Acute Myeloid LeukemiaPhase 1/2NCT06284486
A Multi-Site Break Through Cancer Trial: Phase II Study Investigating Dual Inhibition of BCL2 and Menin in AML MRD Using the Combination of Venetoclax and Revumenib
A Phase 1/2 study testing Venetoclax and Revumenib in people with Acute Myeloid Leukemia, run by M.D. Anderson Cancer Center at 4 sites in United States.
Trial summary
To learn if the combination of venetoclax and revumenib can help to control MRD-positive AML. Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Acute Myeloid Leukemia
- What is being tested?
- Venetoclax and Revumenib
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 8 participants
- Where
- 4 sitesUnited States
- Study dates
- 23 Sep 2024to 30 Dec 2028
- Phase
- Phase 1/2
- 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 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Age ≥ 12 years with weight ≥ 45Kg.
- ECOG performance status of ≤ 2. (if aged ≥18 years); Karnofsky Performance Scale of ≥50 (if aged ≥16 years and <18 years); Lansky Performance Score of ≥50 (if aged <16 years).
- Leukemia status:
- Known history of NPM1mt, or KMT2Ar, or NUP98r AML.
- Bone marrow assessment showing no leukemia by morphology (blasts <5%) in first remission following high intensity chemotherapy or at least 2 cycles of low intensity therapy (e.g. hypomethylating agent or low-dose cytarabine-based), or in second remission following any therapy, with MRD ≥ 0.1% identified by multiparameter flow cytometry using central lab testing.
- No clinically active extramedullary disease.
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- Baseline ejection fraction must be > 40%.
- Adequate hepatic function (direct bilirubin < 1.5x upper limit of normal (ULN) unless increase is due leukemic involvement, and AST and/or ALT < 3x ULN unless considered due to leukemic involvement, in which case direct bilirubin or AST and/or ALT < 5x ULN will be considered eligible).
- Adequate renal function with an estimated glomerular filtration rate ≥ 60 mL/min based on local institutional practice for age-appropriate determination.
- Able to swallow pills.
- Participants or parent/guardian is willing and able to provide informed consent. Interval from prior treatment to time of initiation will be at least 14 days for cytotoxic or non-cytotoxic (immunotherapy agent(s), or an interval of 5 half-lives of the prior therapy, whichever is shorter. Concurrent therapy for central nervous system (CNS) prophylaxis or continuation of therapy for controlled CNS disease is permitted.
- Women of childbearing potential must agree to adequate methods of contraception during the study and at least 3 months after the last treatment. Males must be surgically or biologically sterile or agree to use an adequate method of contraception during the study and at least 3 months after the last treatment.
Who cannot join
- Prior treatment with a menin inhibitor.
- Participants who are expected to receive standard therapy (either intensive or hypomethylating agent and venetoclax) with continued tolerability and benefit.
- Participants who are expected to be able to proceed with stem cell transplantation within the next 30 days.
- Participants with any concurrent uncontrolled medical condition, laboratory abnormality, or psychiatric illness which could place the patient at unacceptable risk of study treatment.
- The use of other chemotherapeutic agents or anti-leukemic agents is not permitted during study with the following exceptions (1) intrathecal chemotherapy for prophylactic use or for controlled CNS leukemia. (2) use of hydroxyurea for patients with rapidly proliferative disease or for control of counts during differentiation syndrome. (3) use of steroids for treatment of differentiation syndrome.
- Participants with any severe gastrointestinal or metabolic condition which could interfere with the absorption of oral study medications.
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- Participants with a concurrent active malignancy under treatment.
- Known active hepatitis B (HBV) or Hepatitis C (HCV) or HIV infection.
- Female subjects who are pregnant or breast-feeding.
- Participant has an active uncontrolled infection.
- Any of the following within the 6 months prior to study entry: myocardial infarction, uncontrolled/unstable angina, congestive heart failure (New York Heart Association Classification Class ≥II), life-threatening, uncontrolled arrhythmia, cerebrovascular accident, or transient ischemic attack.
- QTc >450 msec for males and QTc >470 msec for females using the Fridericia Formula.
- History of or any concurrent condition, therapy, or laboratory abnormality that in the Investigator's opinion might confound the results of the study, interfere with the participants's participation for the full duration of the study, or is not in the best interest of the patient to participate.
- Clinically active central nervous system (CNS) leukemia.
- Participants on immunosuppressive therapy post-HSCT at the time of screening (must be off all systemic immunosuppression therapy for at least 2 weeks and calcineurin inhibitors for at least 4 weeks). The use of topical steroids for cutaneous graft-versus-host disease (GVHD) or stable systemic steroid doses less than or equal to 20 mg of prednisone daily are permitted.
- Participants with Grade > 2 active acute GVHD, moderate or severe limited chronic GVHD, or extensive chronic GVHD of any severity.
Trial locations and contacts
4 sites in 1 country. Contact the study team or a site near you to ask about taking part.
United States4 sites, 4 recruiting
- Recruiting
Johns Hopkins University
Baltimore, Maryland, 21218
Alexander Ambinder, MD, aambind1@jhmi.edu
- Recruiting
Dana-Farber Cancer Center
Boston, Massachusetts, 02215
Jacqueline Garcia, MD, jacqueline_garcia@dfci.harvard.edu
- Recruiting
MD Anderson Cancer Center
Houston, Texas, 77030
Ghayas Issa, MD, 713-745-6798, gcissa@mdanderson.org
Ghayas Issa, MD
- Recruiting
Memorial Sloan Kettering Cancer Center
New York, New York, 10065
Aaron Goldberg, MD, 646-608-3752, goldbera@mskcc.org
Aaron Goldberg, MD
About this study
To learn if the combination of venetoclax and revumenib can help to control MRD-positive AML.
Read the detailed description
Primary Objectives
- Phase I: To determine the safety, tolerability, and recommended phase II dose (RP2D) of the combination of revumenib and venetoclax for patients with acute myeloid leukemia (AML) and detectable minimal or measurable residual disease (MRD).
- Phase II: To assess the efficacy of the combination of venetoclax and revumenib in clearance of MRD in patients with AML.
Secondary Objectives
- To assess overall survival (OS), relapse-free survival (RFS), event-free survival (EFS) and duration of response (DOR).
- To determine clinical flow and genetic MRD concordance rate
Exploratory Objectives
- To evaluate molecular and cellular markers that may be predictive of antitumor activity and/or resistance.
- To correlate MRD negativity with clinical outcomes (survival and relapse risk)
- To evaluate concordance of standard and novel MRD assays
- To explore the safety and activity of venetoclax plus revumenib in the pediatric population
- Conditions
- Acute Myeloid Leukemia
- Being tested
- Venetoclax, Revumenib
- Medical subject headings
- Leukemia, Myeloid, Acute
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VenetoclaxDrug
Also called ABT-199, GDC-0199
Given by PO
RevumenibDrug
Also called SNDX-5613
Given by PO
| Study group | Role | What they receive |
|---|---|---|
| Venetoclax + Revumenib | Experimental | Participants may receive the combination of venetoclax and revumenib for up to 1 year, and then 1 more year of venetoclax alone. You will no longer be able to take the study drug(s) if the disease gets worse or if intolerable side effects occur. Participants will take venetoclax by mouth on 1 time a day at about the same time each day, on Days 1-14 of each cycle. Take each dose with about 1 cup of water within 30 minutes after a meal, preferably breakfast. Receives: Drug: Venetoclax; Drug: Revumenib |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety and adverse events (AEs)
Incidence of Adverse Events, Graded According to National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) Version (v) 5.0
Measured: Through study completion; an average of 1 year.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Feb 2024
- Study start23 Sep 2024
- Primary completion30 Dec 2026 (estimated)
- Study completion30 Dec 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 LeukemiaStudies of the same condition, open and under way.
- More trials run by M.D. Anderson Cancer CenterThe 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 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 8 participants (estimated)
Sponsor and investigators
- Lead sponsor
- M.D. Anderson Cancer Center
- Sponsor type
- University, hospital or other
- Collaborators
- Syndax Pharmaceuticals, AbbVie
- Responsible party
- Sponsor
- Investigators
- Ghayas Issa, MD, Principal investigator, M.D. Anderson Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06284486
- Sponsor's protocol ID
- 2023-0794
- Other IDs
- NCI-2024-01721
- Status last verified
- Sep 2026
- First submitted
- 22 Feb 2024
- Last updated
- 18 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 18 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multi-Site Break Through Cancer Trial: Phase II Study Investigating Dual Inhibition of BCL2 and Menin in AML MRD Using the Combination of Venetoclax and Revumenib
- ClinicalTrials.gov ID
- NCT06284486
- Acronym / sponsor ID
- 2023-0794
- Conditions
- Acute Myeloid Leukemia
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 8 participants (estimated)
- Lead sponsor
- M.D. Anderson Cancer CenterUniversity, hospital or other
- Collaborators
- Syndax Pharmaceuticals, AbbVie
- Study start
- 23 Sep 2024 (actual)
- Primary completion
- 30 Dec 2026 (estimated)
- Study completion
- 30 Dec 2028 (estimated)
- First posted
- 29 Feb 2024 (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.
To learn if the combination of venetoclax and revumenib can help to control MRD-positive AML.
Detailed description
Primary Objectives
- Phase I: To determine the safety, tolerability, and recommended phase II dose (RP2D) of the combination of revumenib and venetoclax for patients with acute myeloid leukemia (AML) and detectable minimal or measurable residual disease (MRD).
- Phase II: To assess the efficacy of the combination of venetoclax and revumenib in clearance of MRD in patients with AML.
Secondary Objectives
- To assess overall survival (OS), relapse-free survival (RFS), event-free survival (EFS) and duration of response (DOR).
- To determine clinical flow and genetic MRD concordance rate
Exploratory Objectives
- To evaluate molecular and cellular markers that may be predictive of antitumor activity and/or resistance.
- To correlate MRD negativity with clinical outcomes (survival and relapse risk)
- To evaluate concordance of standard and novel MRD assays
- To explore the safety and activity of venetoclax plus revumenib in the pediatric population
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Venetoclax + Revumenib | Experimental | Drug: Venetoclax, Drug: RevumenibParticipants may receive the combination of venetoclax and revumenib for up to 1 year, and then 1 more year of venetoclax alone. You will no longer be able to take the study drug(s) if the disease gets worse or if intolerable side effects occur. Participants will take venetoclax by mouth on 1 time a day at about the same time each day, on Days 1-14 of each cycle. Take each dose with about 1 cup of water within 30 minutes after a meal, preferably breakfast. |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (12 listed)
- Age ≥ 12 years with weight ≥ 45Kg.
- ECOG performance status of ≤ 2. (if aged ≥18 years); Karnofsky Performance Scale of ≥50 (if aged ≥16 years and <18 years); Lansky Performance Score of ≥50 (if aged <16 years).
- Leukemia status:
- Baseline ejection fraction must be > 40%.
- Adequate hepatic function (direct bilirubin < 1.5x upper limit of normal (ULN) unless increase is due leukemic involvement, and AST and/or ALT < 3x ULN unless considered due to leukemic involvement, in which case direct bilirubin or AST and/or ALT < 5x ULN will be considered eligible).
Key exclusion criteria (16 listed)
- Prior treatment with a menin inhibitor.
- Participants who are expected to receive standard therapy (either intensive or hypomethylating agent and venetoclax) with continued tolerability and benefit.
- Participants who are expected to be able to proceed with stem cell transplantation within the next 30 days.
- Participants with any concurrent uncontrolled medical condition, laboratory abnormality, or psychiatric illness which could place the patient at unacceptable risk of study treatment.
- The use of other chemotherapeutic agents or anti-leukemic agents is not permitted during study with the following exceptions (1) intrathecal chemotherapy for prophylactic use or for controlled CNS leukemia. (2) use of hydroxyurea for patients with rapidly proliferative disease or for control of counts during differentiation syndrome. (3) use of steroids for treatment of differentiation syndrome.
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 |
|---|---|---|
| VenetoclaxAlso: ABT-199, GDC-0199Given by PO | Drug | Venetoclax + Revumenib |
| RevumenibAlso: SNDX-5613Given by PO | Drug | Venetoclax + Revumenib |
Primary outcome
Safety and adverse events (AEs)
Time frame: Through study completion; an average of 1 year.
Incidence of Adverse Events, Graded According to National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) Version (v) 5.0
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 18 Sep 2026.
- MD Anderson Cancer CenterLink provided by the sponsor.
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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.
Ghayas Issa, MD principal investigator
M.D. Anderson Cancer Center
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Venetoclax and Revumenib in people with Acute Myeloid Leukemia, run by M.D. Anderson Cancer Center at 4 sites in United States.
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 12 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 18 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in United States. 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 combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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 Ghayas Issa, MD ((713) 745-6798, gcissa@mdanderson.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety and adverse events (AEs) (time frame: Through study completion; an average of 1 year.). 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 18 Sep 2026.