Can a cancer drug stop a blood disorder before it turns deadly?
NCT ID NCT06802146
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This pilot study is for people with high-risk CCUS, a blood condition that can lead to cancer. Participants can choose to be watched or take an oral drug called Inqovi (decitabine/cedazuridine). The goal is to see if early treatment is feasible and safe. The study is small and early, so it won't prove whether the drug works, but it may guide future research.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- Decitabine and cedazuridine (Inqovi)
- What this could lead to
- If successful, this could show that early treatment of high-risk CCUS is feasible and safe, potentially preventing progression to blood cancer.
- What could go wrong
- This is a very early, small pilot study (108 participants) focused on feasibility and safety, not effectiveness. The drug may cause side effects, and it's unknown if early treatment truly prevents cancer.
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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Early phase 1
The earliest testing in people: a first look at safety, in a very small group.
- Participants
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About 108 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Feb 2025
- Expected to finish
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Dec 2028
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: * Age ≥18 years. * Unexplained cytopenia(s) for at least 4 months (at least two separate labs within 4 months including at time of screening must meet this criteria). Cytopenia(s) defined as the presence of ≥ 1 of the following: * Hemoglobin (Hgb) \<12 g/dL for women and \<13g/dL for men * Absolute neutrophil count (ANC) \< 1.8 × 109/L\* * Platelet count (Plt) \<150 × 109/L \*Patients known to have a Duffy-null genotype must have anemia (Hgb \< 12g/dL for women, Hgb \<13g/dL for men) and/or thrombocytopenia (Plt \< 150 × 109/L) to be eligible for this study. * 1 pathogenic variant detected in any myeloid driver gene with a VAF of at least 0.02 (2%) identified by local next generation sequencing (NGS) of peripheral blood or bone marrow sample within 3 months from screening bone marrow biopsy. * Participants must have a high risk score per the Clonal Hematopoiesis Risk Calculator (CHRS). See APPENDIX C for calculation. * Screening bone marrow biopsy must not be diagnostic of any overt hematologic malignancy by morphologic assessment and must be consistent with a diagnosis of clonal cytopenia of unknown significance (CCUS) as determined by multi-institutional hematopathology review. * ECOG performance status 0-2 (see Appendix A). * Participants must meet the following organ function as defined below: * Serum aspartate aminotransferase/serum glutamic oxaloacetic transaminase (AST/SGOT) and alanine aminotransferase (ALT/SGPT) ≤3x upper limit of normal (ULN). * Serum total bilirubin \<1.5x ULN. Higher levels are acceptable if these can be attributed to ineffective erythropoiesis or Gilbert's syndrome. In these cases, approval from the study Sponsor-Investigator is required. * Creatinine clearance greater than 40 mL/min based on the Cockcroft-Gault glomerular filtration rate estimation. * Ability to understand and the willingness to sign a written informed consent document. * For participants of the early pharmacologic intervention cohort: women of childbearing potential must use highly effective contraception during treatment for at least 6 months after the last dose and males with female partners of reproductive potential should use effective contraception during treatment and for 3 months after the last dose. Exclusion Criteria: * Concurrent primary malignancy requiring active cytotoxic chemotherapy and/or ionizing radiation therapy. * Known inherited bone marrow failure disorder and/or germline predisposition to hematologic malignancy. * Receipt of anti-cancer therapy including any cytotoxic chemotherapy, ionizing radiation therapy, immunomodulatory agents such as lenalidomide, and targeted anti-cancer therapies including PARP inhibitors within the last 6 months. Patients with complete surgical resection of a tumor are not excluded from this study. * Anti-cancer therapy, including any cytotoxic chemotherapy, ionizing radiation therapy, immunomodulatory agents such as lenalidomide and targeted agents such as PARP inhibitors, planned in the next 6 months. Patients on hormonal adjuvant therapy for nonmetastatic breast and prostate cancer or other minimally-myelosuppressive maintenance therapies for non-metastatic cancer may be eligible at the discretion of the study PI. * Diagnosis of MDS, MPN, CMML, AML or any other hematolymphoid malignancy in the patient's lifetime. This includes individuals with MDS-defining chromosomal abnormalities identified via conventional karyotype or FISH. * Presence of a concurrent hematologic malignancy precursor state, such as smoldering multiple myeloma (SMM), and smoldering Waldenstrom's macroglobulinemia. * Presence of an early-stage hematologic precursor state-such as monoclonal gammopathy of undetermined significance (MGUS) and monoclonal B cell lymphocytosis (MBL). * Active uncontrolled systemic fungal, bacterial, or viral infection (defined as ongoing signs/symptoms related to the infection without improvement despite appropriate antibiotics, antiviral therapy, and/or other treatment). * Recent (within 3 months) vaccination with any live attenuated vaccine or vaccination with live attenuated vaccine planned during the next 15 months. \*Live attenuated vaccines include measles, mumps, rubella (MMR combined vaccine), rotavirus, smallpox, chickenpox, and yellow fever. * Laboratory evidence indicative of clinically significant red cell hemolysis. * Hypersplenism and/or evidence of portal hypertension on physical exam or imaging. * Pregnant or lactating.
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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.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The places running it
1 site. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Locations
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Dana-Farber Cancer Institute
RECRUITINGBoston, Massachusetts, 02215, United States
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