Targeted drug shows promise as maintenance after transplant for rare blood cancers
NCT ID NCT03515512
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This phase 1 study tested the drug enasidenib as a maintenance therapy after a stem cell transplant in 23 people with IDH2-mutant acute myeloid leukemia or chronic myelomonocytic leukemia. The goal was to find the safest dose and see how well patients tolerated it. Enasidenib works by blocking a mutated protein that can fuel cancer growth. The study was completed, and results help guide future research on using targeted therapy to prevent relapse after transplant.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- Enasidenib (a targeted therapy drug that blocks the mutated IDH2 protein, which can fuel cancer growth)
- What this could lead to
- If this works, it could offer a way to keep IDH2-mutant blood cancers in remission after a stem cell transplant, potentially reducing the chance of relapse.
- What could go wrong
- This is a very early (phase 1) study with only 23 participants, so we don't yet know if enasidenib truly prevents relapse. The drug may cause side effects, and not all patients may benefit.
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
-
23 people
The number who actually took part.
- Started
-
Jul 2018
- Finished
-
Feb 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 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 IDH2-mutant acute myeloid leukemia (AML), myelodysplastic syndrome (MDS) or chronic myelomonocytic leukemia (CMML). * IDH2 mutations will include any IDH2 R140 or R172 alterations * Eligibility and enrollment will be based on local IDH2 mutational testing performed at any center. The presence of an IDH2 mutation at the time of initial diagnosis or any other time thereafter is necessary and sufficient. The presence of an IDH2 mutation at time of enrollment is not necessary for the purposes of eligibility. * Between the ages of 18 and 75 years * Will undergo first allogeneic hematopoietic stem cell transplantation (HSCT) for their malignancy. Conditioning may have been 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 50%, as measured by MUGA scan or echocardiogram * Female patients of childbearing potential must have a negative pregnancy test, as measured by serum or urine testing * The effects of enasidenib on the developing human fetus are unknown. For this reason women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) during the entire study treatment period and through 6 months 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 \< 50%, as measured by MUGA scan or echocardiogram) * Current or history of ventricular or life-threatening arrhythmias or diagnosis of long-QT syndrome * Systemic infection requiring IV antibiotic or antifungal or antiviral therapy within 7 days preceding the first dose of study drug, or other severe infection * Known dysphagia, short-gut syndrome, gastroparesis, or other condition(s) that limits the ingestion or gastrointestinal absorption of drugs administered orally * Uncontrolled hypertension (systolic blood pressure \[BP\] \> 180 mmHg or diastolic BP \> 100 mmHg) * 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 * Concomitant receipt of the following sensitive CYP substrate medications that have a narrow therapeutic range (unless the participant can be transferred to other medications at least 5 half-lives prior to the start of study treatment): paclitaxel and docetaxel (CYP2C8), phenytoin (CYP2C9), S-mephenytoin (CYP2C19), thioridazine (CYP2D6), theophylline, and tizanidine (CYP1A2) * Concomitant receipt of the breast cancer resistance protein (BCRP) transporter-sensitive substrate rosuvastain (unless the participant can be transferred to another medication at least 5 half-lives prior to the start of study treatment) * 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.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for Acute myeloid leukemia are added.
Genom att skicka in godkänner du våra Användarvillkor
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, 02215, United States
-
Johns Hopkins Cancer Center
Baltimore, Maryland, 21287, United States
-
Massachusetts General Hospital
Boston, Massachusetts, 02214, United States
-
Ohio State University Comprehensive Cancer Center
Columbus, Ohio, 43210, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Can a p53-Targeting drug boost chemotherapy in Hard-to-Treat blood cancers?
- Can a Platelet-Boosting drug help control a rare bone marrow disorder?
- Can adding venetoclax make donor stem cell transplants safer for High-Risk blood cancers?
- Can an HDAC inhibitor wipe out residual leukemia cells?
- Can an experimental pill block a cancer-driving enzyme in hard-to-treat leukemia?
- Two-Drug combo targets leukemia that outsmarted its first treatment