Can a pill keep blood cancer away after transplant?
NCT ID NCT04980404
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This phase 1 trial tests whether the oral drug Inqovi (decitabine/cedazuridine) can help prevent myelodysplastic syndrome (MDS) or chronic myelomonocytic leukemia (CMML) from returning after a stem cell transplant. About 22 adults who have had a reduced-intensity transplant will take Inqovi as maintenance therapy. The main goal is to find the safest and most tolerable dose schedule.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- Inqovi (decitabine and cedazuridine combination tablet)
- What this could lead to
- If it works, this could point toward a way to keep MDS or CMML from coming back after a stem cell transplant.
- What could go wrong
- This is a very early phase 1 trial with only 22 people, so it is mainly checking safety and dosing. It may not show a clear benefit, and side effects like graft-versus-host disease are possible.
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 1
The first testing in people. Mainly checks safety and dose, usually in a small group.
- Participants
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About 22 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Sep 2021
- Expected to finish
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Nov 2026
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 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: * Pathologically confirmed diagnosis of myelodysplastic syndrome (MDS) or chronic myelomonocytic leukemia (CMML). * Subjects should have less than 5% myeloblasts on a bone marrow biopsy within 42 days prior to the start of conditioning. * Age ≥ 18 * Will undergo first allogeneic hematopoietic stem cell transplantation (HSCT) for their malignancy. * Transplantation will be performed with the use of 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 0-2. * Participants must have normal organ and function as defined below: * AST (SGOT), ALT (SGPT) and Alkaline phosphatase \< 3x institutional upper limit of normal (ULN) * Total bilirubin \< 1.5 x ULN (with the exception of subjects with a history of Gilbert's syndrome) * Calculated creatinine clearance ≥ 30 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 decitabine/cedazuridine 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. Eligibility Criteria Prior to Treatment (Post HCT) * Maintenance therapy may begin at any time between day 30 and day 120 following hematopoietic cell transplantation. Participants must meet the following criteria to be eligible to treatment on this study: * 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 escalation of corticosteroid dose or addition of other agent in the 4 weeks prior to Cycle 1 Day 1. * There is no morphological evidence of relapsed/recurrent/residual disease (as assessed by post HCT bone marrow biopsy and aspirate). * There is no systemic infection requiring IV antibiotic or antifungal or antiviral therapy within 7 days of starting decitabine/cedazuridine * ANC ≥ 1000/µL * Platelets ≥ 50,000/µL * AST (SGOT), ALT (SGPT) and Alkaline phosphatase \< 3x institutional upper limit of normal (ULN) * Total bilirubin \< 1.5 x ULN (with the exception of subjects with a history of Gilbert's syndrome) * Calculated creatinine clearance ≥ 30 mL/min (Cockcroft-Gault formula) Exclusion Criteria: * Prior allogeneic hematopoietic stem cell transplants. * History of other malignancy(ies) unless * the participant has been disease-free for at least 12 months 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 uncontrolled 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 * Uncontrolled intercurrent illness that would limit compliance with study requirements. * Breastfeeding women
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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
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Massachusetts General Hospital
Boston, Massachusetts, 02114, United States
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Other studies related to the condition(s) this trial covers.
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