Venetoclax and Azacitidine for the Treatment of High-Risk Recurrent or Refractory Myelodysplastic Syndrome

Stopped early · Phase 1/Phase 2

Conditions studied: Chronic Myelomonocytic Leukemia, Myelodysplastic Syndrome, Recurrent Myelodysplastic Syndrome, Refractory Myelodysplastic Syndrome, Therapy-Related Myelodysplastic Syndrome

In brief

This phase I/II trial studies the side effects and best dose of venetoclax when given together with azacitidine in treating patients with high-risk myelodysplastic syndrome that has come back (recurrent) or does not respond to treatment (refractory). Drugs used in chemotherapy, such as venetoclax and azacitidine, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading.

Key facts

Study ID: NCT04160052
Run by: M.D. Anderson Cancer Center
People needed: 51
Starts: 2019-10-01
Expected to finish: 2026-02-03
Last updated by the study team: 2026-02-12

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

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

You may not qualify if…

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

Where it is running

M D Anderson Cancer Center — Houston, Texas, United States

Full record on ClinicalTrials.gov

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