Acute Myeloid LeukemiaPhase 1NCT06158100
Venetoclax in Combination With Azacitidine (VEN/AZA) Followed by Donor Lymphocyte Infusion (DLI) for Patients With Very High-Risk Acute Myeloid Leukemia (AML) Undergoing Allogeneic Hematopoietic Cell Transplant (HCT)
A Phase 1 study testing Venetoclax, Azacitidine and Donor Lymphocyte Infusion in people with Acute Myeloid Leukemia, run by Antonio M Jimenez Jimenez at 1 site in United States.
Trial summary
The purpose of this study is to see the effects of an investigational combination treatment of venetoclax, azacitidine, and donor lymphocyte infusion (DLI) in patients with high-risk AML receiving allogeneic hematopoietic cell transplantation, and to assess if the combination treatment is well tolerated and prevents… Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Acute Myeloid Leukemia
- What is being tested?
- Venetoclax, Azacitidine and Donor Lymphocyte Infusion
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 2 participants
- Where
- 1 siteUnited States
- Study dates
- 4 Dec 2024to 2 Jun 2026
- Phase
- Phase 1
- Are there results?
- None posted in the registry
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 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Male and female patients between the ages of 18-75.
- Patients with a histologic diagnosis of AML in morphological remission (<5% bone marrow (BM) blasts) prior to allogeneic hematopoietic cell transplantation and very high-risk for relapse defined as: (i) Presence of measurable residual disease (MRD) by multicolor flow cytometry (MFC) prior to transplant and receiving a reduced intensity conditioning (RIC) or nonmyeloablative (NMA) regimen (ii) Presence of MRD by MFC at day +30 post-transplant (iii) All patients with monosomal karyotype (MK) and those with 17p/tumor protein p53 (TP53) mutated disease irrespective of MRD status and intensity of conditioning regimen.
- Adequate hematopoietic recovery after HCT, defined as:
- Absolute neutrophil count (ANC) >= 1 x 10^9/L without daily use of myeloid growth factors
- Platelet count >= 50 x 10^9/L without platelet transfusion within 1 week
- Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1, or 2
Show all 12Show fewer
- Serum creatinine =< 1.5 mg/dL or creatinine clearance greater or equal than 40 cc/min
- Serum bilirubin =< 1.5 x upper limit of normal (ULN)
- Aspartate transaminase (AST) or alanine transaminase (ALT) =< 2.5 x ULN
- Alkaline phosphatase =< 2.5 x UL
- Negative serum or urine pregnancy test for women with reproductive potential.
- A negative donor-specific antibody (DSA) assay (i.e., Micro-Flow Imaging (MFI) <m3000) for recipients of any mismatched graft (including haploidentical) HCT.
Who cannot join
- Active disease (>5% blasts or any evidence of extra-medullary disease) at the time of transplantation or at day +30
- Active acute graft-versus-host disease (aGVHD) requiring systemic IST or history of aGVHD grade III or higher.
- Active chronic GVHD requiring systemic immunosuppressive therapy (IST).
- Active uncontrolled systemic fungal, bacterial, or viral infection
- Known active viral infection with human immunodeficiency virus (HIV), hepatitis B virus (HBV), or hepatitis C virus (HCV)
- Significant active cardiac disease within the previous 6 months, including: New York Heart Association (NYHA) class III or IV congestive heart failure. Unstable angina, angina requiring surgical or medical intervention, and/or myocardial infarction.
- History of any other malignancy within 2 years prior to study entry, except for: adequately treated in situ carcinoma of the cervix or carcinoma in situ of breast; basal cell carcinoma of the skin or localized squamous cell carcinoma of the skin; previous malignancy confined and surgically resected (or treated with other modalities) with curative intent; myelodysplastic syndrome.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Phase 1 Study of Venetoclax in Combination With Azacitidine (VEN/AZA) Followed by Donor Lymphocyte Infusion (DLI) for Patients With Very High-Risk Acute Myeloid Leukemia (AML) Undergoing Allogeneic Hematopoietic Cell Transplant (HCT)
The purpose of this study is to see the effects of an investigational combination treatment of venetoclax, azacitidine, and donor lymphocyte infusion (DLI) in patients with high-risk AML receiving allogeneic hematopoietic cell transplantation, and to assess if the combination treatment is well tolerated and prevents disease relapse after transplant.
- Conditions
- Acute Myeloid Leukemia
- Being tested
- Venetoclax, Azacitidine, Donor Lymphocyte Infusion
- Keywords
- Hematopoietic Cell Transplant (HCT)
- 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.
VenetoclaxDrug
Also called Venclexta, Venclyxto
Venetoclax will be given orally (PO) at the assigned doses of 50 or 100 mg once daily for 7, 14 or 21 days starting at day 1 of each 28-day cycle, for up to 12 cycles.
AzacitidineDrug
Also called Vidaza, Azadine
Azacitidine will be administered at a dose of 20mg/m^2 once daily subcutaneously (SC) or intravenously (IV) for five days (Days 1-5) of each 28-day cycle.
Donor Lymphocyte InfusionBiological
Donor lymphocyte infusions (DLI) may be administered at the discretion of the treating physician, if certain criteria are met. Participants may receive up to three (3) infusions of donor lymphocytes (a type of white blood cell) at the following dose levels and study timepoints:
- DLI 1: 1x10^6 cluster of differentiation 3+ (CD3+) cells/kg, after cycle 3
- DLI 2: 5x10^6 CD3+ cells/kg, after cycle 5, if there is persistent MRD
- DLI 3: 1x10^7 CD3+ cells/kg, after cycle 7, if there is persistent MRD.
| Study group | Role | What they receive |
|---|---|---|
| VEN/AZA Dose Escalation/De-Escalation Cohort | Experimental | Participants in this group will begin Venetoclax and Azacitidine (VEN/AZA) combination therapy between day +42 and day +100 following hematopoietic cell transplant (HCT) infusion. VEN/AZA combination therapy will be administered for up to six (6) cycles, followed by up to six (6) additional cycles of Venetoclax monotherapy in the absence of disease progression or unacceptable toxicity. Each cycle is 28 days.
Participants may also receive donor lymphocyte infusions (DLI) at the discretion of the treating physician, if certain criteria are met.
Participants will receive up to one year (12 cycles) of study therapy, followed by up to one year of follow-up. Total participation duration is up to two years. Receives: Drug: Venetoclax; Drug: Azacitidine; Biological: Donor Lymphocyte Infusion |
| VEN/AZA Expansion Cohort | Experimental | Participants in this group will receive VEN/AZA therapy at the most appropriate dose determined in Part 1. Participants may also receive donor lymphocyte infusions (DLI) at the discretion of the treating physician, if certain criteria are met.
Participants will receive up to one year (12 cycles) of study therapy, followed by up to one year of follow-up. Total participation duration is up to two years. Receives: Drug: Venetoclax; Drug: Azacitidine; Biological: Donor Lymphocyte Infusion |
What is being measured
The primary outcome is the main question the study is designed to answer.
Recommended Phase 2 Dose (RP2D)
The RP2D of VEN/AZA therapy will be determined as the maximum tolerated dose of study treatment as assessed by treating physician using the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version 5.0.
Measured: Up to 13 months
Number of Participants Experiencing Treatment-Related Toxicity
The number of participants experiencing treatment-related toxicity. Toxicity is defined as including dose limiting toxicities (DLTs), serious adverse events (SAEs) and adverse events (AEs) in study participants after starting study therapy. Toxicity will be assessed using the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version 5.0, per physician discretion.
Measured: Up to 13 months
Secondary outcomes (5)
Recurrence-Free Survival (RFS)
Recurrence-free survival (RFS) is defined as the elapsed time measured in months between the treatment start date (Cycle 1 Day 1) and date of documented disease recurrence or death from any cause. Alive patients without recurrence will be censored at the date of last documentation of recurrence-free status.
Measured: Up to 24 months
Overall Survival (OS)
Overall survival (OS) is defined as the elapsed time measured in months between the treatment start date (Cycle 1 Day 1) and date of death from any cause. Alive patients will be censored at the date last known to be alive.
Measured: Up to 24 months
Proportion of Participants with Treatment-Related Mortality (TRM)
Treatment-related Mortality (TRM) is defined as the proportion of participants that died after starting study therapy, during the time of observation and in the absence of disease progression.
Measured: 180 days
Number of Participants with acute GVHD After Allogeneic Hematopoietic Cell Transplant (HCT)
The number of participants with acute graft-versus-host disease (aGVHD) among study participants will be reported at +100 and +180 days post-allogeneic HCT.
Measured: Up to 180 days
Number of Participants with chronic GVHD After Allogeneic Hematopoietic Cell Transplant (HCT)
The number of participants with chronic graft-versus-host disease (cGVHD) among study participants will be reported at one (1) year post-allogeneic HCT.
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Dec 2023
- Study start4 Dec 2024
- Primary completion2 Jun 2026
- Study completion2 Jun 2026
Why it stopped: Funding Sponsor decision due to review of efficacy data from other trials involving investigational product.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acute Myeloid LeukemiaStudies of the same condition, open and under way.
- 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
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Model description
- Phase 1 dose escalation/de-escalation and dose expansion design.
- Masking
- None (open label)
- Enrolment
- 2 participants (actual)
Sponsor and investigators
- Lead sponsor
- Antonio M Jimenez Jimenez
- Sponsor type
- University, hospital or other
- Collaborators
- AbbVie
- Responsible party
- Sponsor-investigator, Antonio M Jimenez Jimenez, University of Miami
- Investigators
- Antonio M Jimenez Jimenez, MD, Principal investigator, University of Miami
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06158100
- Sponsor's protocol ID
- 20230190
- Status last verified
- Jun 2026
- First submitted
- 27 Nov 2023
- Last updated
- 13 Jul 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 13 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1 Study of Venetoclax in Combination With Azacitidine (VEN/AZA) Followed by Donor Lymphocyte Infusion (DLI) for Patients With Very High-Risk Acute Myeloid Leukemia (AML) Undergoing Allogeneic Hematopoietic Cell Transplant (HCT)
- ClinicalTrials.gov ID
- NCT06158100
- Acronym / sponsor ID
- 20230190
- Conditions
- Acute Myeloid Leukemia
- Keywords
- Hematopoietic Cell Transplant (HCT)
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Funding Sponsor decision due to review of efficacy data from other trials involving investigational product.
- Enrolment
- 2 participants (actual)
- Lead sponsor
- Antonio M Jimenez JimenezUniversity, hospital or other
- Collaborators
- AbbVie
- Study start
- 4 Dec 2024 (actual)
- Primary completion
- 2 Jun 2026 (actual)
- Study completion
- 2 Jun 2026 (actual)
- First posted
- 6 Dec 2023 (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 study is to see the effects of an investigational combination treatment of venetoclax, azacitidine, and donor lymphocyte infusion (DLI) in patients with high-risk AML receiving allogeneic hematopoietic cell transplantation, and to assess if the combination treatment is well tolerated and prevents disease relapse after transplant.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- Phase 1 dose escalation/de-escalation and dose expansion design.
| Arm | Type | Receives |
|---|---|---|
| VEN/AZA Dose Escalation/De-Escalation Cohort | Experimental | Drug: Venetoclax, Drug: Azacitidine, Biological: Donor Lymphocyte InfusionParticipants in this group will begin Venetoclax and Azacitidine (VEN/AZA) combination therapy between day +42 and day +100 following hematopoietic cell transplant (HCT) infusion. VEN/AZA combination therapy will be administered for up to six (6) cycles, followed by up to six (6) additional cycles of Venetoclax monotherapy in the absence of disease progression or unacceptable toxicity. Each cycle is 28 days. Participants may also receive donor lymphocyte infusions (DLI) at the discretion of the treating physician, if certain criteria are met. Participants will receive up to one year (12 cycles) of study therapy, followed by up to one year of follow-up. Total participation duration is up to two years. |
| VEN/AZA Expansion Cohort | Experimental | Drug: Venetoclax, Drug: Azacitidine, Biological: Donor Lymphocyte InfusionParticipants in this group will receive VEN/AZA therapy at the most appropriate dose determined in Part 1. Participants may also receive donor lymphocyte infusions (DLI) at the discretion of the treating physician, if certain criteria are met. Participants will receive up to one year (12 cycles) of study therapy, followed by up to one year of follow-up. Total participation duration is up to two years. |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (12 listed)
- Male and female patients between the ages of 18-75.
- Patients with a histologic diagnosis of AML in morphological remission (<5% bone marrow (BM) blasts) prior to allogeneic hematopoietic cell transplantation and very high-risk for relapse defined as: (i) Presence of measurable residual disease (MRD) by multicolor flow cytometry (MFC) prior to transplant and receiving a reduced intensity conditioning (RIC) or nonmyeloablative (NMA) regimen (ii) Presence of MRD by MFC at day +30 post-transplant (iii) All patients with monosomal karyotype (MK) and those with 17p/tumor protein p53 (TP53) mutated disease irrespective of MRD status and intensity of conditioning regimen.
- Adequate hematopoietic recovery after HCT, defined as:
- Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1, or 2
- Serum creatinine =< 1.5 mg/dL or creatinine clearance greater or equal than 40 cc/min
Key exclusion criteria (7 listed)
- Active disease (>5% blasts or any evidence of extra-medullary disease) at the time of transplantation or at day +30
- Active acute graft-versus-host disease (aGVHD) requiring systemic IST or history of aGVHD grade III or higher.
- Active chronic GVHD requiring systemic immunosuppressive therapy (IST).
- Active uncontrolled systemic fungal, bacterial, or viral infection
- Known active viral infection with human immunodeficiency virus (HIV), hepatitis B virus (HBV), or hepatitis C virus (HCV)
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 |
|---|---|---|
| VenetoclaxAlso: Venclexta, VenclyxtoVenetoclax will be given orally (PO) at the assigned doses of 50 or 100 mg once daily for 7, 14 or 21 days starting at day 1 of each 28-day cycle, for up to 12 cycles. | Drug | VEN/AZA Dose Escalation/De-Escalation Cohort, VEN/AZA Expansion Cohort |
| AzacitidineAlso: Vidaza, AzadineAzacitidine will be administered at a dose of 20mg/m^2 once daily subcutaneously (SC) or intravenously (IV) for five days (Days 1-5) of each 28-day cycle. | Drug | VEN/AZA Dose Escalation/De-Escalation Cohort, VEN/AZA Expansion Cohort |
| Donor Lymphocyte InfusionDonor lymphocyte infusions (DLI) may be administered at the discretion of the treating physician, if certain criteria are met. Participants may receive up to three (3) infusions of donor lymphocytes (a type of white blood cell) at the following dose levels and study timepoints: * DLI 1: 1x10^6 cluster of differentiation 3+ (CD3+) cells/kg, after cycle 3 * DLI 2: 5x10^6 CD3+ cells/kg, after cycle 5, if there is persistent MRD * DLI 3: 1x10^7 CD3+ cells/kg, after cycle 7, if there is persistent MRD. | Biological | VEN/AZA Dose Escalation/De-Escalation Cohort, VEN/AZA Expansion Cohort |
Primary outcome
Recommended Phase 2 Dose (RP2D)
Time frame: Up to 13 months
The RP2D of VEN/AZA therapy will be determined as the maximum tolerated dose of study treatment as assessed by treating physician using the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version 5.0.
Number of Participants Experiencing Treatment-Related Toxicity
Time frame: Up to 13 months
The number of participants experiencing treatment-related toxicity. Toxicity is defined as including dose limiting toxicities (DLTs), serious adverse events (SAEs) and adverse events (AEs) in study participants after starting study therapy. Toxicity will be assessed using the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version 5.0, per physician discretion.
5 secondary outcomes
Recurrence-Free Survival (RFS)
Time frame: Up to 24 months
Overall Survival (OS)
Time frame: Up to 24 months
Proportion of Participants with Treatment-Related Mortality (TRM)
Time frame: 180 days
Number of Participants with acute GVHD After Allogeneic Hematopoietic Cell Transplant (HCT)
Time frame: Up to 180 days
Number of Participants with chronic GVHD After Allogeneic Hematopoietic Cell Transplant (HCT)
Time frame: 1 year
Results and safety
The study was stopped early (reason given: Funding Sponsor decision due to review of efficacy data from other trials involving investigational product.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 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. This study is not recruiting at the moment.
Antonio M Jimenez Jimenez, MD principal investigator
University of Miami
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Venetoclax, Azacitidine and Donor Lymphocyte Infusion in people with Acute Myeloid Leukemia, run by Antonio M Jimenez Jimenez at 1 site 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 12 inclusion and 7 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: stopped early, as of the sponsor's last update on 13 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 1 site 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.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: Funding Sponsor decision due to review of efficacy data from other trials involving investigational product.). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Recommended Phase 2 Dose (RP2D) (time frame: Up to 13 months). The study lists 2 primary outcomes in all. 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 13 Jul 2026.