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Cancer drug cocktail shows promise for tough blood cancers, but study halted early

NCT ID NCT04550442

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early This study
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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.

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

Phase 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

34 people

The number who actually took part.

Started

Sep 2020

Finished

May 2026

Lead sponsor

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

18 years and older

Sex

Anyone

Healthy volunteers

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

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

  • M D Anderson Cancer Center

    Houston, Texas, 77030, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.