Venetoclax + Azacitidine for Myelodysplastic Syndrome
What You Need to Know Before You Apply
What is the purpose of this trial?
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.
Do I need to stop my current medications for the trial?
The trial protocol does not specify if you need to stop taking your current medications. However, hydroxyurea is allowed to lower white cell count before starting venetoclax.
What safety data exists for Venetoclax in humans?
How is the drug combination of Venetoclax and Azacitidine unique for treating myelodysplastic syndrome?
The combination of Venetoclax and Azacitidine is unique for treating myelodysplastic syndrome because it offers a new option for patients who have not responded to previous treatments, providing meaningful benefits like improved blood counts and independence from blood transfusions, which are not standard outcomes with existing therapies.678910
What data supports the effectiveness of the drug combination Venetoclax and Azacitidine for Myelodysplastic Syndrome?
Research shows that the combination of Venetoclax and Azacitidine has been effective in treating acute myeloid leukemia, improving remission rates and survival. In patients with relapsed or refractory myelodysplastic syndromes, this combination has shown promising results, including complete remission and improved overall survival.6791011
Who Is on the Research Team?
Guillermo Garcia-Manero
Principal Investigator
M.D. Anderson Cancer Center
Are You a Good Fit for This Trial?
This trial is for adults with high-risk myelodysplastic syndrome that's either come back or isn't responding to treatment. They should have normal bilirubin and creatinine levels, not be pregnant or breastfeeding, and agree to use contraception. People who've had BCL2 inhibitor therapy or have low-risk MDS aren't eligible.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive venetoclax orally once daily on days 1-7 or 1-14 and azacitidine subcutaneously or intravenously over 15 minutes on days 1-5. Cycles repeat every 4-8 weeks in the absence of disease progression or unacceptable toxicity.
Follow-up
Participants are monitored for safety and effectiveness after treatment completion, with follow-up at 30 days and then every 3-6 months for up to 5 years.
What Are the Treatments Tested in This Trial?
Interventions
- Azacitidine
- Venetoclax
Azacitidine is already approved in European Union, United States, Canada, Japan for the following indications:
- Acute myeloid leukemia
- Chronic myelomonocytic leukemia
- Myelodysplastic syndromes
- Myelodysplastic syndromes
- Chronic myelomonocytic leukemia
- Myelodysplastic syndromes
- Acute myeloid leukemia
- Myelodysplastic syndromes
- Acute myeloid leukemia
Find a Clinic Near You
Who Is Running the Clinical Trial?
M.D. Anderson Cancer Center
Lead Sponsor
National Cancer Institute (NCI)
Collaborator