and HIPAA authorization for release of personal health information.
* Age ≥ 18 years of age at the time of enrollment
* Confirmed diagnosis of AML
* Planned initial treatment with azacitidine and venetoclax
* Ability to read and understand the English and/or Spanish language
* As determined by the enrolling investigator, ability of the participant to understand and comply with study procedures for the entire length of the study

Exclusion Criteria:

* None

Where this trial is running

Charlotte, North Carolina and 1 other locations

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 Leukemia, Myeloid, Acute
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.