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Nucleoside analogue

Venetoclax + Azacitidine for Myelodysplastic Syndrome

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
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
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
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 5 years
Awards & highlights

Study Summary

This trial is testing the combination of venetoclax and azacitidine to see if it is effective and has manageable side effects in treating patients with high-risk myelodysplastic syndrome.

Who is the study for?
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.Check my eligibility
What is being tested?
The trial is testing the combination of two chemotherapy drugs, Venetoclax and Azacitidine, to find the safest dose and see how well they work together in treating patients with aggressive forms of myelodysplastic syndrome.See study design
What are the potential side effects?
Venetoclax and Azacitidine can cause side effects like killing healthy cells along with cancer cells which might lead to fatigue, infection risk increase due to low blood cell counts, bleeding issues from low platelets, nausea, vomiting, constipation or diarrhea.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I am not pregnant and if I can have children, I agree to use birth control during and 3 months after the study.
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I have high-risk MDS or MDS that didn't respond to previous treatment.
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I am a male and will use birth control during and for 3 months after the study.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 5 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Incidence of adverse events (Phase I)
Maximum tolerated dose (MTD) of the combination regimen of venetoclax and azacitidine (Phase I)
Secondary outcome measures
Duration of response
Event-free survival
Overall response rate (Phase 2)
+11 more
Other outcome measures
Biological markers of response
Effects of therapy on myelodysplastic syndrome

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 orally PO QD on days 1-7 or 1-14 and azacitidine SC or IV over 15 minutes 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
Venetoclax
2019
Completed Phase 3
~1990
Azacitidine
2012
Completed Phase 3
~1440

Find a Location

Who is running the clinical trial?

National Cancer Institute (NCI)NIH
13,627 Previous Clinical Trials
40,927,225 Total Patients Enrolled
M.D. Anderson Cancer CenterLead Sponsor
2,962 Previous Clinical Trials
1,803,249 Total Patients Enrolled
Guillermo Garcia-ManeroPrincipal InvestigatorM.D. Anderson Cancer Center
9 Previous Clinical Trials
1,442 Total Patients Enrolled

Media Library

Azacitidine (Nucleoside analogue) Clinical Trial Eligibility Overview. Trial Name: NCT04160052 — Phase 1 & 2
Myelodysplastic Syndrome Research Study Groups: Treatment (venetoclax, azacitidine)
Myelodysplastic Syndrome Clinical Trial 2023: Azacitidine Highlights & Side Effects. Trial Name: NCT04160052 — Phase 1 & 2
Azacitidine (Nucleoside analogue) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04160052 — Phase 1 & 2

Frequently Asked Questions

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

Is this research currently enrolling participants?

"As per clinicaltrials.gov, this research project is presently recruiting participants. It was first posted on October 1st 2019 and last modified on June 8th 2022."

Answered by AI

In what other capacities has Venetoclax been examined?

"At present, 340 Venetoclax trials are active with 54 in Phase 3. Toronto is one of the prime locations for these studies; however, there exist 11,189 clinical trial sites that conduct research on this medication."

Answered by AI

How many individuals are currently participating in this trial's proceedings?

"Absolutely. The clinicaltrials.gov website attests to the fact that this trial is currently recruiting individuals, as it was initially posted on October 1st 2019 and recently updated on June 8th 2022. 116 patients are needed from one particular medical site for successful completion of the experiment."

Answered by AI

To what maladies is Venetoclax typically prescribed as a remedy?

"Venetoclax is regularly used for induction chemotherapy, yet can also be beneficial in alleviating the symptoms associated with refractory anemias, leukemia, myelocytic and acute multilineage dysplasia."

Answered by AI
~17 spots leftby Dec 2024