New combo therapy aims to keep leukemia from returning
NCT ID NCT07392242
First seen Jun 26, 2026 · Last updated Jul 17, 2026 · Updated 2 times
Summary
This phase 2 study tests whether a three-drug combination (ivosidenib, azacitidine, and venetoclax) followed by maintenance therapy with ivosidenib alone can prevent acute myeloid leukemia (AML) from coming back in patients with an IDH1 mutation. The trial enrolls 45 people aged 60 or older with newly diagnosed AML. Researchers will measure how long patients stay cancer-free and track 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
- ivosidenib, azacitidine, venetoclax
- What this could lead to
- If successful, this approach could help keep acute myeloid leukemia from returning after initial treatment, offering longer remission for patients with IDH1 mutations.
- What could go wrong
- This is a small, early-phase trial (45 people) with no control group, so results may not apply broadly. The drugs can cause side effects like low blood counts or infections.
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 45 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Jan 2026
- Expected to finish
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Jan 2028
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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60 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: * Participant must be at least 60 years of age at the time of signing the informed consent form (ICF). * Participant is willing and able to adhere to the study visit schedule and other protocol requirements. * Participant has newly diagnosed AML as per World Health Organization 2022 or European leukemia Network 2022 guidelines. * Participant has IDH1-R132 mutation present prior to initiating Ivo/Aza/Ven confirmed by CLIA approved local testing via next-generation sequencing (NGS) and/or polymerase chain reaction (PCR). Other 2-HG producing IDH1 variants may be eligible after discussion with MSK principal investigator. 1. At MSK, this testing will utilize the MSK-REACT, a rapid multi-gene NGS panel used in all new AML diagnoses that is clinically validated by the Laboratory of Diagnostic Molecular Pathology pursuant to the requirements of CLIA'88 and approved by New York State. Other sites may use local CLIA-certified laboratories and validated clinical assays as per standard of care. 2. The patient's chart will be utilized for screening purposes * Participant has Eastern Cooperative Oncology Group (ECOG) performance status of 0-3 * Participant must have a WBC count \<25,000/μL at the time of initiation of study drug (leukapheresis may be performed and/or hydroxyurea may be administered to decrease the WBC count to \<25,000/μL). * Participant has adequate organ function defined as: 1. Serum aspartate aminotransferase/serum glutamic oxaloacetic transaminase (AST/SGOT) and alanine aminotransferase (ALT/SGPT) ≤ 3 x ULN, unless considered due to leukemic organ involvement. 2. Serum total bilirubin \< 3.0 x ULN. Higher levels are acceptable if these can be attributed to ineffective erythropoiesis, leukemia organ involvement or Gilbert's syndrome. 3. Serum creatinine \< 2 x ULN or creatinine clearance 30 mL/min based on the Cockroft-Gault glomerular filtration rate (GFR) estimation. Exclusion Criteria: * Participant with acute promyelocytic leukemia * Participants who have previously received ivosidenib or venetoclax * Participant receiving any other investigational anti-cancer agents. Cytoreductive therapy such as hydroxyurea is permitted. * Participants with immediate life-threatening, severe complications of leukemia such as uncontrolled bleeding, pneumonia with hypoxia or shock, and/or disseminated intravascular coagulation * Participant has active uncontrolled systemic fungal, bacterial, or viral infection (defined as ongoing signs/symptoms related to the infection without improvement despite appropriate antibiotics, antiviral therapy, and/or other treatment). * Participants who are planned for allogeneic stem cell transplantation based on the assessment of the treating clinician. * Participant has significant active cardiac disease within 6 months prior to start of study treatment, including New York Heart Association (NYHA) class III or IV congestive heart failure; acute coronary syndrome (ACS); and/or stroke * Participant has active viral infection with human immunodeficiency virus (HIV), or active infection with hepatitis B virus (HBV) or hepatitis C virus (HCV). As per exclusion criteria 5, patients receiving appropriate treatment would only be excluded if there is no improvement. * Participant is known to have dysphagia, short-gut syndrome, gastroparesis, or other conditions that limit the ingestion or gastrointestinal absorption of drugs administered orally. * Participant has QTc interval (i.e., Fridericia's correction \[QTcF\]) ≥ 450 ms (mean of triplicate ECG) or other factors that increase the risk of QT prolongation or ventricular arrhythmic events (e.g. family history of long QT interval syndrome). Participants with a QTcF over 450 ms due * Male or female participants not willing to comply with contraceptive requirements
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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
7 sites. 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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Memoral Sloan Kettering at Basking Ridge (Limited Protocol Activities)
RECRUITINGBasking Ridge, New Jersey, 07920, United States
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Memorial Sloan Kettering Bergen (Limited Protocol Activities)
RECRUITINGMontvale, New Jersey, 07645, United States
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Memorial Sloan Kettering Cancer Center (All Protocol Activities)
RECRUITINGNew York, New York, 10065, United States
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Memorial Sloan Kettering Monmouth (Limited Protocol Activities)
RECRUITINGMiddletown, New Jersey, 07748, United States
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Memorial Sloan Kettering Nassau (Limited Protocol Activites)
RECRUITINGRockville Centre, New York, 11553, United States
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Memorial Sloan Kettering Suffolk- Commack (Limited Protocol Activities)
RECRUITINGCommack, New York, 11725, United States
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Memorial Sloan Kettering Westchester (Limited Protocol Activities)
RECRUITINGHarrison, New York, 10604, United States
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Other studies related to the condition(s) this trial covers.
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- Two-Drug combo targets leukemia that outsmarted its first treatment
- 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