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DNA Methyltransferase Inhibitor

Venetoclax + Azacitidine for Myelodysplastic Syndrome and Chronic Myelomonocytic Leukemia

Phase 1 & 2

Waitlist Available

Led By Guillermo Garcia-Manero

Research Sponsored by M.D. Anderson Cancer Center

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

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

Eastern Cooperative Oncology Group (ECOG)/performance status (PS) =< 2

Must not have

Patient 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

Female subject has positive results for pregnancy test

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 5 years post-treatment

Awards & highlights

All Individual Drugs Already Approved

Approved for 5 Other Conditions

No Placebo-Only Group

Summary

This trial is testing the side effects and best dose of venetoclax when given with azacitidine to treat patients with high-risk myelodysplastic syndrome or chronic myelomonocytic leukemia that has come back or has not responded to treatment.

Who is the study for?

This trial is for adults with high-risk myelodysplastic syndrome or chronic myelomonocytic leukemia that's resistant to treatment or has returned. They must have had previous unsuccessful therapy, be in good physical condition (ECOG/PS ≤2), and have proper liver and kidney function. Women of childbearing age need a negative pregnancy test and agree to contraception; men also must use birth control.

What is being tested?

The trial is testing the combination of two drugs: Venetoclax, which may block cancer cell growth enzymes, and Azacitidine, a chemotherapy drug that kills or stops cancer cells from growing. The study aims to find the best dose and see how well these drugs work together against specific blood cancers.

What are the potential side effects?

Potential side effects include nausea, vomiting, diarrhea, low blood counts leading to increased infection risk or bleeding problems, fatigue, liver issues indicated by abnormal tests results. There might also be reactions related to venetoclax such as tumor lysis syndrome.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I am a male and will use birth control during and for 3 months after the study.

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I can perform daily activities with minimal assistance.

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My kidney function is within the required range.

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My high-risk MDS did not improve after 4 HMA therapy cycles.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have a chronic illness or allergy that may affect my participation in the study.

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I am pregnant.

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My MDS is classified as low or intermediate-1 risk.

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I have previously received BCL2 inhibitor therapy.

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I cannot take medicine by mouth due to a digestive condition.

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My heart condition limits my physical activity beyond basic tasks.

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I have a serious infection that is not under control and needs treatment.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 5 years post-treatment

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 5 years post-treatment

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years post-treatment for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Maximum tolerated dose (MTD) (Phase I)

Overall response rate (ORR) (Phase II)

Secondary study objectives

Duration of response

Event-free survival

Overall survival

+6 more

Side effects data

From 2022 Phase 3 trial • 389 Patients • NCT02005471

33%

Neutropenia

11%

SARS-CoV-2 test positive

11%

Sepsis

11%

Abdominal pain

11%

Pneumonia

11%

Rhinovirus infection

11%

COVID-19

11%

Gastroenteritis

11%

Pneumonia pseudomonal

11%

Electrocardiogram QT prolonged

11%

Anaemia

11%

Neutrophil count decreased

11%

Hypokalaemia

11%

Febrile neutropenia

11%

Supraventricular tachycardia

11%

Blood creatinine increased

11%

White blood cell count decreased

11%

Dermatitis

100%

80%

60%

40%

20%

Study treatment Arm

Bendamustine + Rituximab Crossover Substudy

Venetoclax + Rituximab Re-Treatment Substudy

Venetoclax + Rituximab Main Study

Bendamustine + Rituximab Main Study

Awards & Highlights

All Individual Drugs Already Approved

Therapies where all constituent drugs have already been approved are likely to have better-understood side effect profiles.

Approved for 5 Other Conditions

This treatment demonstrated efficacy for 5 other conditions.

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: Treatment (venetoclax, azacitidine)Experimental Treatment2 Interventions

Patients receive venetoclax PO daily on days 1-14 and azacitidine IV over 15 minutes or SC on days 1-5. Cycles repeat every 4-8 weeks in the absence of disease progression or unacceptable toxicity.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Azacitidine

FDA approved

Venetoclax

FDA approved

0 / 11Eligibility criteria met