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.
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.
Study summary
The purpose of this research study is to look at how safe and useful a drug called azacitidine in combination with a drug called venetoclax, is in people with accelerated or blast phase BRC-ABL negative myeloproliferative neoplasms.
Eligibility
Sex
ALL
Min age
18 Years
Max age
—
Healthy volunteers
No
Inclusion Criteria:
* Ability to voluntarily provide written informed consent.
* 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:
* History of allogeneic stem cell transplant for MPN.
* 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.
Primary outcome measure(s)
Proportion of participants achieving complete remission (CR). — 3 years
Proportion of participants achieving complete remission with incomplete hematologic recovery (CRi). — 3 years
Proportion of participants achieving reversion to chronic myeloproliferative neoplasm (CMPN). — 3 years
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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