Can a targeted drug boost stem cell transplants for leukemia?
NCT ID NCT06954987
First seen Jun 25, 2026 · Last updated Sep 10, 2026 · Updated 3 times
Summary
This phase II trial tests whether adding venetoclax to standard chemotherapy and stem cell transplant, followed by venetoclax maintenance, helps adults with acute myeloid leukemia (AML) stay in remission longer. About 244 participants will receive either venetoclax or a placebo alongside their transplant. The goal is to see if this approach improves event-free survival and reduces the chance of cancer returning.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- venetoclax
- What this could lead to
- If successful, this could improve long-term control of AML by reducing the chance of cancer returning after a stem cell transplant.
- What could go wrong
- This is a mid-stage trial with only 244 participants, so results may not apply to everyone. Adding venetoclax may increase side effects like infections or organ damage.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
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About 244 people
The number the study aims to enrol. It can still change while the study runs.
- Expected to start
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Oct 2026
An estimate. Start dates often move.
- Expected to finish
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May 2028
An estimate. End dates often move.
- Lead sponsor
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A government research agency
The lead sponsor is the US National Institutes of Health.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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18 to 75 years
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * Patients must be registered to the Master Screening and Reassessment Protocol (MSRP) and assigned to this protocol by the MATCHBox Treatment Verification Team * STEP 1: History of acute myeloid leukemia (AML) by World Health Organization (WHO) criteria in first complete remission with morphologic complete remission (CR) or CR with incomplete hematologic recovery (CRi) defined as less than 5% bone marrow blasts by morphology with no circulating leukemic myeloblasts and no known extramedullary disease * STEP 1: MRD status by MDNet must have been performed * STEP 1 COHORT 1: Human leukocyte antigen (HLA)-matched donor defined as one of the following: * Sibling donor must be a 6/6 match at HLA-A and -B (intermediate or higher resolution) and -DRB1 (at high resolution using deoxyribonucleic acid \[DNA\]-based typing) * Related donor other than sibling must be an 8/8 match for HLA-A, -B, -C and -DRB1 at high resolution using DNA-based typing * Unrelated donor must be an 8/8 match at HLA-A, -B, -C and -DRB1 at high resolution using DNA-based typing * Age 40-75 years * STEP 1 COHORT 1: Not recommended for a myeloablative regimen by treating investigator * STEP 1 COHORT 2: Haploidentical or HLA-mismatched unrelated donor defined as one of the following: * Haploidentical donors: Related donor must be a haploidentical 3/6, 4/6, or 5/6 match at HLA-A and -B (intermediate or higher resolution) and -DRB1 (at high resolution using DNA-based typing) * HLA-mismatched unrelated donors: Unrelated donor must be a 6/8 or 7/8 match at HLA-A, -B, -C and -DRB1 at high resolution using DNA-based typing, ideally age \< 35 * Age 18-75 years * STEP 1 COHORT 2: Not recommended for a myeloablative regimen by treating investigator * STEP 1: Prior venetoclax therapy is allowed unless there is evidence of progression or no response (failure to achieve remission) to venetoclax-based regimen \< 2 months prior to registration * STEP 1: No prior allogeneic or autologous hematopoietic cell transplantation * STEP 1: Anti-leukemic therapy must be completed more than 14 days prior to registration with the exception of venetoclax * STEP 1: Karnofsky performance status ≥ 60 * STEP 1: Total bilirubin \< 2 x upper limit of normal (ULN) * STEP 1: Aspartate aminotransferase (AST) (serum glutamic oxaloacetic transaminase \[SGOT\])/alanine aminotransferase (ALT) (serum glutamic pyruvic transaminase \[SGPT\]) \< 3 x upper limit of normal (ULN) * STEP 1: Alkaline phosphatase ≤ 2.5 x upper limit of normal (ULN) * STEP 1: Pulmonary function diffusion capacity of the lung for carbon monoxide (DLCO) (corrected for hemoglobin) ≥ 40% and forced expiratory volume (FEV1) ≥ 50% * STEP 1: Calculated (Calc.) creatinine clearance ≥ 40 mL/min/based on the Cockcroft-Gault formula * STEP 1: Not pregnant and not nursing, because this study involves an agent that has known genotoxic, mutagenic and teratogenic effects. Therefore, for women of childbearing potential only, a negative pregnancy test done ≤ 7 days prior to registration is required * STEP 1: Patients with a prior or concurrent malignancy whose natural history or treatment does not have the potential to interfere with the safety or efficacy assessment of the investigational regimen are eligible for this trial * STEP 1: Patients with known HIV infection on effective anti-retroviral therapy with undetectable viral load within 6 months prior to registration are eligible for this trial * STEP 1: For patients with evidence of chronic hepatitis B virus (HBV) infection, the HBV viral load must be undetectable on suppressive therapy, if indicated * STEP 1: Patients with a history of hepatitis C virus (HCV) infection must have been treated and cured. For patients with HCV infection who are currently on treatment, they are eligible if they have an undetectable HCV viral load * STEP 1: Patients with known history or current symptoms of cardiac disease, or history of treatment with cardiotoxic agents, should have a clinical risk assessment of cardiac function using the New York Heart Association (NYHA) Functional Classification. To be eligible for this trial, patients should have ejection fraction ≥ 40% and NYHA functional status class II or better * STEP 1: No history of liver cirrhosis * STEP 1: No history of sinusoidal occlusion syndrome of the liver * STEP 1: No known intolerance or allergy to any protocol-defined agents * STEP 1: No known medical condition causing an inability to swallow oral formulations of agents * STEP 2: Patient has completed the conditioning and transplantation regimens * STEP 2: Patient has no evidence of AML morphologic relapse * STEP 2: Patient does not have flow measurable/minimal residual disease by myeloMATCH central labs (MDNet) testing defined as ≥ 0.1% on day 100+ (between day 80+ and 110+) marrow * STEP 2: Absolute neutrophil count \> 1,000/mcL (confirmed by a lab draw performed on two separate occasions \[\> 2 days apart\]) * STEP 2: Platelet count \> 50,000/mcL * STEP 2: AST (SGOT)/ALT (SGPT) \< 3 x upper limit of normal (ULN) * STEP 2: Total bilirubin \< 2 mg/dL x upper limit of normal (ULN) * STEP 2: Calc. creatinine clearance ≥ 40 mL/min/based on the Cockcroft-Gault formula * STEP 2: Patient does not have active/current grade 3-4 acute GVHD * STEP 2: Patient does not have evidence of grade 3 or higher sinusoidal occlusion syndrome/veno-occlusive disease of the liver as defined by European Society for Blood and Marrow Transplantation (EBMT) criteria
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The official record
The full official record for this study. This one lists no contact details, but it is the first place any would appear.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
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Other studies related to the condition(s) this trial covers.
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