Active — Not RecruitingPhase 1/2

Azacitidine, Venetoclax, and Pevonedistat in Treating Patients With Newly Diagnosed Acute Myeloid Leukemia

United States40 participantsStarted 2019-02-27

Plain-language summary

This phase I/II trial studies the best dose of venetoclax when given together with azacitidine and pevonedistat and to see how well it works in treating patients with newly diagnosed acute myeloid leukemia. Drugs used in chemotherapy, such as 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. Venetoclax may stop the growth of cancer cells by blocking Bcl-2, a protein needed for cancer cell survival. Pevonedistat may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Giving azacitidine, venetoclax, and pevonedistat may work better in treating patients with acute myeloid leukemia.

Who can participate

Age range

18 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * AML COHORT ONLY: Patients must have a new diagnosis (i.e., no prior therapy for AML) of AML per World Health Organization (WHO) 2016 criteria and any one of the following (i.e. therapy-related AML or AML with myelodysplastic-related changes per WHO): * A history of MDS * A history of a myeloproliferative neoplasm (MPN) including polycythemia vera (PV), essential thrombocythemia (ET), myelofibrosis (MF, whether primary \[pre-fibrotic or overt\] or post-polycythemia vera \[PV\]/essential \[E\]), chronic neutrophilic leukemia (CNL), chronic eosinophilic leukemia (CEL), MPN-unclassifiable (MPN-U) or myeloid neoplasm with a rearrangement of PDGFRA, PDGFRB or FGFR1 * A history of MDS/MPN such as chronic myelomonocytic leukemia (CMML), MDS/MPN-unclassifiable (MDS/MPN-U), MDS/MPN with ringed sideroblasts and thrombocytosis (MDS/MPN-RS-T) or atypical chronic myeloid leukemia (aCML), BCR-ABL negative * An MDS-related cytogenetic abnormality other than del9q * The presence of dysplasia in \>= 50% cells in \>= 2 myeloid lineages, unless accompanied by mutant NPM1 or biallelic CEBPA mutations * Exposure to prior chemotherapy or radiation therapy for another malignancy * NEWLY DIAGNOSED MDS/CMML COHORT ONLY: Diagnosis of MDS or CMML with intermediate-2 or high-risk disease by the International Prognostic Scoring System (IPSS) * MDS/CMML POST-HMA FAILURE COHORT ONLY: Diagnosis of MDS or CMML with intermediate-1, intermediate-2, or high-risk disease by the…

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Incidence of dose-limiting toxicities (DLTs) (Phase I)

Timeframe: Up to 28 days from treatment start date

Complete response (CR)/complete response with incomplete bone marrow recovery (CRi) rate (Phase II acute myeloid leukemia cohort)

Timeframe: Up to 168 days (6 cycles)

Complete response rate of the combination regimen (Phase II newly diagnosed myelodysplastic syndrome [MDS]/chronic myelomonocytic leukemia [CMML] cohort)

Timeframe: Up to 168 days (6 cycles)

CR + marrow CR (mCR) + partial remission (PR) + hematological improvement (HI) rate of the combination regimen (Phase II MDS/CMML post-hypomethylating agent failure cohort)

Timeframe: Up to 168 days (6 cycles)

Trial details

NCT IDNCT03862157

SponsorM.D. Anderson Cancer Center

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2027-01-31

View on ClinicalTrials.gov More Acute Myeloid Leukemia trials

Plain-language summary

Who can participate

Age range

18 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

What they're measuring

Incidence of dose-limiting toxicities (DLTs) (Phase I)

Timeframe: Up to 28 days from treatment start date

Complete response (CR)/complete response with incomplete bone marrow recovery (CRi) rate (Phase II acute myeloid leukemia cohort)

Timeframe: Up to 168 days (6 cycles)

Complete response rate of the combination regimen (Phase II newly diagnosed myelodysplastic syndrome [MDS]/chronic myelomonocytic leukemia [CMML] cohort)

Timeframe: Up to 168 days (6 cycles)

CR + marrow CR (mCR) + partial remission (PR) + hematological improvement (HI) rate of the combination regimen (Phase II MDS/CMML post-hypomethylating agent failure cohort)

Timeframe: Up to 168 days (6 cycles)