Could a gentle pill stop blood cancer before it starts?
NCT ID NCT07435636
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tests a low-dose oral drug (decitabine with tetrahydrouridine) in 80 adults aged 60–85 who have a high-risk blood condition called CCUS, which often leads to blood cancer. The drug aims to reduce harmful mutations in blood cells and restore normal blood production without the harsh side effects of standard cancer treatments. Participants will receive either the drug or a placebo for 24 weeks to see if it can safely prevent disease progression.
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Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
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About 80 people
The number the study aims to enrol. It can still change while the study runs.
- Expected to start
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Apr 2026
An estimate. Start dates often move.
- Expected to finish
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Oct 2030
An estimate. End dates often move.
- Lead sponsor
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A government agency
The lead sponsor is a government body.
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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60 to 85 years
- 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: 1. Age ≥ 60 and ≤ 85 years old 2. Clonal Cytopenia of Uncertain Significance (CCUS), defined by all of the following: a. Persistent cytopenia, present on at least two occasions, at least four months apart, with no other cause identified: i. Hemoglobin (Hb) \< 120 g/L in people born female, and \< 130 g/L in people born male ii. Platelet count \< 150 x 109/L iii. Absolute Neutrophil Count (ANC) \< 1.8 x109/L b. Clonal hematopoiesis (CH) driver mutation confirmed by custom gene panel mutation analysis c. absence of features diagnostic for defined myeloid neoplasm (MN) on bone marrow examination 3. CH driver mutation variant allele fraction (VAF) of ≥ 10% 4. For participants living with HIV: 1. Receiving and adherent to suppressive antiretroviral therapy for at least 12 months 2. CD4+T cell count ≥ 0.35 x 109/L 3. HIV viral load \< 50 copies/mL 6\. Performance status by Eastern Cooperative Oncology Group (ECOG) Criteria of 0 or 1 7. For participants who are of childbearing potential, or whose partners are of childbearing potential: 1. Agreement to use at least two highly effective (per Clinical Trial Facilitation Group) contraceptive methods throughout the course of the trial, and for 6 months following the last dose of trial drug 2. Refrain from donating eggs or sperm during the same period 3. Confirmation of a negative serum pregnancy test at screening and at the beginning of each treatment cycle visit (for female participants of childbearing potential) 8. Provision of signed written informed consent document prior to any trial-related assessments or procedures being carried out Exclusion Criteria: 1. ANC \< 0.5 x109/L 2. Serum AST (Aspartate transaminase) or ALT (Alanine aminotransaminase) \> 3 times of upper limit of normal 3. Calculated or measured creatinine clearance ≤ 50 mL/min 4. Significant active cardiac disease within the previous 6 months, including: 1. New York Heart Association (NYHA) class III or IV congestive heart failure 2. Unstable angina or angina requiring surgical or medical intervention 3. Myocardial infarction 4. New or unstable cardiac arrhythmia. Stable or controlled arrhythmias are permitted 5. Active systemic infections: 1. Infection with ongoing signs/symptoms related to the infection without improvement despite appropriate anti-infectives 2. Active Hepatitis B infection (HBV) (defined as HBsAg positive, or HBcAb positive and measurable HBV DNA; participants who are HBcAb positive must have HBV DNA assayed during screening) 3. Active Hepatitis C Virus (HCV) will be ineligible if there is clinical hepatic dysfunction or other systemic manifestations of HCV disease, or if the hepatic eligibility parameters above are not met. Consideration should be given to curative HCV therapy prior to enrolment in consultation with HCV clinician 6. Any history of hematological or solid malignancy in previous the 5 years unless the participant has been free of disease for ≥ 36 months. However, participants with the following history/concurrent conditions are not excluded: 1. Basal or squamous cell carcinoma of the skin 2. Carcinoma in situ of the cervix 3. Carcinoma in situ of the breast 4. Incidental histologic finding of prostate cancer (T1a or T1b using the tumor, nodes, metastasis \[TNM\] clinical staging system) 7. Known hypersensitivity to trial drugs or their constituents 8. Currently enrolled in the treatment phase of an interventional investigational trial. 9. Pregnant or breast-feeding individuals 10. Any condition not already outlined above which, in the opinion of the Principal Investigator, would place the participant at risk if they participated or would jeopardize adherence, follow up, or confound the ability to interpret trial data
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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
3 sites. 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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Canberra Health Services
Canberra, Australian Capital Territory, 2605, Australia
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Prince of Wales Hospital
Randwick, New South Wales, 2031, Australia
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Westmead Hospital
Westmead, New South Wales, 2145, Australia
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