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NCT Number: NCT06580106

Toxicity Genetic Determinants and Response to Azacitidine and Venetoclax in AML

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.

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Key information

Conditions

Leukemia, Myeloid, Acute Hematologic Diseases Hemic and Lymphatic Diseases Leukemia Leukemia, Myeloid Neoplasms Neoplasms by Histologic Type

Age range

18 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

Levine Cancer Institute, Charlotte, North Carolina, United States

Loading trial locations.

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Written informed consent and HIPAA authorization for release of personal health information.
  • Age ≥ 18 years of age at the time of consent
  • 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

Treatment and study plan

Biospecimen samples

Other

Buccal swabs and Blood samples will be collected throughout study.

Primary outcomes

  1. Toxicity side effect

    Time frame: From initiation of venetoclax through 30 days after last dose

    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)

Secondary outcomes

  1. Dose Modification

    Time frame: Approximately 6 months or until last dose of Venetoclax, whichever came first

    A binary variable indicating whether a participant experienced a dose modification, including delay or reduction

  2. Disease Response

    Time frame: Up to 3 years

    For participants diagnosed with AML, response will be defined as a binary variable indicating if they had a Complete Remission (CR), Complete Remission with partial hematologic recovery (CRh), Complete Remission with incomplete hematologic recovery (CRi), Morphologic Leukemia-free state (MLFS), or partial response (PR) to induction therapy using the European LeukemiaNet (ELN 2022) criteria. Otherwise they will be considered a non-responder

  3. Venetoclax levels

    Time frame: Approx 6 months

    Will be defined as the maximum concentration of Venetoclax and is measured at 6 months if the participant is still receiving venetoclax

  4. Overall survival

    Time frame: Approx 3 years

    Duration of time from date of enrollment to death. Participants who are alive or lost to follow-up at the time of the analysis will be censored at the last known date they were alive

  5. Dose modification due to nausea or diarrhea

    Time frame: Approximately 6 months or until last dose of Venetoclax, whichever came first

    A binary variable indicating whether a participant experienced a dose modification, including delay or reduction due to nausea or diarrhea

  6. Metabolizer status checklist

    Time frame: Approximately 6 months or until last dose of Venetoclax, whichever came first

    A categorical variable indicating whether a participant is a high, normal or low metabolizer based on pharmacogenomics analysis of SNP data

Study contacts

Contact information is provided by the study sponsor or research team.

Courtney Schepel

CONTACT

[email protected]

(980) 292-0817

Sponsors and collaborators

Lead sponsor

Wake Forest University Health Sciences

Other

Collaborators

  • Atrium Health Levine Cancer Institute
  • Swim Across America

Registry information

Official study title

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

Important dates

Study start
2025
Primary completion
2027
Study completion
2030
First posted
Aug 30, 2024
Registry last updated
Jul 24, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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