Cancer drug cocktail shows promise for tough blood cancers, but study halted early
NCT ID NCT04550442
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tested a combination of two drugs, venetoclax and azacitidine, in 34 adults with high-risk blood cancers (MDS or CMML) that returned or didn't respond to earlier treatment. The goal was to find the safest dose and see if the combo could shrink or control the cancer. The study was stopped early, but the results may still help doctors understand how to treat these hard-to-manage conditions.
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Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 1/2
Runs two stages together: safety and dose first, then whether the treatment works.
- Participants
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34 people
The number who actually took part.
- Started
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Sep 2020
- Finished
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May 2026
- 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: 1. Participants with post HMA-failure high-risk MDS (Int-2 or high risk by the IPSS with overall score \>/=1.5) with excess blasts \>5% with failure defined as prior receipt of 4 cycles of HMA therapy with failure to attain a response, or progression of disease or relapse at any time after prior response to HMA therapy. 2. Participants with relapsed/refractory chronic myelomonocytic leukemia (CMML) and therapy-related MDS are also eligible. 3. Hydroxyurea is allowed to lower the white cell count \</= 10,000/µl prior to initiation of venetoclax. 4. Adequate hepatic function including total bilirubin ≤ 2.0 x upper limit of normal (ULN) unless increase is due to Gilbert's disease or leukemic involvement, and ALT/AST ≤ 3.0 x ULN unless considered due to leukemic involvement. 5. Adequate renal function as calculated using the modified Cockcroft-Gault equation of ≥ 30ml/min, OR creatinine \< 2x ULN, unless related to the disease. 6. Signed written informed consent. 7. Females must be surgically or biologically sterile or postmenopausal (amenorrheic for at least 12 months) or if of childbearing potential, must have a negative serum or urine pregnancy test within 72 hours before the start of the treatment. Women of childbearing potential must agree to use an adequate method of contraception during the study and until 3 months after the last treatment. 8. Males must be surgically or biologically sterile or agree to use an adequate method of contraception during the study until 3 months after the last treatment. 9. Age \>/= 18 years of age. 10. ECOG/PS ≤2 Exclusion Criteria: 1. Participants having received any prior BCL2 inhibitor therapy. 2. Participants with MDS with IPSS risk categories Low or Int-1 (overall IPSS score \< 1.5). 3. Participants with known HIV infection (due to potential drug-drug interactions between antiretroviral medications and venetoclax). HIV testing will be performed at screening, only if required per local guidelines or institutional standards. 4. Participants known to be positive for hepatitis B or C infection \[HCV Ab indicative of a previous or current infection; and/or positive HBs Ag or detected sensitivity on HBV-DNA PCR test for HBc Ab and/or HBs Ab positivity\] with the exception of those with an undetectable viral load within 3 months of screening. (Hepatitis B or C testing is not required). Subjects with serologic evidence of prior vaccination to HBV \[i.e., HBs Ag-, and anti-HBs+\] may participate. 5. Participants has received strong and/or moderate CYP3A inducers within 7 days prior to the initiation of study treatment. 6. Participants has consumed grapefruit, grapefruit products, Seville oranges (including marmalade containing Seville oranges) or Starfruit within 3 days prior to the initiation of study treatment. 7. Participants has a cardiovascular disability status of New York Heart Association Class \> 2. Class 2 is defined as cardiac disease in which patients are comfortable at rest but ordinary physical activity results in fatigue, palpitations, dyspnea, or anginal pain. 8. Participant has chronic respiratory disease that requires continuous oxygen, or significant history of renal, neurologic, psychiatric, endocrinologic, metabolic, immunologic, hepatic, cardiovascular disease, any other medical condition or known hypersensitivity to any of the study medications including excipients of azacitidine that in the opinion of the investigator would adversely affect his/her participating in this study. 9. Participant has a malabsorption syndrome or other condition that precludes enteral route of administration. 10. Participant exhibits evidence of other clinically significant uncontrolled systemic infection requiring therapy (viral, bacterial or fungal). 11. Participant has received a live attenuated vaccine within 4 weeks prior to the first dose of study drug. 12. Participant has a history of other malignancies within 2 years prior to study entry, with the exception of: * Adequately treated in situ carcinoma of the cervix uteri or carcinoma in situ of breast; * Basal cell carcinoma of the skin or localized squamous cell carcinoma of the skin; * Previous malignancy confined and surgically resected (or treated with other modalities) with curative intent; requires discussion with TA MD. 13. Participant has a white blood cell count \> 10 × 109/L. (Hydroxyurea or leukapheresis are permitted to meet this criterion) 14. Female subject has positive results for pregnancy test. 15. Participants with (Grade \> 1) unresolved from prior treatment (including chemotherapy, targeted therapy, immunotherapy, experimental agents, radiation, or surgery)
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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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M D Anderson Cancer Center
Houston, Texas, 77030, United States
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Other studies related to the condition(s) this trial covers.
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