Leukemia, Myeloid, AcuteNCT06580106
Toxicity Genetic Determinants and Response to Azacitidine and Venetoclax in AML
An observational study of Leukemia, Myeloid, Acute, run by Wake Forest University Health Sciences at 2 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Leukemia, Myeloid, Acute
- What is being tested?
- Biospecimen samples
- How many people
- 50 participants
- Where
- 2 sitesUnited States
- Study dates
- 9 Apr 2025to Jan 2030
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Participants diagnosed with AML who are receiving or are planned to receive azacitidine plus venetoclax will be identified in clinic and presented informed consent for this study.
Who may be able to join
- 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
Who cannot join
- None
Trial locations and contacts
2 sites in 1 country. Contact the study team or a site near you to ask about taking part.
United States2 sites, 2 recruiting
- Recruiting
Levine Cancer Institute
Charlotte, North Carolina, 28204
Courtney Schepel, (980) 292-0817, courtney.schepel@advocatehealth.org
Brittany Ragon, MD
- Recruiting
Wake Forest Baptist Comprehensive Cancer Center
Winston-Salem, North Carolina, 27157
Timothy Pardee, MD, 336-716-2466, tspardee@wakehealth.edu
Timothy Pardee, MD
About this study
Official 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
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.
Read the 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.
- Conditions
- Leukemia, Myeloid, Acute
- Being tested
- Biospecimen samples
- Medical subject headings
- Leukemia, Myeloid, Acute
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Biospecimen samplesOther
Buccal swabs and Blood samples will be collected throughout study.
| Study group | Role | What they receive |
|---|---|---|
| AML participants who are receiving or are planned to receive azacitidine plus venetoclax | 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. CYP3A activity will also be evaluated. Demographic and cancer related history will be acquired for each participant. During study participation, cancer treatment details including administration, dose modifications, delays, and reductions, including specific grade 3 toxicities, stem cell transplant status, symptom burden, disease response, and survival will be collected. Participants will be taken off study after three years. Receives: Other: Biospecimen samples |
What is being measured
The primary outcome is the main question the study is designed to answer.
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)
Measured: From initiation of venetoclax through 30 days after last dose
Secondary outcomes (6)
Dose Modification
A binary variable indicating whether a participant experienced a dose modification, including delay or reduction
Measured: Approximately 6 months or until last dose of Venetoclax, whichever came first
Disease Response
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
Measured: Up to 3 years
Venetoclax levels
Will be defined as the maximum concentration of Venetoclax and is measured at 6 months if the participant is still receiving venetoclax
Measured: Approx 6 months
Overall survival
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
Measured: Approx 3 years
Dose modification due to nausea or diarrhea
A binary variable indicating whether a participant experienced a dose modification, including delay or reduction due to nausea or diarrhea
Measured: Approximately 6 months or until last dose of Venetoclax, whichever came first
Metabolizer status checklist
A categorical variable indicating whether a participant is a high, normal or low metabolizer based on pharmacogenomics analysis of SNP data
Measured: Approximately 6 months or until last dose of Venetoclax, whichever came first
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Aug 2024
- Study start9 Apr 2025
- Primary completionJul 2027 (estimated)
- Study completionJan 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Leukemia, Myeloid, AcuteStudies of the same condition, open and under way.
- More trials run by Wake Forest University Health SciencesThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case only
- Time perspective
- Prospective
- Enrolment
- 50 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Wake Forest University Health Sciences
- Sponsor type
- University, hospital or other
- Collaborators
- Atrium Health Levine Cancer Institute, Swim Across America
- Responsible party
- Sponsor
- Investigators
- Brittany Ragon, MD, Principal investigator, Atrium Health Wake Forest Baptist Comprehensive Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06580106
- Sponsor's protocol ID
- IRB00116209
- Other IDs
- LCI-LEU-AML-VENTOX-001
- Status last verified
- Jul 2026
- First submitted
- 14 Aug 2024
- Last updated
- 24 Jul 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 24 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official 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
- ClinicalTrials.gov ID
- NCT06580106
- Acronym / sponsor ID
- IRB00116209
- Conditions
- Leukemia, Myeloid, Acute
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 50 participants (estimated)
- Lead sponsor
- Wake Forest University Health SciencesUniversity, hospital or other
- Collaborators
- Atrium Health Levine Cancer Institute, Swim Across America
- Study start
- 9 Apr 2025 (actual)
- Primary completion
- Jul 2027 (estimated)
- Study completion
- Jan 2030 (estimated)
- First posted
- 30 Aug 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Case only
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| AML participants who are receiving or are planned to receive azacitidine plus venetoclax | – | Other: Biospecimen samplesBuccal 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. CYP3A activity will also be evaluated. Demographic and cancer related history will be acquired for each participant. During study participation, cancer treatment details including administration, dose modifications, delays, and reductions, including specific grade 3 toxicities, stem cell transplant status, symptom burden, disease response, and survival will be collected. Participants will be taken off study after three years. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Participants diagnosed with AML who are receiving or are planned to receive azacitidine plus venetoclax will be identified in clinic and presented informed consent for this study.
Key inclusion criteria (6 listed)
- 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
Key exclusion criteria (1 listed)
- None
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Biospecimen samplesBuccal swabs and Blood samples will be collected throughout study. | Other | AML participants who are receiving or are planned to receive azacitidine plus venetoclax |
Primary outcome
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)
6 secondary outcomes
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
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Jul 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Brittany Ragon, MD principal investigator
Atrium Health Wake Forest Baptist Comprehensive Cancer Center
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Leukemia, Myeloid, Acute, run by Wake Forest University Health Sciences at 2 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 24 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Courtney Schepel ((980) 292-0817, courtney.schepel@advocatehealth.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Toxicity side effect (time frame: From initiation of venetoclax through 30 days after last dose). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Jul 2026.