Ivosidenib as Post-HSCT Maintenance for AML with IDH1 Mutation
This study is testing whether ivosidenib, a drug that targets the IDH1 mutation, can help prevent acute myeloid leukemia (AML) from returning after a stem cell transplant (HSCT). You may be able to join if you are between 18 and 75 years old, have AML with an IDH1 mutation, and are planning to have an HSCT. Participants will receive either ivosidenib or a placebo (an inactive pill) after their transplant. The main goal is to see if ivosidenib helps you stay free of relapse. The study is currently unclear on its recruitment status and plans to enroll about 75 people.
- Study design
- This is a randomized, single-blinded, multi-center Phase 2 study comparing ivosidenib to a placebo in about 75 participants.
- What's involved
- You would participate for an estimated 3.5 years, including up to 24 months of study treatment, with evaluations and follow-up.
- Compensation
- Not stated in the trial record.
- Follow-up
- After 24 months of treatment, participants are followed for up to 12 additional months.
AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.
NCT06707493
Ivosidenib as Post-HSCT Maintenance for AML
Recruiting
PHASE2Ages 18–75InterventionalTreatmentMassachusetts General HospitalInvestigator-initiated
~75 participants
Updated 2026-06-18 on ClinicalTrials.gov
What's tested:IvosidenibPlacebo
At a glance
Recruiting sites
6 of 6 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Relapse-Free Survival (RFS)
Measured over Time of randomization to 24 months post-randomization, death, or disease relapse whichever occurs first.
Conditions
Where it's being run
6 sites across 5 statesMassachusetts2
Georgia1
Ohio1
Utah1
Wisconsin1
Study leadership
- Amir T Fathi, MD · PRINCIPAL_INVESTIGATOR · Massachusetts General Hospital
Who to contact
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Do you actually qualify for this trial?
Add a private profile and we'll compare every criterion below against your situation — and tell you which ones are met, uncertain, or excluding.
Eligibility criteria
Inclusion
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
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.
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
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)
What this trial measures
- Relapse-Free Survival (RFS)Time of randomization to 24 months post-randomization, death, or disease relapse whichever occurs first.
Relapse-Free Survival is defined as the time from randomization following transplant to disease relapse or death due to any cause, whichever occurs first. Participants alive without relapse are censored at the date of last seen alive. The primary analysis will be performed using the Kaplan-Meier method with log-rank test.