RecruitingObservational

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

NCT ID: NCT06580106Sponsor: Wake Forest University Health SciencesLast updated: 2026-02-02

Summary

The purpose of this research is to see how certain genetic variations relate to side effects and outcomes experienced while receiving treatment with azacitidine and venetoclax.

Detailed description

This is a prospective pilot study of the association of SNPs and venetoclax levels with toxicity and response to azacitidine plus venetoclax (Aza/Ven) as well as pharmacogenomics and venetoclax levels in patients with newly diagnosed AML determined to be unfit for intensive induction. Newly diagnosed AML patients over 18 years old who receive Aza/Ven as standard of care will be eligible for this study. Buccal swabs for SNPs and pharmacogenomic analysis can occur at any point before or after starting treatment during the study period. Venetoclax peak and trough levels will be obtained during SOC Aza/Ven treatments. Participants will be recruited initially at AHWFBCCC locations.

Arms & interventions

OtherBiospecimen samples
Buccal swabs and Blood samples will be collected throughout study.

Outcome measures

Primary

Toxicity side effect
Defined as a binary variable indicating whether a participant experienced a Grade 3 or higher of specific side effects (including infections, anemia, thrombocytopenia, febrile neutropenia, neutropenia, nausea, diarrhea)
Time frame: From initiation of venetoclax through 30 days after last dose

Secondary

Dose Modification
Time frame: Approximately 6 months or until last dose of Venetoclax, whichever came first
Disease Response
Time frame: Up to 3 years
Venetoclax levels
Time frame: Approx 6 months
Overall survival
Time frame: Approx 3 years
Dose modification due to nausea or diarrhea
Time frame: Approximately 6 months or until last dose of Venetoclax, whichever came first
Metabolizer status checklist
Time frame: Approximately 6 months or until last dose of Venetoclax, whichever came first

Eligibility criteria

Sex: AllAge: 18 Years and olderHealthy volunteers: No

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

Study locations (2)

Levine Cancer Institute

Charlotte, North Carolina, 28204

Recruiting

Courtney Schepel · Contact

(980) 292-0817 courtney.schepel@advocatehealth.org

Brittany Ragon, MD · Principal Investigator

Wake Forest Baptist Comprehensive Cancer Center

Winston-Salem, North Carolina, 27157

Recruiting

Timothy Pardee, MD · Contact

336-716-2466 tspardee@wakehealth.edu

Timothy Pardee, MD · Principal Investigator