Myeloproliferative NeoplasmPhase 2NCT05074355
Study of Venetoclax and Azacitidine in Advanced BCR-ABL Negative Myeloproliferative Neoplasms
A Phase 2 study testing Azacitidine and Venetoclax in people with Myeloproliferative Neoplasm, run by University Health Network, Toronto at 1 site in Canada.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Myeloproliferative Neoplasm
- What is being tested?
- Azacitidine and Venetoclax
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 40 participants
- Where
- 1 siteCanada
- Study dates
- 8 Nov 2023to 31 Dec 2026
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
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- 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.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Canada1 site, 1 recruiting
- Recruiting
Princess Margaret Cancer Centre
Toronto, Ontario, M5G 2M9
Vikas Gupta, M.D., 416-946-2885
Vikas Gupta, M.D.
About this study
Official title: Venetoclax and Azacitidine Combination Therapy for Patients With Accelerated or Blast Phase BCR-ABL Negative Myeloproliferative Neoplasm (VAAMP)
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.
Read the 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.
- Conditions
- Myeloproliferative Neoplasm
- Being tested
- Azacitidine, Venetoclax
- Medical subject headings
- Myeloproliferative Disorders
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AzacitidineDrug
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.
VenetoclaxDrug
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 group | Role | What they receive |
|---|---|---|
| Azacitidine and Venetoclax | Experimental | 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. Receives: Drug: Azacitidine; Drug: Venetoclax |
What is being measured
The primary outcome is the main question the study is designed to answer.
Proportion of participants achieving complete remission (CR).
Measured: 3 years
Proportion of participants achieving complete remission with incomplete hematologic recovery (CRi).
Measured: 3 years
Proportion of participants achieving reversion to chronic myeloproliferative neoplasm (CMPN).
Measured: 3 years
Secondary outcomes (5)
Average number of days from the first dose of azacytidine and venetoclax to the date of death.
Measured: 3 years
Average number of days from CR until relapse.
Measured: 3 years
Average number of days from CRi until relapse.
Measured: 3 years
Average number of days from CMPN until relapse.
Measured: 3 years
The proportion of patients proceeding to allogeneic stem cell transplantation in those eligible for transplantation.
Measured: 3 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Oct 2021
- Study start8 Nov 2023
- Primary completion31 Dec 2026 (estimated)
- Study completion31 Dec 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Myeloproliferative NeoplasmStudies of the same condition, open and under way.
- More trials run by University Health Network, TorontoThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 40 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University Health Network, Toronto
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Vikas Gupta, M.D., Principal investigator, Princess Margaret Cancer Centre
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05074355
- Sponsor's protocol ID
- VAAMP
- Other IDs
- 21-5928
- Status last verified
- Jul 2026
- First submitted
- 29 Sep 2021
- Last updated
- 6 Jul 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 6 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Venetoclax and Azacitidine Combination Therapy for Patients With Accelerated or Blast Phase BCR-ABL Negative Myeloproliferative Neoplasm (VAAMP)
- ClinicalTrials.gov ID
- NCT05074355
- Acronym / sponsor ID
- VAAMP
- Conditions
- Myeloproliferative Neoplasm
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 40 participants (estimated)
- Lead sponsor
- University Health Network, TorontoUniversity, hospital or other
- Study start
- 8 Nov 2023 (actual)
- Primary completion
- 31 Dec 2026 (estimated)
- Study completion
- 31 Dec 2026 (estimated)
- First posted
- 12 Oct 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Azacitidine and Venetoclax | Experimental | Drug: Azacitidine, Drug: VenetoclaxA 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. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- 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.
Key exclusion criteria (16 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| AzacitidineAzacitidine 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 | Azacitidine and Venetoclax |
| VenetoclaxVenetoclax 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. | Drug | Azacitidine and Venetoclax |
Primary outcome
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
5 secondary outcomes
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
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Jul 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Vikas Gupta, M.D. principal investigator
Princess Margaret Cancer Centre
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Azacitidine and Venetoclax in people with Myeloproliferative Neoplasm, run by University Health Network, Toronto at 1 site in Canada.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 16 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 6 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Canada. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Vikas Gupta, M.D. (416-946-2885, vikas.gupta@uhn.ca), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Proportion of participants achieving complete remission (CR). (time frame: 3 years). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 Jul 2026.