New drug cocktail shows promise against tough leukemia
NCT ID NCT04188405
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tested a combination of three drugs (decitabine, venetoclax, and ponatinib) in 20 adults with a rare, aggressive leukemia called Philadelphia chromosome-positive acute myeloid leukemia or chronic myelogenous leukemia in blast phase. The goal was to see if the drugs could shrink or eliminate the cancer enough to allow for a stem cell transplant. The treatment aims to control the disease, not cure it, as ongoing management is typically needed.
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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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20 people
The number who actually took part.
- Started
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May 2020
- Finished
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Jun 2026
- 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 years and older
- 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 Philadelphia (Ph)+ acute myeloid leukemia (AML) or myeloid accelerated phase (AP)-chronic myelogenous leukemia (CML) or blast phase (BP)-CML (either t\[9;22\] and/or BCR-ABL1 positive by fluorescent in situ hybridization or polymerase chain reaction). Both untreated and relapsed/refractory patients are eligible * Performance status =\< 3 (Eastern Cooperative Oncology Group \[ECOG\] scale) * Total serum bilirubin =\< 2 x upper limit of normal (ULN), unless due to Gilbert's syndrome, hemolysis or the underlying leukemia approved by the principal investigator (PI) * Alanine aminotransferase (ALT) =\< 1.5 x ULN, unless due to the underlying leukemia approved by the PI * Aspartate aminotransferase (AST) =\< 1.5 x ULN unless due to the underlying leukemia approved by the PI * Creatinine clearance \>= 30 mL/min * Serum lipase =\< 1.5 x ULN * Amylase =\< 1.5 x ULN * Ability to swallow * Signed informed consent Exclusion Criteria: * Active serious infection not controlled by oral or intravenous antibiotics (e.g. persistent fever or lack of improvement despite antimicrobial treatment) * History of acute pancreatitis within 6 months of study or history of chronic pancreatitis * Uncontrolled hypertriglyceridemia (triglycerides \> 450 mg/dL) * Active secondary malignancy that in the investigator's opinion will shorten survival to less than 1 year * Active grade III-V cardiac failure as defined by the New York Heart Association criteria * Clinically significant, uncontrolled, or active cardiovascular disease, specifically including, but not restricted to: * Myocardial infarction (MI), stroke, revascularization, unstable angina or transient ischemic attack within 6 months * Left ventricular ejection fraction (LVEF) less than lower limit of normal per local institutional standards prior to enrollment * Diagnosed or suspected congenital long QT syndrome * Clinically significant atrial or ventricular arrhythmias (such as uncontrolled, clinically significant atrial fibrillation, ventricular tachycardia, ventricular fibrillation, or torsades de pointes) as determined by the treating physician * Prolonged corrected QT (QTc) interval on pre-entry electrocardiogram (\> 480 msec) unless corrected after electrolyte replacement * History of venous thromboembolism including deep venous thrombosis or pulmonary embolism within the past 3 months, excluding line-associated deep venous thrombosis (DVT) of the upper extremity * Uncontrolled hypertension (diastolic blood pressure \> 100 mmHg; systolic \> 150 mmHg) * Consumed grapefruit, grapefruit products, Seville oranges, or star fruit within 3 days prior to starting venetoclax * Treatment with any investigational antileukemic agents or chemotherapy agents in the last 7 days before study entry, unless full recovery from side effects has occurred or patient has rapidly progressive disease judged to be life-threatening by the investigator. Prior recent treatment with corticosteroids, hydroxyurea, or a Food and Drug Administration (FDA)-approved BCR-ABL tyrosine kinase inhibitor (TKI) is permitted * Pregnant and lactating women will not be eligible; women of childbearing potential should have a negative pregnancy test prior to entering on the study and be willing to practice methods of contraception throughout the study period. Women do not have childbearing potential if they have had a hysterectomy or are postmenopausal without menses for 12 months. In addition, men enrolled on this study should understand the risks to any sexual partner of childbearing potential and should practice an effective method of birth control
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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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M D Anderson Cancer Center
Houston, Texas, 77030, United States
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Other studies related to the condition(s) this trial covers.
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