Acute Myeloid LeukemiaPhase 1NCT06440135
Ziftomenib Maintenance Post Allo-HCT
A Phase 1 study testing Ziftomenib in people with Acute Myeloid Leukemia and Acute Myeloid Leukemia in Remission, run by Massachusetts General Hospital at 2 sites in United States.
Trial summary
The purpose of this study is to test the safety, effects, and recommended dose of an investigational drug, ziftomenib, in addition to the standard treatment on blood cancer with Allogeneic Hematopoietic Cell Transplantation (allo-HCT). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Acute Myeloid Leukemia, Acute Myeloid Leukemia in Remission, NPM1 Mutation
- What is being tested?
- Ziftomenib
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 22 participants
- Where
- 2 sitesUnited States
- Study dates
- 11 Jun 2024to 1 Apr 2029
- Phase
- Phase 1
- 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
- 18 years or older.
- Pathologically confirmed diagnosis of acute myeloid leukemia (AML).
- Complete remission (CR) or complete remission with incomplete count recovery (CRi) at screening.
- Complete remission (CR):
- no circulating blasts in peripheral blood and <5% blasts in bone marrow
- no extramedullary disease
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- platelet count ≥100 x 10(9)/L and/or absolute neutrophil count ≥1000/µL
- Complete remission with incomplete count recovery (CRi):
- no circulating blasts in peripheral blood and <5% blasts in bone marrow
- no extramedullary disease
- platelet count <100 x 10(9)/L and/or absolute neutrophil count <1000/µL
- Presence of at least one of the following molecular mutations:
- KMT2A rearrangement
- Eligibility and enrollment will be based on local mutational testing.
- The presence of a KMT2A rearrangement (excluding partial tandem duplication [PTD]) at the time of initial diagnosis or any other time thereafter is sufficient.
- Participants may receive additional treatment for AML between consent and transplant.
- NPM1 mutation
- Eligibility and enrollment will be based on local mutational testing.
- For participants being transplanted in CR1, the presence of a NPM1 mutation at screening is necessary for the purposes of eligibility.
- For participants being transplanted in greater than or equal to CR2, the presence of a NPM1 mutation at the time of consent is not necessary for eligibility and its presence at the time of initial diagnosis or any other time thereafter is sufficient.
- Participants may receive additional treatment for AML between consent and transplant.
- Treatment with a menin inhibitor prior to transplant is permitted. However, patients who experienced AML relapse or progression while being treated with a menin inhibitor prior to transplant are ineligible.
- Will undergo first allogeneic HCT for their malignancy.
- Transplantation will be performed with the use of conventional myeloablative (MAC) or reduced intensity conditioning (RIC).
- HCT Donor will be one of the following:
- 5/6 or 6/6 (HLA-A, B, DR) matched related donor
- 7/8 or 8/8 (HLA-A, B, DR, C) matched unrelated donor. Matching in the unrelated setting must be at the allele level.
- Haploidentical related donor, defined as ≥ 3/6 (HLA-A, B, DR) matched
- ≥ 4/6 (HLA-A, B, DR) umbilical cord blood (UCB). Matching in the UCB setting is at the antigen level. Recipients may receive either one or two UCB units. In the case of 2 UCB units, both units must have been at least 4/6 matched with the recipient.
- Any non-investigational GVHD prophylaxis regimen is allowed.
- Eastern Cooperative Oncology Group (ECOG) performance status 0-2.
- Participants must have normal organ and function as defined below:
- AST (SGOT), ALT (SGPT) and Alkaline phosphatase < 3x institutional upper limit of normal (ULN)
- Total bilirubin < 1.5 x institutional ULN (with the exception of subjects with a history of Gilbert's syndrome, for which the total bilirubin must be < 5 x ULN)
- Calculated creatinine clearance ≥ 30 mL/min (Cockcroft-Gault formula)
- LVEF must be ≥50%, as measured by MUGA scan or echocardiogram.
- Female patients of childbearing potential must have a negative pregnancy test, as measured by serum or urine testing.
- The effects of ziftomenib on the developing human fetus are unknown. For this reason women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) during the entire study treatment period and through 6 months after the last dose of treatment.
- Ability to understand and the willingness to sign a written informed consent document.
Who cannot join
- History of other malignancy(ies) unless
- the participant has been disease-free for at least 2 years and is deemed by the investigator to be at low risk of recurrence of that malignancy, or
- the cancer has been deemed indolent with no progression over the last 2 years, and deemed by the investigator to be at low risk for further progression during the course of study and follow-up
- the only prior malignancy was cervical cancer in situ and/or basal cell or squamous cell carcinoma of the skin
- Known diagnosis of active hepatitis B or hepatitis C
- Current or history of congestive heart failure New York Heart Association (NHYA) class 3 or 4, or any history of documented diastolic or systolic dysfunction (LVEF < 50%, as measured by multigated acquisition (MUGA) scan or echocardiogram)
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- Current or history of ventricular or life-threatening arrhythmias or diagnosis of long-QT syndrome
- Systemic uncontrolled infection
- Known dysphagia, short-gut syndrome, gastroparesis, or other condition(s) that limits the ingestion or gastrointestinal absorption of drugs administered orally
- Uncontrolled hypertension (systolic blood pressure [BP] > 180 mmHg or diastolic BP > 100 mmHg)
- QTc interval (i.e., Friderica's correction [QTcF]) ≥ 480 ms or other factors that increase the risk of QT prolongation or arrhythmic events (e.g., heart failure, hypokalemia, family history of long QT interval syndrome) at screening
- Uncontrolled intercurrent illness that would limit compliance with study requirements.
- Persons who are pregnant or lactating.
Trial locations and contacts
2 sites in 1 country. Contact the study team or a site near you to ask about taking part.
United States2 sites, 2 recruiting
- Recruiting
Massachusetts General Hospital
Boston, Massachusetts, 02114
Zachariah DeFilipp, MD, (617) 726-5765, zdefilipp@mgh.harvard.edu
- Recruiting
Ohio State University Wexner Medical Center- James Cancer Hospital
Columbus, Ohio, 43210
Hannah Choe, MD, 614-293-7526, Hannah.Choe@osumc.edu
Hannah Choe, MD
About this study
Official title: An Open Label Phase I Study of Ziftomenib as Maintenance Therapy Following Allogeneic Hematopoietic Cell Transplantation
The purpose of this study is to test the safety, effects, and recommended dose of an investigational drug, ziftomenib, in addition to the standard treatment on blood cancer with Allogeneic Hematopoietic Cell Transplantation (allo-HCT). This study plans to learn more about ziftomenib, which targets and inhibits negative interactions within cancer cells related to AML, when given after allo-HCT, to determine if it improves outcomes following allo-HCT.
The name of the study drug involved in this study is:
- Ziftomenib
Read the detailed description
This is a prospective, multi-center, open-label, phase I study of ziftomenib as maintenance therapy following allogeneic hematopoietic cell transplantation (HCT). This study is testing whether ziftomenib, combined with the standard allo-HCT treatment, is safe and effective in treating blood cancer. This study will test if ziftomenib improves outcomes after allo-HCT.
The study drug is given after the allo-HCT, in combination with standard treatment and aftercare.
This study consists of 2 parts:
Part A (Dose Escalation): The investigators are looking to find the highest dose of the study intervention that can be administered safely without severe or unmanageable side effects, not everyone who participates in this research study will receive the same dose of the study intervention. The dose given will depend on the number of participants who have been enrolled prior and how well the dose was tolerated. Once determined, this highest dose will then be used in the dose expansion part of the study.
Part B (Expansion Cohort): Participants will be treated at the respective dose as determined during Part A(Dose Escalation).
Ziftomenib administered after allo-HCT may work to enhance graft-versus-leukemia effects, selectively target residual leukemic cells, or suppress leukemic stem cells, among other mechanisms. The U.S. Food and DrugAdministration (FDA) has not currently approved ziftomenib as a treatment for any disease but it is being studied in Phase 1/2 interventional clinical trials for participants with relapsed or refractory acute myelogenous leukemia.
The estimated length of the study is 2 years. Participants will begin treatment 30 to 90 days after allo-HCT, and treatment will continue for up to 12 months. Then they will be followed for 12 to 24 months after study treatment ends.
It is expected that about 22 people will take part in this research study.
- Conditions
- Acute Myeloid Leukemia, Acute Myeloid Leukemia in Remission, NPM1 Mutation, KMT2A Rearrangement
- Being tested
- Ziftomenib
- Keywords
- Acute Myeloid Leukemia, Allogeneic hematopoietic cell transplantation (HCT), Allo-HCT, NPM1 Mutation, KMT2A Rearrangement
- 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.
ZiftomenibDrug
Also called KO-539
Taken orally once per day
| Study group | Role | What they receive |
|---|---|---|
| Ziftomenib | Experimental | Participants will begin treatment 30 to 90 days after allo-HCT and only after disease remission is confirmed, and treatment will continue for up to 12 months. All participants will receive ziftomenib. Participants will undergo allo-HCT as a part of their standard care.
Participants will undergo the following procedures:
* Screening
* Allo-HCT (standard care), including pre and post-treatment as a part of standard care
* 30 - 90 days after allo-HCT, participants will take the study drug orally once per day for up to 12 28-day cycles
* End of treatment visit
* Follow-up data will be collected every 3 months for 24 months from the start of treatment Receives: Drug: Ziftomenib |
What is being measured
The primary outcome is the main question the study is designed to answer.
Maximum Tolerated Dose (Dose Escalation)
Defined as the highest dose level at which 1 or 0 of 6 patients experience a Dose Limiting Toxicity (DLT). Toxicities will be graded and documented according to NCI CTCAE version 5.0.
A non-hematologic DLT is any grade 3 adverse event (AE) lasting >72 hours or any grade greater than or equal to 4 AE that is at least possibly related to the study drug with exceptions.
Any ≥ grade 2 non-hematologic toxicity that the participant finds intolerable or renders the participant unable to take 75% or more of the assigned doses (e.g. multiple dose interruptions) during the first cycle will be considered a DLT.
Measured: 28 days
Secondary outcomes (11)
Occurrence of ziftomenib-related toxicities
Defined as ziftomenib-related toxicities detected and categorized according to severity. Toxicities will be graded and documented according to NCI CTCAE version 5.0.
Measured: Day 0 to last treatment dose, up to 336 days
Incidence of acute Graft versus Host Disease (GVHD) during treatment
Defined as cumulative incidence of acute GVHD from start of ziftomenib in subjects receiving maintenance therapy after allogeneic HCT. Clinical stage and grade of acute graft-versus-host-disease (GVHD) is based on MAGIC Criteria (Harris 2016). The incidence of acute GVHD grade II-IV and grade III-IV will be estimated for each treatment group using the cumulative incidence estimate, treating death prior to acute GVHD as a competing event.
Measured: Day 0 to end of treatment visit, up to 366 days (336 days of treatment + 30 days end of treatment)
Incidence of chronic Graft versus Host Disease (GVHD) during treatment
The incidence of chronic GVHD will be estimated for each treatment group using the cumulative incidence estimate, treating death prior to chronic GVHD as a competing event. Chronic GVHD will be assessed as per the 2014 National Institutes of Health consensus development project on criteria for clinical trials in chronic graft-versus-host disease (Jagasia 2015).
Measured: Day 0 to end of treatment visit, up to 366 days (336 days of treatment + 30 days end of treatment)
Non-relapse mortality (NRM)
The incidence of non-relapse mortality will be estimated for each treatment group using the cumulative incidence estimate, treating disease relapse or progression as a competing event. AML treatment response is based on 2022 ELN recommendations (Dohner 2022).
Measured: Day 0 to end of treatment visit, up to 1086 days (336 days of treatment + 30 days end of treatment + 24 months follow-up))
Leukemia-Free Survival (LFS)
Leukemia-Free Survival is defined as the time from first dose of study drug to the earlier of leukemia relapse or death due to any cause. Participants alive and leukemia-free are censored at the date of last disease evaluation. AML treatment response is based on 2022 ELN recommendations (Dohner 2022).
Measured: Day 0 to end of treatment visit, up to 1086 days (336 days of treatment + 30 days end of treatment + 24 months follow-up))
Overall Survival (OS)
Overall Survival is defined as the time from first dose of study drug to the date of death due to any cause. Participants who are alive at the analysis / cutoff date will be censored at the last contact date.
Measured: Day 0 to end of treatment visit, up to 1086 days (336 days of treatment + 30 days end of treatment + 24 months follow-up))
GVHD-free, relapse-free survival (GRFS)
GVHD, relapse-free survival is defined as the time from first dose of study drug to the earlier of grade III-IV acute GVHD, chronic GVHD requiring systemic immune suppression, leukemia relapse or death due to any cause. Participants alive and without one of these three events are censored at the date of last disease evaluation. AML treatment response is based on 2022 ELN recommendations (Dohner 2022).
Measured: Day 0 to end of treatment visit, up to 1086 days (336 days of treatment + 30 days end of treatment + 24 months follow-up))
Proportion of successfully screened that do not reach study treatment
Defined as proportion of subjects who successfully screen for study prior to transplantation but who do not reach the maintenance phase due to transplant-related morbidity or mortality.
Measured: 30 days (Screening to Day 0)
Plasma Concentration of ziftomenib and metabolites
Characterize the pharmacokinetics (PK) of ziftomenib and metabolites when ziftomenib is given as maintenance therapy after allogeneic HCT by measuring the plasma concentration of ziftomenib and metabolites.
Measured: Up to 62 days (Cycle 1 Day 1 - Cycle 3 Day 1 (+/- 5 days)
Plasma Concentration of oral immunosuppressive agents and ziftomenib
Assess PK drug-drug interaction between ziftomenib and oral systemic immunosuppressive agents performed by measuring plasma concentrations.
Measured: Up to 34 days (Day -7 to -14 after HCT prior to co-administration and on Cycle 1 Day 1 and Day 15 (+/- 5 days) of co-administration)
Number of Participants with Treatment-Related Adverse Events
The severity/intensity of adverse events will be graded based upon the subject's symptoms according to the current active minor version of the Common Terminology Criteria for Adverse Events (CTCAE, Version 5.0). All participants will be followed and assessed for adverse events 30 days after removal from protocol therapy or until death, whichever occurs first.
Measured: Day 0 to end of treatment visit, up to 366 days (336 days of treatment + 30 days end of treatment)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Jun 2024
- Study start11 Jun 2024
- Primary completion1 Apr 2027 (estimated)
- Study completion1 Apr 2029 (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 Massachusetts General HospitalThe 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
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 22 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Massachusetts General Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Zachariah Michael DeFilipp, Massachusetts General Hospital
- Investigators
- Zachariah DeFilipp, MD, Principal investigator, Massachusetts General Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06440135
- Sponsor's protocol ID
- 24-096
- Status last verified
- Oct 2026
- First submitted
- 5 Mar 2024
- Last updated
- 6 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: The Dana-Farber / Harvard Cancer Center encourages and supports the responsible and ethical sharing of data from clinical trials. De-identified participant data from the final research dataset used in the published manuscript may only be shared under the terms of a Data Use Agreement. Requests may be directed to: Zachariah DeFilipp, MD. The protocol and statistical analysis plan will be made available on Clinicaltrials.gov only as required by federal regulation or as a condition of awards and agreements supporting the research.
Study at a glance
From the registry record, last updated by the sponsor on 6 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open Label Phase I Study of Ziftomenib as Maintenance Therapy Following Allogeneic Hematopoietic Cell Transplantation
- ClinicalTrials.gov ID
- NCT06440135
- Acronym / sponsor ID
- 24-096
- Conditions
- Acute Myeloid Leukemia, Acute Myeloid Leukemia in Remission, NPM1 Mutation, KMT2A Rearrangement
- Keywords
- Acute Myeloid Leukemia, Allogeneic hematopoietic cell transplantation (HCT), Allo-HCT, NPM1 Mutation, KMT2A Rearrangement
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 22 participants (estimated)
- Lead sponsor
- Massachusetts General HospitalUniversity, hospital or other
- Study start
- 11 Jun 2024 (actual)
- Primary completion
- 1 Apr 2027 (estimated)
- Study completion
- 1 Apr 2029 (estimated)
- First posted
- 3 Jun 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.
The purpose of this study is to test the safety, effects, and recommended dose of an investigational drug, ziftomenib, in addition to the standard treatment on blood cancer with Allogeneic Hematopoietic Cell Transplantation (allo-HCT). This study plans to learn more about ziftomenib, which targets and inhibits negative interactions within cancer cells related to AML, when given after allo-HCT, to determine if it improves outcomes following allo-HCT.
The name of the study drug involved in this study is:
- Ziftomenib
Detailed description
This is a prospective, multi-center, open-label, phase I study of ziftomenib as maintenance therapy following allogeneic hematopoietic cell transplantation (HCT). This study is testing whether ziftomenib, combined with the standard allo-HCT treatment, is safe and effective in treating blood cancer. This study will test if ziftomenib improves outcomes after allo-HCT.
The study drug is given after the allo-HCT, in combination with standard treatment and aftercare.
This study consists of 2 parts:
Part A (Dose Escalation): The investigators are looking to find the highest dose of the study intervention that can be administered safely without severe or unmanageable side effects, not everyone who participates in this research study will receive the same dose of the study intervention. The dose given will depend on the number of participants who have been enrolled prior and how well the dose was tolerated. Once determined, this highest dose will then be used in the dose expansion part of the study.
Part B (Expansion Cohort): Participants will be treated at the respective dose as determined during Part A(Dose Escalation).
Ziftomenib administered after allo-HCT may work to enhance graft-versus-leukemia effects, selectively target residual leukemic cells, or suppress leukemic stem cells, among other mechanisms. The U.S. Food and DrugAdministration (FDA) has not currently approved ziftomenib as a treatment for any disease but it is being studied in Phase 1/2 interventional clinical trials for participants with relapsed or refractory acute myelogenous leukemia.
The estimated length of the study is 2 years. Participants will begin treatment 30 to 90 days after allo-HCT, and treatment will continue for up to 12 months. Then they will be followed for 12 to 24 months after study treatment ends.
It is expected that about 22 people will take part in this research study.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Ziftomenib | Experimental | Drug: ZiftomenibParticipants will begin treatment 30 to 90 days after allo-HCT and only after disease remission is confirmed, and treatment will continue for up to 12 months. All participants will receive ziftomenib. Participants will undergo allo-HCT as a part of their standard care. Participants will undergo the following procedures: * Screening * Allo-HCT (standard care), including pre and post-treatment as a part of standard care * 30 - 90 days after allo-HCT, participants will take the study drug orally once per day for up to 12 28-day cycles * End of treatment visit * Follow-up data will be collected every 3 months for 24 months from the start of treatment |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (39 listed)
- 18 years or older.
- Pathologically confirmed diagnosis of acute myeloid leukemia (AML).
- Complete remission (CR) or complete remission with incomplete count recovery (CRi) at screening.
- Presence of at least one of the following molecular mutations:
- Treatment with a menin inhibitor prior to transplant is permitted. However, patients who experienced AML relapse or progression while being treated with a menin inhibitor prior to transplant are ineligible.
Key exclusion criteria (13 listed)
- History of other malignancy(ies) unless
- Known diagnosis of active hepatitis B or hepatitis C
- Current or history of congestive heart failure New York Heart Association (NHYA) class 3 or 4, or any history of documented diastolic or systolic dysfunction (LVEF < 50%, as measured by multigated acquisition (MUGA) scan or echocardiogram)
- Current or history of ventricular or life-threatening arrhythmias or diagnosis of long-QT syndrome
- Systemic uncontrolled infection
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 |
|---|---|---|
| ZiftomenibAlso: KO-539Taken orally once per day | Drug | Ziftomenib |
Primary outcome
Maximum Tolerated Dose (Dose Escalation)
Time frame: 28 days
Defined as the highest dose level at which 1 or 0 of 6 patients experience a Dose Limiting Toxicity (DLT). Toxicities will be graded and documented according to NCI CTCAE version 5.0. A non-hematologic DLT is any grade 3 adverse event (AE) lasting >72 hours or any grade greater than or equal to 4 AE that is at least possibly related to the study drug with exceptions. Any ≥ grade 2 non-hematologic toxicity that the participant finds intolerable or renders the participant unable to take 75% or more of the assigned doses (e.g. multiple dose interruptions) during the first cycle will be considered a DLT.
11 secondary outcomes
Occurrence of ziftomenib-related toxicities
Time frame: Day 0 to last treatment dose, up to 336 days
Incidence of acute Graft versus Host Disease (GVHD) during treatment
Time frame: Day 0 to end of treatment visit, up to 366 days (336 days of treatment + 30 days end of treatment)
Incidence of chronic Graft versus Host Disease (GVHD) during treatment
Time frame: Day 0 to end of treatment visit, up to 366 days (336 days of treatment + 30 days end of treatment)
Non-relapse mortality (NRM)
Time frame: Day 0 to end of treatment visit, up to 1086 days (336 days of treatment + 30 days end of treatment + 24 months follow-up))
Leukemia-Free Survival (LFS)
Time frame: Day 0 to end of treatment visit, up to 1086 days (336 days of treatment + 30 days end of treatment + 24 months follow-up))
Overall Survival (OS)
Time frame: Day 0 to end of treatment visit, up to 1086 days (336 days of treatment + 30 days end of treatment + 24 months follow-up))
GVHD-free, relapse-free survival (GRFS)
Time frame: Day 0 to end of treatment visit, up to 1086 days (336 days of treatment + 30 days end of treatment + 24 months follow-up))
Proportion of successfully screened that do not reach study treatment
Time frame: 30 days (Screening to Day 0)
Plasma Concentration of ziftomenib and metabolites
Time frame: Up to 62 days (Cycle 1 Day 1 - Cycle 3 Day 1 (+/- 5 days)
Plasma Concentration of oral immunosuppressive agents and ziftomenib
Time frame: Up to 34 days (Day -7 to -14 after HCT prior to co-administration and on Cycle 1 Day 1 and Day 15 (+/- 5 days) of co-administration)
Number of Participants with Treatment-Related Adverse Events
Time frame: Day 0 to end of treatment visit, up to 366 days (336 days of treatment + 30 days end of treatment)
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 Oct 2026.
- Individual participant dataSharing planned: Yes. The Dana-Farber / Harvard Cancer Center encourages and supports the responsible and ethical sharing of data from clinical trials. De-identified participant data from the final research dataset used in the published manuscript may only be shared under the terms of a Data Use Agreement. Requests may be directed to: Zachariah DeFilipp, MD. The protocol and statistical analysis plan will be made available on Clinicaltrials.gov only as required by federal regulation or as a condition of awards and agreements supporting the research.
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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.
Zachariah DeFilipp, MD principal investigator
Massachusetts General Hospital
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Ziftomenib in people with Acute Myeloid Leukemia and Acute Myeloid Leukemia in Remission, run by Massachusetts General Hospital at 2 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 39 inclusion and 13 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 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 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 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.
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 Zachariah DeFilipp, MD ((617) 726-5765, zdefilipp@mgh.harvard.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Maximum Tolerated Dose (Dose Escalation) (time frame: 28 days). 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 Oct 2026.