New hope for tough AML: drug cocktail targets resistant leukemia
NCT ID NCT04774393
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study is testing a combination of oral medications for acute myeloid leukemia (AML) that has returned or not responded to treatment. The drugs include decitabine/cedazuridine, venetoclax, and either ivosidenib or enasidenib. The goal is to see if this combination is safe and effective in controlling the disease. About 84 participants will take part in this early-phase trial.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- Decitabine, cedazuridine, venetoclax, ivosidenib, or enasidenib
- What this could lead to
- If successful, this could offer a new treatment option for patients with hard-to-treat AML, potentially improving remission rates.
- What could go wrong
- This is an early-phase trial with a small number of participants, so results may not apply to everyone. The drug combination may cause side effects or not work as expected.
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 1/2
Runs two stages together: safety and dose first, then whether the treatment works.
- Participants
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About 84 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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May 2021
- Expected to finish
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Nov 2027
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 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 a diagnosis of relapsed or refractory acute myeloid leukemia (AML) (including biphenotypic or bilineage leukemia including a myeloid component or isolated extramedullary AML); OR * Patients (\> 60 year old) with newly diagnosed AML not eligible for intensive chemotherapy are also eligible * To be considered not eligible for intensive chemotherapy, participants must be defined by the following: Age 75 years or older, or Age 18 to 74 years with at least one of the following comorbidities: * Severe cardiac disorder (eg, congestive heart failure requiring treatment, ejection fraction ≤50%, or chronic stable angina). * Severe pulmonary disorder (eg, DLCO ≤65% or forced expiratory volume in 1 second \[FEV1\] ≤65%). * Creatinine clearance ≥30 mL/min to \<45 mL/min. * Moderate hepatic impairment with total bilirubin \>1.5 to ≤3.0 × upper limit of normal (ULN) * ECOG performance status of 2 or 3 * Age \>= 18 years * Subjects must have documented IDH1 or IDH2 gene mutation * Eastern Cooperative Oncology Group (ECOG) performance status =\< 2 * Adequate renal function including creatinine \< 2 unless related to the disease * Direct bilirubin \< 2 x upper limit of normal (ULN) unless increase is due to Gilbert's disease or leukemic involvement * Aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) \< 3 x ULN unless considered due to leukemic involvement, in which case direct bilirubin or AST and/or ALT \< 5 x ULN will be considered eligible) * In the absence of rapidly proliferative disease, the interval from prior treatment to time of initiation will be at least 7 days for cytotoxic or non-cytotoxic (immunotherapy agent(s). Oral hydroxyurea and/or cytarabine (up to 2 g/m\^2) for patients with rapidly proliferative disease is allowed before the start of study therapy, as needed, for clinical benefit and after discussion with the principle investigator (PI). Concurrent therapy for central nervous system (CNS) prophylaxis or continuation of therapy for controlled CNS disease is permitted * Male subjects who are sexually active with a women of childbearing potential (WOCBP) and who have not had vasectomies must be willing to use a barrier method of contraception and refrain from sperm donation from initial study drug until 90 days after last dose of study drug * Willing and able to provide informed consent Exclusion Criteria: * Patients with t(15;17) karyotypic abnormality or acute promyelocytic leukemia (French-American-British \[FAB\] class M3-AML) * Patients with any concurrent uncontrolled clinically significant medical condition including life-threatening severe infection, or psychiatric illness, which could place the patient at unacceptable risk of study treatment * Patients with active graft-versus-host-disease (GVHD) status post stem cell transplant (patients without active GVHD on chronic suppressive immunosuppression and/or phototherapy for chronic skin GVHD are permitted after discussion with the PI) * Patients with any severe gastrointestinal or metabolic condition which could interfere with the absorption of oral study medications * Corrected QT (QTc) interval using Fridericia's formula (QTcF) \>= 450 msec. Bundle branch block and prolonged QTc interval are permitted after discussion with the PI * Known active hepatitis B (HBV) or hepatitis C (HCV) infection or known human immunodeficiency virus (HIV) infection * Subject has a white blood cell count \> 25 x 10\^9/L. (Note: Hydroxyurea is permitted to meet this criterion) * Nursing women, women of childbearing potential (WOCBP) with positive urine pregnancy test, or women of childbearing potential who are not willing to maintain adequate contraception * Appropriate highly effective method(s) of contraception include oral or injectable hormonal birth control, intrauterine device (IUD), and double barrier methods (for example a condom in combination with a spermicide)
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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 places running it
1 site. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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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.
Contacts and locations
Locations
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M D Anderson Cancer Center
RECRUITINGHouston, Texas, 77030, United States
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Other studies related to the condition(s) this trial covers.
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- Tweaking donor cells may shield older transplant patients from a dangerous complication
- Can a drug and donor cells stop leukemia from returning after transplant?
- New drug combination targets Hard-to-Treat blood cancers