Study of Venetoclax and Azacitidine in Advanced BCR-ABL Negative Myeloproliferative Neoplasms
NCT ID: NCT05074355
Last Updated: 2025-12-18
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE2
40 participants
INTERVENTIONAL
2023-11-08
2026-12-31
Brief Summary
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Detailed Description
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This study will be done in multiple stages:
Safety Run-In Period - 7 participants will receive the study drugs to ensure that the combination is safe and tolerable.
Stage 1 - About 15 participants will receive the study drugs and will be evaluated to see whether they respond to the study drugs.
Stage 2 - If enough participants in Stage 1 respond to the study drugs, then Stage 2 will begin. During this stage, an additional 25 participants will take part in the study to further see if participants respond to the study drugs.
Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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Azacitidine and Venetoclax
A treatment cycle is 28 days long.
Azacitidine will be given by injection under the skin, once a day, for the first 6 days of every cycle.
Venetoclax will be given orally, once a day, as follows at the discretion of their study doctors:
Cycle 1:
* Day 1 - 100 mg
* Day 2 - 200 mg
* Days 3 to 28 - 400 mg
Cycle 2:
* Participants with a response to the study drugs will continue taking 400 mg from Days 1 to 21, with no study drug from Days 22 to 28 during Cycle 2.
* Participants who have not yet responded to the study drugs will continue taking 400 mg from Days 1 to 28 during Cycle 2.
Cycle 3 and subsequent cycles:
* Participants with a response to the study drugs will continue to take 400 mg from Days 1 to 21, with no study drug from Days 22 to 28.
* Participants whose disease has not worsened will continue taking 400 mg from Days 1 to 28.
* Participants have not responded to the study drugs will be withdrawn from the study.
Venetoclax
Venetoclax is a drug that blocks a protein called B-cell lymphoma (BCL2) protein from working. BCL2 is a protein that helps control whether a cell lives or dies and is thought to help cancer cells to live. Blocking BCL2 is believed to help kill cancer cells.
Azacitidine
Azacitidine is a hypomethylating agent that works by activating certain genes in the body to help cells mature and to kill abnormal bone marrow cells.
Interventions
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Venetoclax
Venetoclax is a drug that blocks a protein called B-cell lymphoma (BCL2) protein from working. BCL2 is a protein that helps control whether a cell lives or dies and is thought to help cancer cells to live. Blocking BCL2 is believed to help kill cancer cells.
Azacitidine
Azacitidine is a hypomethylating agent that works by activating certain genes in the body to help cells mature and to kill abnormal bone marrow cells.
Eligibility Criteria
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Inclusion Criteria
* Documented diagnosis per World Health Organization (WHO) 2016 criteria of BCR-ABL negative myeloproliferative neoplasms (MPN).
* Documented MPN transformation to accelerated phase (AP) or blast phase (BP) without prior blast reduction therapy for their AP/BP disease.
* Eastern Cooperative Oncology Group (ECOG) performance status 0-2.
* Adequate organ function.
* Must practice at least one reliable method of birth-control starting at least on cycle 1 day 1 until at least 90 days after the last dose of study drug.
* Female participants of childbearing potential must have a negative serum pregnancy test within 14 days prior to cycle 1 day 1.
Exclusion Criteria
* Previous treatment with venetoclax, navitoclax, azacytidine or other hypomethylating agents (HMA).
* White blood cell count \>25 x 10\^9/L.
* Current enrollment in another interventional study.
* Presence of any active uncontrolled infection such as bacterial or fungal infections progressing despite adequate antimicrobial treatment.
* Myocardial infarction in the preceding 3 months.
* Active human immunodeficiency virus (HIV), hepatitis B (HBV), hepatitis C (HCV) infection.
* History of active malignancy in the previous 2 years.
* Any psychiatric illness or social circumstances or significant co-morbid conditions that may compromise study participation.
* Pregnant or breastfeeding women.
* Patients with known central nervous system (CNS) involvement with acute myeloid leukemia (AML) or CNS extramedullary hematopoiesis.
* Patients with t (15;17)
* Patients who have received strong and/or moderate CYP3A inducers within 7 days prior to the initiation of study treatment.
* Active COVID-19 infection.
* History of prior blast-reduction therapy for AP/BP-MPN.
* Preceding history MDS, chronic myelomonocytic leukemia (CMML), and other myelodysplastic syndromes (MDS)/MPN overlap syndromes.
18 Years
ALL
No
Sponsors
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University Health Network, Toronto
OTHER
Responsible Party
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Principal Investigators
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Vikas Gupta, M.D.
Role: PRINCIPAL_INVESTIGATOR
Princess Margaret Cancer Centre
Locations
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Princess Margaret Cancer Centre
Toronto, Ontario, Canada
Countries
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Central Contacts
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Facility Contacts
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Vikas Gupta, M.D.
Role: primary
Other Identifiers
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21-5928
Identifier Type: OTHER
Identifier Source: secondary_id
VAAMP
Identifier Type: -
Identifier Source: org_study_id