New hope: targeted pill may keep blood cancer in check after transplant

NCT ID NCT03564821

What the study statuses mean

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Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tested the drug ivosidenib as a maintenance therapy for people with IDH1-mutant blood cancers (like AML) after a stem cell transplant. The goal was to find the safest dose and see if it could help prevent the cancer from coming back. 18 participants took the pill daily, starting at 500 mg, with a lower dose option if needed. The study was completed, and results help guide future research on this approach.

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

Phase 1

The first testing in people. Mainly checks safety and dose, usually in a small group.

Participants

18 people

The number who actually took part.

Started

Jan 2019

Finished

Jun 2023

Lead sponsor

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

18 years and older

Sex

Anyone

Healthy volunteers

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

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), myelodysplastic syndrome (MDS) or chronic myelomonocytic leukemia (CMML). 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). * HSCT Donor will be one of the following: * 5/6 or 6/6 (HLA-A, B, DR) matched related donor * 7/8 or 8/8 (HLA-A, B, DR, C) matched unrelated donor. Matching in the unrelated setting must be at the allele level. * Haploidentical related donor, defined as ≥ 3/6 (HLA-A, B, DR) matched --≥ 4/6 (HLA-A, B, DR) umbilical cord blood (UCB). Matching in the UCB setting is at the antigen level. Recipients may receive either one or two UCB units. In the case of 2 UCB units, both units must have been at least 4/6 matched with the recipient. * 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. * Evidence of relapsed/recurrent/residual 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 * Systemic infection requiring IV antibiotic therapy within 7 days preceding the first dose of study drug, or other severe infection * Uncontrolled intercurrent illness that would limit compliance with study requirements. * HIV-positive participants on combination antiretroviral therapy are ineligible because of the potential for pharmacokinetic interactions with study drug. In addition, these participants are at increased risk of lethal infections when treated with marrow-suppressive therapy.

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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

  • Dana Farber Cancer Institute

    Boston, Massachusetts, 02115, United States

  • Johns Hopkins Cancer Center

    Baltimore, Maryland, 21218, United States

  • Massachusetts General Hospital

    Boston, Massachusetts, 02214, United States

  • Ohio State University Comprehensive Cancer Center

    Columbus, Ohio, 43210, United States

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