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Myelodysplastic SyndromesPhase 1NCT04980404

Inqovi Maintenance Therapy in Myeloid Neoplasms

A Phase 1 study testing Inqovi in people with Myelodysplastic Syndromes and Chronic Myelomonocytic Leukemia, run by Massachusetts General Hospital at 1 site in United States.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
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Trial summary

This research is being done to see if the drug Inqov is effective in reducing the chance of myelodysplastic syndrome (MDS) or chronic myelomonocytic leukemia (CMML) relapsing after standard of care stem cell transplant. Read more about this study

Who is it for?
Ages 18 years and older
What is being tested?
Inqovi
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
22 participants
Where
1 siteUnited States
Study dates
17 Sep 2021to Nov 2026
Phase
Phase 1
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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:
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  • 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)

Who cannot join

  • 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)
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  • 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

Trial locations and contacts

1 site in 1 country.

United States1 site

  • Massachusetts General Hospital

    Boston, Massachusetts, 02114

Other trials in United States

About this study

Official title: A Phase Ib Study of Oral Decitabine/Cedazuridine as Maintenance Therapy Following Allogeneic Hematopoietic Cell Transplantation for Patients With Myeloid Neoplasms

This research is being done to see if the drug Inqov is effective in reducing the chance of myelodysplastic syndrome (MDS) or chronic myelomonocytic leukemia (CMML) relapsing after standard of care stem cell transplant.

  • This research study involves the study drug Inqovi, which is a combination of the drugs decitabine and cedazuridine.
Read the detailed description

This is a prospective, non-randomized, open-label, phase Ib study of oral Inqov-decitabine/cedazuridine, given as maintenance therapy following allogeneic hematopoietic cell transplantation for patients with myeloid neoplasms

The U.S. Food and Drug Administration (FDA) has approved Inqovi for myelodysplastic syndrome (MDS) or chronic myelomonocytic leukemia (CMML) relapse but it has not been investigated in the post-transplant setting.

Inqovi is made up of the two study drugs decitabine and cedazuridine. Decitabine is believed to work by stopping cancer cells from growing and spreading. Cedazuridine is believed to work by slowing down how quickly the body breaks down decitabine, which normally breaks down too quickly to be effective.

The research study procedures include screening for eligibility and study treatment, including evaluations and follow up visits.

As the study is looking for the highest dose of Inqovi that can be administered safely without severe or unmanageable side effects not everyone will receive the same dose of the study drug. Dosage will depend on the number of participants who have been enrolled in the study before and how well they have tolerated their doses.

Participants will receive study treatment for up to 12 months and will be followed for up to 24 months after starting the study drug.

It is expected that about 22 people will take part in this research study.

Taiho Oncology, Inc., a pharmaceutical company, is supporting this research study by providing funding for the study, including the study drug.

Being tested
Inqovi
Keywords
Myelodysplastic Syndromes, Chronic Myelomonocytic Leukemia, Stem Cell Leukemia
Medical subject headings
Myelodysplastic Syndromes, Leukemia, Myelomonocytic, Chronic, Leukemia, Biphenotypic, Acute

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • InqoviDrug

    Also called decitabine, cedazuridine

    Tablet combination of drugs decitabine and cedazuridine, given orally.

Study groups in NCT04980404
Study groupRoleWhat they receive
Dose Escalation InqoviExperimentalStudy will follow a standard '3+3' dose escalation design: * Initial group of 3 participants will receive Inqovi (decitabine/cedazuridine) on days 1-3 of a 42 day cycle/dose-limiting toxicity (DLT) period. * Additional enrollment, dosage and study cyles will be determined by number of dose-limiting toxicity (DLT) that occur in initial group
Receives: Drug: Inqovi
Recommended Phase 2 Dose Expansion (RP2S) InqoviExperimentalOnce the Recommended Phase 2 Dose Expansion (RP2S) is established, 10 additional participants will be enrolled and receive Inqovi (decitabine/cedazuridine) on days 1-3 of a 28 day study cycle.
Receives: Drug: Inqovi

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Recommended Phase 2 Schedule Dose

    Identify the recommended phase II schedule of oral decitabine/cedazuridine through standard 3+3 Dose escalation model.

    Measured: 42 Days

Secondary outcomes (6)

  1. Median number of days of Inqovi tolerated

    Median number of day of Inqovi tolerated tabulated and reported descriptively.

    Measured: Up to 2 years

  2. Cumulative incidence of acute GVHD

    Cumulative incidence of acute GVHD tabulated and reported descriptively.

    Measured: Up to 2 years

  3. Cumulative incidence of significant chronic GVHD

    Cumulative incidence of significant chronic GVHD tabulated and reported descriptively.

    Measured: Up to 2 years

  4. Overall survival Rate

    Assessed using Kaplan-Meier

    Measured: The time from first dose of study drug to the date of death due to any cause up to 2 years

  5. Relapse-free survival Rate

    Assessed using Kaplan-Meier

    Measured: The time from first dose of study drug to the earlier of relapse or death due to any cause up to 2 years

  6. Proportion of subjects who successfully screen for study prior to transplantation but who do not reach the maintenance phase due to transplant related morbidity or mortality.

    Proportion of subjects who successfully screen for study prior to transplantation but who do not reach the maintenance phase due to transplant related morbidity or mortality tabulated and reported descriptively.

    Measured: Up to 2 years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted28 Jul 2021
  2. Study start17 Sep 2021
  3. Primary completion24 Aug 2023
  4. Study completionNov 2026 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Sequential
Masking
None (open label)
Enrolment
22 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04980404
Sponsor's protocol ID
21-214
Status last verified
Mar 2026
First submitted
18 Jul 2021
Last updated
30 Mar 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Yes: The Dana-Farber / Harvard Cancer Center encourages and supports the responsible and ethical sharing of data from clinical trials. De-identified participant data from the final research dataset used in the published manuscript may only be shared under the terms of a Data Use Agreement. Requests may be directed to: \[contact information for Sponsor Investigator or designee\]. The protocol and statistical analysis plan will be made available on Clinicaltrials.gov only as required by federal regulation or as a condition of awards and agreements supporting the research.

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing Inqovi in people with Myelodysplastic Syndromes and Chronic Myelomonocytic Leukemia, run by Massachusetts General Hospital at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 30 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: under way, not looking for participants, as of the sponsor's last update on 30 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Recommended Phase 2 Schedule Dose (time frame: 42 Days). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04980404.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 30 Mar 2026.

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