New pill aims to keep AML away after transplant
NCT ID NCT06707493
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This phase 2 study tests whether the drug ivosidenib can help prevent acute myeloid leukemia (AML) from coming back after a stem cell transplant. About 75 adults with a specific IDH1 gene mutation will receive either ivosidenib or a placebo daily. The main goal is to see if the drug extends the time patients stay cancer-free.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- ivosidenib (a targeted pill that blocks a mutated IDH1 protein)
- What this could lead to
- If it works, this could help keep AML in remission longer after a stem cell transplant, reducing the chance of the cancer coming back.
- What could go wrong
- This is a mid-stage trial with only 75 people, so results may not be conclusive. Ivosidenib can cause side effects like heart rhythm changes or liver issues, and it may not prevent relapse for everyone.
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 75 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 2030
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 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: * Pathologically confirmed diagnosis of IDH1(R132)-mutant acute myeloid leukemia (AML). IDH1 mutations could have been detected by any mutational technique at any prior point including at diagnosis or remission. * Between the ages of 18 and 75 years * Will undergo allogeneic hematopoietic stem cell transplantation (HSCT) for their malignancy. Conditioning may be either conventional myeloablative (MAC) or reduced intensity conditioning (RIC). There will be no restrictions on type of graft source. * ECOG performance status ≤ 2 * Participants must have normal organ and marrow function as defined below: * Absolute neutrophil count ≥ 1000/µL without growth factor support (e.g. GCSF) in the previous 7 days. * Platelet count ≥ 50,000/µL without transfusional support in the previous 7 days. * AST (SGOT), ALT (SGPT) and Alkaline phosphatase \< 3x institutional upper limit of normal (ULN) * Direct bilirubin \< 2.0 mg/dL * Calculated creatinine clearance ≥ 40 mL/min (Cockcroft-Gault formula) * LVEF must be equal to or greater than 40%, as measured by MUGA scan or echocardiogram * Female patients of childbearing potential must have a negative pregnancy test * The effects of ivosidenib on the developing human fetus are unknown. For this reason female participants of child-bearing potential and male participants must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) during the entire study treatment period and through 90 days after the last dose of treatment * Ability to understand and the willingness to sign a written informed consent document. Exclusion Criteria: * Prior allogeneic hematopoietic stem cell transplants. * Morphologically relapsed or refractory disease, as assessed by bone marrow aspirate and biopsy performed within 42 days prior to study entry * History of other malignancy(ies) unless * the participant has been disease-free for at least 5 years and is deemed by the investigator to be at low risk of recurrence of that malignancy, or * the only prior malignancy was cervical cancer in situ and/or basal cell or squamous cell carcinoma of the skin * Known diagnosis of active hepatitis B or hepatitis C * Current or history of congestive heart failure New York Heart Association (NHYA) class 3 or 4, or any history of documented diastolic or systolic dysfunction (LVEF \< 40%, as measured by MUGA scan or echocardiogram) * Current or history of ventricular or life-threatening arrhythmias or diagnosis of long-QT syndrome * QTc interval (i.e., Friderica's correction \[QTcF\]) ≥ 450 ms or other factors that increase the risk of QT prolongation or arrhythmic events (e.g., heart failure, hypokalemia, family history of long QT interval syndrome) at screening * Uncontrolled intercurrent illness that would limit compliance with study requirements. Post-transplantation Pre-Treatment Criteria Treatment may begin at any time between day 45 and day 90 following stem cell transplantation. However, at time of treatment start, it must be ensured that: * The patient has continued willingness and interest in participating in the study. * There is no systemic infection requiring IV antibiotic therapy within 7 days preceding the first dose of study drug, or other severe infection * Chimerism studies reveal that ≥ 70% of blood or bone marrow cells, or of the CD33 expressing fraction, are of donor origin, * There is no acute graft versus host disease (GVHD), requiring an equivalent dose of ≥ 0.5mg/kg/day of prednisone within one week of starting ivosidenib / placebo, or have escalation of systemic immunosuppression in terms of increase of corticosteroids or addition of new agent/modality within two weeks of starting ivosidenib / placebo. * For prophylaxis for GVHD, agents that are permitted for administration on study: * Tacrolimus * Cyclosporine * Sirolimus * Cyclophosphamide * Mycophenolate Mofetil * Methotrexate * ATG * Ruxolitinib * Vedolizumab * As standards of care may change, any other prophylactic agents used should be discussed with the PI. * Investigational agents, defined as not approved for any indication, are forbidden unless the participant comes off study. * Agents used to treat GVHD that are permitted for administration on study: * Any agent used in prophylaxis may be continued (see list above) * Ruxolitinib * Etanarcept * ATG * Belumosidil * Axatilimab * Rituximab * Fecal microbiota transplantation * Alpha1-Antitrypsin * Pregnyl * Extracorporal photopheresis (ECP) * As standards of care may change, any other treatment agents used should be discussed with the PI. * Investigational agents, defined as not approved for any indication, are forbidden unless the participant comes off study. * There is no evidence of relapsed/recurrent/residual disease. * Prior to the start of ivosidenib / placebo administration, the participant must have adequate hematological function, defined as: * ANC ≥ 1000/µL * Platelets ≥ 50,000/µL and adequate organ function defined as * Direct bilirubin level \< 2.0 mg/dL * AST (SGOT), ALT (SGPT) and Alkaline phosphatase \< 3x institutional upper limit of normal (ULN) * No presence of congestive heart failure, defined by New York Heart Association (NHYA) criteria as class 3 or 4 * Calculated creatinine clearance ≥ 40 mL/min (Cockcroft-Gault formula)
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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
6 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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Dana Farber Cancer Institute
RECRUITINGBoston, Massachusetts, 02115, United States
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Emory University Hospital
RECRUITINGAtlanta, Georgia, 30322, United States
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Froedtert Hospital & the Medical College of Wisconsin
RECRUITINGMilwaukee, Wisconsin, 53226, United States
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Huntsman Cancer Institute, University of Utah
RECRUITINGSalt Lake City, Utah, 84112, United States
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Massachusetts General Hospital
RECRUITINGBoston, Massachusetts, 02114, United States
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Ohio State University Wexner Medical Center- James Cancer Hospital
RECRUITINGColumbus, Ohio, 43210, United States
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