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DNA Methyltransferase Inhibitor

Venetoclax + Azacitidine for Myelodysplastic Syndrome and Chronic Myelomonocytic Leukemia

Phase 1 & 2
Recruiting
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
Patients with relapsed/refractory chronic myelomonocytic leukemia (CMML) and therapy-related MDS are also eligible
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
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 5 years post-treatment
Awards & highlights

Study 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.Check my eligibility
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.See study design
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 have CMML or therapy-related MDS that has not responded to treatment.
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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 am taking hydroxyurea to reduce my white cell count before starting venetoclax.
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I am not pregnant and if capable of becoming pregnant, I agree to 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.
Select...
My high-risk MDS did not improve after 4 HMA therapy cycles.

Timeline

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

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Maximum tolerated dose (MTD) (Phase I)
Overall response rate (ORR) (Phase II)
Secondary outcome measures
Duration of response
Event-free survival
Overall survival
+9 more

Side effects data

From 2022 Phase 3 trial • 389 Patients • NCT02005471
33%
Neutropenia
11%
Anaemia
11%
Gastroenteritis
11%
Abdominal pain
11%
Electrocardiogram QT prolonged
11%
Febrile neutropenia
11%
Pneumonia pseudomonal
11%
Sepsis
11%
SARS-CoV-2 test positive
11%
Dermatitis
11%
Pneumonia
11%
Blood creatinine increased
11%
Rhinovirus infection
11%
White blood cell count decreased
11%
Neutrophil count decreased
11%
COVID-19
11%
Supraventricular tachycardia
11%
Hypokalaemia
100%
80%
60%
40%
20%
0%
Study treatment Arm
Bendamustine + Rituximab Crossover Substudy
Venetoclax + Rituximab Re-Treatment Substudy
Venetoclax + Rituximab Main Study
Bendamustine + Rituximab Main Study

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

Find a Location

Who is running the clinical trial?

Genentech, Inc.Industry Sponsor
1,538 Previous Clinical Trials
567,695 Total Patients Enrolled
National Cancer Institute (NCI)NIH
13,627 Previous Clinical Trials
40,927,289 Total Patients Enrolled
M.D. Anderson Cancer CenterLead Sponsor
2,962 Previous Clinical Trials
1,803,307 Total Patients Enrolled

Media Library

Azacitidine (DNA Methyltransferase Inhibitor) Clinical Trial Eligibility Overview. Trial Name: NCT04550442 — Phase 1 & 2
Chronic Myelomonocytic Leukemia Research Study Groups: Treatment (venetoclax, azacitidine)
Chronic Myelomonocytic Leukemia Clinical Trial 2023: Azacitidine Highlights & Side Effects. Trial Name: NCT04550442 — Phase 1 & 2
Azacitidine (DNA Methyltransferase Inhibitor) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04550442 — Phase 1 & 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Are we still taking participants for this experiment?

"The information available on clinicaltrials.gov indicates that this trial is currently enrolling patients. This study was originally posted on September 4th, 2020, and the most recent update was on July 21st, 2022. This trial is looking for 58 patients at 1 site."

Answered by AI

Could you please list the other studies that have used Venetoclax?

"350 clinical trials are currently underway that focus on Venetoclax. 55 of these trials have progressed to Phase 3. Most of the trials for Venetoclax are being conducted in Edmonton, Alberta, but 11240 research sites are testing this treatment."

Answered by AI

How many people are eligible to enroll in this study?

"Yes, as the latest update on clinicaltrials.gov shows, this trial is still looking for 58 patients at a single site."

Answered by AI

What are some of the things that Venetoclax has been known to help with?

"Venetoclax is a common treatment for induction chemotherapy, but can also be used to help manage refractory anemias, leukemia, myelocytic, acute, and multilineage dysplasia."

Answered by AI
~13 spots leftby May 2025