Could a targeted pill boost chemo for a tough blood cancer?
NCT ID NCT03709758
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This early-phase trial is testing whether adding the targeted drug venetoclax to standard chemotherapy can safely treat adults newly diagnosed with acute myeloid leukemia (AML). About 50 participants will receive the combination to find the best dose and see how many achieve remission. The goal is to improve on current treatments without causing too many extra side effects.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- Venetoclax (a targeted drug that helps kill cancer cells) combined with standard chemotherapy drugs daunorubicin and cytarabine
- What this could lead to
- If successful, this could lead to a more effective initial treatment for acute myeloid leukemia, potentially improving remission rates.
- What could go wrong
- This is an early Phase 1 trial with only 50 participants, so safety and dosing are still being figured out. The added drug may increase side effects without guaranteeing better outcomes.
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 1
The first testing in people. Mainly checks safety and dose, usually in a small group.
- Participants
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50 people
The number who actually took part.
- Started
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Oct 2018
- Expected to finish
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Sep 2026
An estimate. End dates often move.
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
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 60 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 with AML who are newly diagnosed according to the WHO 2016 Classification and previously untreated with the exception of hydroxyurea. ATRA pretreatment for suspected APL for less than 5 days is allowed. Eligible patients with AML arising from an antecedent hematologic disease (AHD) including MDS, may have been treated for their prior hematologic disease (except for allogenic transplant). * AML patients include de-novo AML, AML evolving from MDS or other AHD and AML after previous cytotoxic therapy or radiation (secondary AML). * For a diagnosis of AML, a bone marrow or peripheral blast count of 20% or more is required. * In AML with monocytic or myelomonocytic differentiation, monoblasts and promonocytes, but not abnormal mature monocytes, are counted as blast equivalents. * Patients must be ≥18 and ≤60 years old. * Eastern Cooperative Oncology Group (ECOG) Performance status of 0 to 2. (See protocol Appendix D.) * LVEF ≥ 45% by MUGA or ECHO at screening. * Adequate renal function as demonstrated by a calculated creatinine clearance ≥ 50 mL/min; determined via urine collection for 24-hour creatinine clearance or by the Cockcroft Gault formula. * Adequate liver function as demonstrated by: * aspartate aminotransferase (AST) ≤ 2.5 × ULN\* * alanine aminotransferase (ALT) ≤ 2.5× ULN\* * total bilirubin ≤ 1.5 × ULN\* * Unless considered due to leukemic organ involvement. * Subjects with Gilbert's Syndrome may have a total bilirubin \> 1.5 × ULN per discussion with the overall study PI * Male subjects must agree to refrain from unprotected sex and sperm donation from initial study drug administration until 90 days after the last dose of study drug. * Females of childbearing potential (i.e., not postmenopausal for at least 1 year or not surgically sterile) must have negative results by a serum pregnancy test performed within 7 days of day 1. * Subject must voluntarily sign and date an informed consent, approved by an Independent Ethics Committee (IEC)/Institutional Review Board (IRB), prior to the initiation of any screening or study-specific procedures. Exclusion Criteria: * Subject has acute promyelocytic leukemia, inversion16, t(8;21) or FLT3 mutant AML as described below. Contact PI with questions. * Inversion 16 and t(8;21): CBF chromosomal abnormalities may be assessed by molecular (PCR), metaphase cytogenetics, or FISH * FLT3: ITD or a point mutation in the TKD loop of variant allele fractions ≥5% by PCR, capillary electrophoresis, or NGS panel capable of defining FLT3 allelic burden * Subject has known active CNS involvement with AML. * Subject has tested positive for HIV (due to potential drug-drug interactions between antiretroviral medications and venetoclax, as well as anticipated venetoclax mechanism-based lymphopenia that may potentially increase the risk of opportunistic infections). Note: HIV testing is not required. * Subject is known to be positive for hepatitis B or C infection with the exception of those with an undetectable viral load within 3 months. (Hepatitis B or C testing is not required). Subjects with serologic evidence of prior vaccination to HBV \[i.e., HBs Ag-, and anti-HBs+\] are allowed. * Subject has received the following within 7 days prior to the initiation of study treatment: * Strong or moderate CYP3A inducers (see Appendix C) * Strong and moderate CYP3A inhibitors (see Appendix C) * Subject has consumed grapefruit, grapefruit products, Seville oranges (including marmalade containing Seville oranges) or Star fruit within 3 days prior to the initiation of study treatment. * Subject has a cardiovascular disability status of New York Heart Association Class ≥ 2. Class 2 is defined as cardiac disease in which patients are comfortable at rest but ordinary physical activity results in fatigue, palpitations, dyspnea, or anginal pain. * Subject has a significant history of renal, neurologic, psychiatric, endocrinologic, metabolic, immunologic, hepatic, cardiovascular disease, or any other medical condition that in the opinion of the investigator would adversely affect his/her participating in this study. * Subject has chronic respiratory disease that requires continuous oxygen use. * Subject has a malabsorption syndrome or other condition that precludes enteral route of administration. * Subject exhibits evidence of other clinically significant uncontrolled condition(s) including, but not limited to: uncontrolled systemic infection. * Subject has a history of other malignancies prior to study entry, with the exception of: * Adequately treated in situ carcinoma of the cervix uteri 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. * Prior malignancies treated with (surgery+/- chemotherapy+/- radiation) that have remained disease free for at least two years after completion of therapy * Subject has a white blood cell count \> 25 × 109/L. Note: Hydroxyurea is permitted to meet this criterion. * Subject treated with any form of chemotherapy, immunotherapy, or investigative agent within 1 month of enrollment.
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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.
Contacts and locations
Locations
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Dana Farber Cancer Institute
Boston, Massachusetts, 02215, United States
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University of Chicago
Chicago, Illinois, 60637, United States
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