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Recruiting NCT05074355

Study of Venetoclax and Azacitidine in Advanced BCR-ABL Negative Myeloproliferative Neoplasms

Phase II Interventional Myeloproliferative Neoplasm

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: Azacitidine, Venetoclax.
Who it may be relevant to
Registry conditions: Myeloproliferative Neoplasm. Basic parameters: from 18 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
Canada
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

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Official title

Venetoclax and Azacitidine Combination Therapy for Patients With Accelerated or Blast Phase BCR-ABL Negative Myeloproliferative Neoplasm (VAAMP)

Overview

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.

Detailed description

All participants in this study will receive azacitidine and venetoclax.

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.

Interventions

  • Drug 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.
  • Drug 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.

Primary outcome measures

  • Proportion of participants achieving complete remission (CR). [Time frame: 3 years]
  • Proportion of participants achieving complete remission with incomplete hematologic recovery (CRi). [Time frame: 3 years]
  • Proportion of participants achieving reversion to chronic myeloproliferative neoplasm (CMPN). [Time frame: 3 years]
Secondary outcome measures (5)
  • Average number of days from the first dose of azacytidine and venetoclax to the date of death. [Time frame: 3 years]
  • Average number of days from CR until relapse. [Time frame: 3 years]
  • Average number of days from CRi until relapse. [Time frame: 3 years]
  • Average number of days from CMPN until relapse. [Time frame: 3 years]
  • The proportion of patients proceeding to allogeneic stem cell transplantation in those eligible for transplantation. [Time frame: 3 years]

Eligibility criteria

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.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
N/A
Model
Single group
Masking
Open label
Primary purpose
Treatment

Study locations

Canada · 1 center
  • Princess Margaret Cancer Centre — Toronto

Identifiers

NCT: NCT05074355 · VAAMP · 21-5928

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗