Promising combo for tough blood cancer hits snag, study stopped early
NCT ID NCT04160052
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tested a combination of two drugs, venetoclax and azacitidine, for people with a high-risk type of bone marrow cancer called myelodysplastic syndrome (MDS) that either came back or didn't respond to treatment. The goal was to find the best dose and see how well it worked. The study was stopped early, so results are limited, but it aimed to control the disease rather than cure it.
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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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51 people
The number who actually took part.
- Started
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Oct 2019
- Finished
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Feb 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: * For phase I, patients can be HMA-naive high-risk MDS (Int-2 or high risk by the International Prognostic Scoring System \[IPSS\] with overall score \>= 1.5) with excess blasts \> 5%, or relapsed/refractory MDS post-HMA 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) with \> 5% blasts * For phase II, patients will be divided into 2 cohorts: Cohort A: patients with HMA-naive high-risk MDS (Int-2 or high risk by the IPSS with overall score \>= 1.5) with excess blasts \> 5%. Cohort B: patients with relapsed/refractory MDS post-HMA 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) with \> 5% blasts are eligible. Note: Patients with chronic myelomonocytic leukemia (CMML) and therapy-related MDS are eligible. Hydroxyurea is allowed to lower the white cell count =\< 10,000/ul prior to initiation of venetoclax * Total bilirubin \< 3 x upper limit of normal (ULN) unless increase is due to Gilbert's disease or leukemic involvement * Alanine aminotransferase (ALT) \< 4 x ULN unless considered due to leukemic involvement * Creatinine \< 2 x ULN unless related to the disease * Signed written informed consent. Consent may be translated for Non-English Speaking Patients per institutional policy. * 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 * 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 Exclusion Criteria: * Patients having received any prior BCL2 inhibitor therapy * Patients with MDS with IPSS risk categories low or Int-1 (overall IPSS score \< 1.5) * Pregnant or breastfeeding * Cognitively impaired patients
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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
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Can a Platelet-Boosting drug help control a rare bone marrow disorder?
- Can adding venetoclax make donor stem cell transplants safer for High-Risk blood cancers?
- Tweaking donor cells may shield older transplant patients from a dangerous complication
- Can a drug and donor cells stop leukemia from returning after transplant?
- New drug combination targets Hard-to-Treat blood cancers
- Can a calming drug make cord blood transplants safer?