Weekly pill cocktail aims to tame resistant blood cancers
NCT ID NCT07710534
First seen Jul 17, 2026 · Last updated Jul 17, 2026
Summary
This phase 2 trial tests whether a once-weekly oral combination of decitabine-cedazuridine and venetoclax is safer and as effective as standard therapy for people with relapsed or refractory acute myeloid leukemia, high-risk myelodysplastic syndrome, or high-risk myeloproliferative neoplasms. Participants are randomly assigned to receive either the new weekly regimen or the standard azacitidine-plus-venetoclax regimen. The study compares side effects and treatment-related complications between the two groups.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- a weekly oral combination of decitabine-cedazuridine and venetoclax
- What this could lead to
- If successful, this weekly oral regimen could offer a more convenient and less toxic treatment option for people with hard-to-treat blood cancers.
- What could go wrong
- This is an early phase 2 trial with only 40 participants, so results may not apply broadly. The combination may still cause serious side effects like low blood counts and infections.
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 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
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About 40 people
The number the study aims to enrol. It can still change while the study runs.
- Expected to start
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Sep 2026
An estimate. Start dates often move.
- Expected to finish
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Dec 2033
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 at time of enrollment * Diagnosis of one of the following by World Health Organization (WHO) International Consensus Classification (ICC) criteria as determined by local assessment: * Relapsed/ refractory acute myeloid leukemia (R/R AML) as defined by ≥5% marrow blasts or unequivocal, measurable extramedullary disease * High Risk Myelodysplastic Syndrome (HR-MDS) (high/very high risk MDS by Revised International Prognostic Scoring System (IPSS-R) or Molecular International Prognostic Scoring System (IPSS-M) * high-risk accelerated-phase myeloproliferative neoplasm (HR/AP-MPN) defined by ≥10% blasts in blood or bone marrow * Eastern Cooperative Oncology Group (ECOG) Performance status 0-3 * White blood cell (WBC) count ≤25 × 109/Liter (L) (cytoreduction with hydroxyurea or steroids is allowed to achieve this) * Aspartate Aminotransferase (AST)/ Alanine Aminotransferase (ALT) ≤3 × upper limit of normal (ULN) (≤5 × ULN if due to leukemic involvement) * Total bilirubin ≤2 × ULN (unless the elevation is due to Gilbert's or hemolysis) * Creatinine clearance ≥ 30 milliliters / minute (mL/min) * Women of child-bearing potential must not be pregnant or breastfeeding and must have a negative pregnancy test at screening. Women of non-childbearing potential are those who have had a hysterectomy or bilateral oophorectomy, or who have completed menopause (no menses for at least one year and age ≥65 or follicle-stimulating hormone levels in the menopausal range). * Subjects and their partners with reproductive potential must agree to use effective contraceptive measures during the study and for 3 months after the last dose of study treatment. Effective contraception includes methods such as oral contraceptives or double-barrier method. Exclusion Criteria: * Prior use of hypomethylating agent and venetoclax in combination (Note, use of hypomethylating agent and/or venetoclax separately in alternative combinations with other drugs is allowed) * Inability to tolerate oral therapies, or medical co-morbidities that significantly impact parenteral absorption * Acute promyelocytic leukemia myeloproliferative neoplasm (MPN) with the Philadelphia chromosome translocation (BCR:ABL) translocation * Clinically significant cardiovascular disease as defined by unstable angina * New York Heart Association class III/IV congestive heart failure * Treatment with any investigational drug or therapy within 2 weeks of study treatment or 5 half-lives before the first dose of study treatment, whichever is shorter * Known hypersensitivity to azacitidine, venetoclax, decitabine or cedazuridine * Cytotoxic chemotherapy or prior azacitidine or decitabine within 2 weeks of first dose of study treatment * Concurrent use of AML/MDS/MPN therapies including lenalidomide, erythropoietin, luspatercept, cytotoxic chemotherapies, targeted agents, etc Note: hydroxyurea is allowed in Cycle 1 if necessary for cytoreduction and/or cytarabine not exceeding a maximum dose of 1 gram per meter squared (g/m2) in Cycle 1 is also allowed for cytoreduction * Uncontrolled intercurrent illness or infection (those with controlled HIV, hepatitis, or other chronic infections are eligible) * Untreated central nervous system disease * Pregnancy or breastfeeding * Other active malignancy requiring systemic therapy during duration of trial or otherwise would confound endpoints (eg, second malignancy present where survival is expected to be less than 6 months)
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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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Virginia Commonwealth University
Richmond, Virginia, 23298, United States
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