Study on Venetoclax and Azacitidine for Treating High-Risk Myelodysplastic Syndrome That Has Returned or Is Not Responding to Treatment

Sponsor: M.D. Anderson Cancer Center

Sponsor score: 0
Inactive

ClinicalTrials ID:NCT04160052

Provider View

This clinical trial is testing the safety and best dose of a combination of two drugs, venetoclax and azacitidine, for patients with high-risk myelodysplastic syndrome (MDS) that has come back or is not responding to treatment. These drugs work by stopping cancer cells from growing and spreading.

Patient Parameters

Program Overview

The purpose of this study is to find out the side effects and best dose of venetoclax when used with azacitidine in patients with high-risk myelodysplastic syndrome (MDS) that has returned or is not responding to treatment. Venetoclax and azacitidine are chemotherapy drugs that work in different ways to stop cancer cells from growing, dividing, or spreading.

Description

This study has two main parts: Phase I and Phase II.

In Phase I, the focus is on determining the safety and tolerability of the drug combination in patients with high-risk myelodysplastic syndrome (MDS) or chronic myelomonocytic leukemia (CMML) who have more than 5% excess blasts in their bone marrow and have not responded to previous treatments.

In Phase II, the study will look at how well the treatment works, including the overall response rate, complete remission rate, and improvements in blood cell counts. It will also assess how long patients remain free from needing red blood cell or platelet transfusions, the response of bone marrow blasts, and the time it takes for the disease to progress to acute myeloid leukemia (AML).

The study will also explore the effects of the treatment on MDS and identify biological markers that may predict how well patients respond to the treatment.

Participants will take venetoclax by mouth daily for 7 or 14 days and receive azacitidine either under the skin or through an IV for 5 days. Treatment cycles repeat every 4-8 weeks unless the disease progresses or side effects become unacceptable.

After finishing the study treatment, patients will have follow-up visits at 30 days and then every 3-6 months for up to 5 years.

Eligibility Criteria

Inclusion Criteria

  • For Phase I, patients must have high-risk MDS with more than 5% excess blasts and have not responded to previous treatments, or have relapsed/refractory MDS after failing previous therapy.
  • For Phase II, patients will be divided into two groups: those with high-risk MDS who have not received previous treatment, and those with relapsed/refractory MDS after previous therapy.
  • Patients with chronic myelomonocytic leukemia (CMML) and therapy-related MDS are eligible.
  • Blood tests must show bilirubin levels less than 3 times the normal limit, unless due to specific conditions.
  • Liver enzyme levels must be less than 4 times the normal limit, unless related to the disease.
  • Kidney function must be within 2 times the normal limit, unless related to the disease.
  • Must provide signed informed consent.
  • Women must not be pregnant and must use effective birth control during the study and for 3 months after.
  • Men must use effective birth control during the study and for 3 months after.

Exclusion Criteria

  • Patients who have received any prior BCL2 inhibitor therapy.
  • Patients with low or intermediate-1 risk MDS.
  • Pregnant or breastfeeding women.
  • Patients with cognitive impairments.

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