Acute Myeloid Leukemia in RemissionPhase 2NCT07163793
Pilot Study of Reduced Venetoclax Exposure
A Phase 2 study testing Azacitidine, Decitabine and Venetoclax in people with Acute Myeloid Leukemia in Remission, run by Northwell Health at 1 site in United States.
Trial summary
Pilot Study of Reduced Venetoclax Exposure Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Acute Myeloid Leukemia in Remission
- What is being tested?
- Azacitidine, Decitabine and Venetoclax
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 41 participants
- Where
- 1 siteUnited States
- Study dates
- 26 Aug 2025to Feb 2028
- Phase
- Phase 2
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
- Stated willingness to comply with all study procedures and availability for the duration of the study
- Ability to take oral medication and be willing to adhere to the study regimen
- Diagnosed by current WHO or ICC criteria with Acute Myeloid Leukemia and treated for initial induction therapy with one of two regimens:
- 5-Azacitidine administered subcutaneously at a dose of 75mg/m2/day X 7 days in combination with VEN (21-28 days/cycle)
- Decitabine administered intravenously at a dose of 20mg/m2/day administered in combination with VEN (21-28 days/cycle)
- Achieving morphological CR/CRi by bone marrow biopsy with <5% blasts within 3 cycles. See Appendix 2 for definitions.
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- ECOG 0-3
- Patient must be able to understand and sign informed consent and additional study documents
- On C1D1 of trial, patient must have count recovery with ANC >1000, platelets > 50, Hemoglobin > 7.7 and without transfusion support for 7 days.
Who cannot join
- Treatment with another investigational drug
- Use of growth factor (G-CSF) within the last 14 days prior to C1D1 of trial treatment.
- Subject has received treatment prior to induction with the following:
- Prior hypomethylating agent or BCL-2 inhibitor for either AML or MDS other than for induction prior to enrollment.
- Prior CAR-T cell therapy. iii. Experimental or investigational drug therapy for 14 days prior to study entry leukemia-directed therapies.
- Subject has:
- Acute promyelocytic leukemia (APL) with t(15;17). ii. Presence of t(9;22) given the potential indication for concurrent tyrosine kinase therapy.
- Known active CNS involvement with AML.
Trial locations and contacts
1 site in 1 country.
United States1 site
Zuckerberg Cancer Center
New Hyde Park, New York, 11042
About this study
Official title: A Pilot Study of Reduced Venetoclax Exposure in Patients With Acute Myeloid Leukemia in Complete Remission
Pilot Study of Reduced Venetoclax Exposure
Read the detailed description
A pilot single-arm clinical trial is proposed to assess the primary objective: the tolerability of 14-day Venetoclax cycles in acute myeloid leukemia (AML) patients who have achieved remission and are ineligible for intensive treatment. Participants in the study will transition to a maintenance regimen that reduces the Venetoclax dosage to 14 days per cycle while continuing the hypomethylating agent (HMA) used during induction. Treatment cycles will occur every 28 days. Participants will continue treatment on study until experiencing a grade 4 cytopenic event lasting more than 7 days, an adverse event requiring regimen modification, relapse, or death.
Our primary hypothesis posits that AML patients receiving Venetoclax for 14 days per cycle will exhibit improved treatment tolerability with a reduced rate of grade 4 cytopenia compared to historical data from the VIALE A trial.
- Conditions
- Acute Myeloid Leukemia in Remission
- Being tested
- Azacitidine, Decitabine, Venetoclax
- Keywords
- AML
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AzacitidineDrug
Also called Vidaza
Given Day 1-7 with Venetoclax Day 1-14 every 28 days until off study
DecitabineDrug
Also called Dacogen
Given Day 1-5 with Venetoclax Day 1-14 every 28 days until off
VenetoclaxDrug
Also called Venclexta, Venclyxto
Venetoclax up to 400mg a day on Day 1-14 every 28 days until off in combination with Azacitidine or Decitabine
| Study group | Role | What they receive |
|---|---|---|
| Azacytadine + Venetoclax | Active comparator | Not described Receives: Drug: Azacitidine; Drug: Venetoclax |
| Decitabine + Venetoclax | Active comparator | Not described Receives: Drug: Decitabine; Drug: Venetoclax |
What is being measured
The primary outcome is the main question the study is designed to answer.
Venetoclax Tolerability Rate
Tolerability rate defined as the proportion of patients who do not experience grade 4 neutropenia (per the CTCAE v5.0) for over 7 continuous days within the first three cycles of maintenance therapy (90 days).
Patient will be evaluated on Day 1 of each cycle. Laboratory studies of prior cycle will be reviewed on that day to identify if patient had >7 continuous days of cytopenia.
Measured: 12 months
Secondary outcomes (10)
Evaluation of Event-Free Survival (EFS)
EFS: Time from treatment-to-treatment discontinuation, relapse, death of any cause, or lost to follow up (Lost to follow up defined as missing scheduled appointment with no response to 3 phone calls and 1 certified letter).
Measured: 12 months
Time to Detriment
Time to detriment defined as time to meaningful change in the EORTC-QLQ-C30. A meaningful change is defined as a decrease by at least 10 points from their baseline evaluation
The EORTC-QLQ0-C30 will be filled out by patient on Day 1 of every cycle for the first 12 cycles of maintenance therapy.
Measured: 48 months
Number of pRBC transfusion
Number of pRBC transfusion received while on trial therapy.
Number of platelet transfusion received while on trial therapy.
Number of transfusions to be described per patient year.
Data collected on Day 1 of every cycle.
Measured: 48 months
Evaluate Number of Hospital Admissions
To be evaluated at time of trial therapy discontinuation or 12 cycles of therapy, whichever occurs first.
Measured: 12 months
Rate of adverse events/toxicities
dverse events/Toxicities related to treatment.
List of adverse events/toxicities expected listed in Section 8.
Measured: 48 months
Evaluation of Relapse-Free Survival (RFS)
RFS: Time from treatment initiation to relapse, death, or lost to follow up (Lost to follow up defined as missing scheduled appointment with no response to 3 phone calls and 1 certified letter).
Measured: 12 months
Evaluation of Overall Survival (OS)
OS: Time to death or lost to follow up (Lost to follow up defined as missing scheduled appointment with no response to 3 phone calls and 1 certified letter)
Measured: 12 months
Evaluate Number of Days Admitted
To be evaluated at time of trial therapy discontinuation or 12 cycles of therapy, whichever occurs first.
Measured: 12 months
Evaluate Number of Unanticipated lab appointments
To be evaluated at time of trial therapy discontinuation or 12 cycles of therapy, whichever occurs first.
Measured: 12 months
Evaluate Number of Unanticipated clinic visits
To be evaluated at time of trial therapy discontinuation or 12 cycles of therapy, whichever occurs first.
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Sep 2025
- Study start26 Aug 2025
- Primary completionOct 2026 (estimated)
- Study completionFeb 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acute Myeloid Leukemia in RemissionStudies of the same condition, open and under way.
- 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
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 41 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Northwell Health
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Boiclair Stephanie, MD, Principal investigator, Northwell Health
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07163793
- Sponsor's protocol ID
- 24-0579
- Status last verified
- Oct 2026
- First submitted
- 7 Aug 2025
- Last updated
- 8 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No