Ziftomenib Maintenance Post Allo-HCT
An Open Label Phase I Study of Ziftomenib as Maintenance Therapy Following Allogeneic Hematopoietic Cell Transplantation
Who can join
As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.
- Age
- 18 Years and older
- Sex
- All sexes
- Healthy volunteers
- Not accepted
- Compensation
- Not stated
Other requirements are listed under Eligibility below. Only the study team can confirm whether you can join.
What to expect
No one can promise a trial will help you personally — that is what the research is trying to find out.
What participation involves
- Time periods mentioned: 12 months
Only what the study's summary and detailed description on ClinicalTrials.gov say. Time periods may describe the whole study or a part of it — ask the study team what applies to you.
Show the study's own words
- "Participants will begin treatment 30 to 90 days after allo-HCT, and treatment will continue for up to 12 months."
- Compensation
- Not stated
- Study-related exams and procedures are typically provided at no cost. This is how many trials work in general — confirm the details for this study with the study team.
- Participants are monitored closely by the research team throughout the study.
- Some participants may receive access to an investigational treatment, which is still being studied.
- Your participation helps researchers learn whether this approach could help future patients.
About this study
In plain language
This study tests ziftomenib, an experimental drug, added to standard care after a donor stem cell transplant. It is for adults with acute myeloid leukemia (AML), a blood cancer, with certain gene changes. Researchers will study its safety, find a recommended dose, and see if it improves results after transplant.
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
Compensation is read from the study's own description and may be missing — always confirm with the study team.
Eligibility
Who can join
- Be age 18 or older with AML confirmed by lab testing.
- Be in remission: no leukemia cells called blasts in the blood, less than 5% blasts in bone marrow, and no leukemia outside the marrow. Blood cell counts may still be low.
- Have a qualifying KMT2A or NPM1 gene change. The required timing of the gene test depends on the gene change and your remission history.
- Plan to have your first donor stem cell transplant, with an allowed donor match and either full-strength or lower-strength treatment to prepare for it.
- Be able to care for yourself and be out of bed for more than half of waking hours.
- Meet liver and kidney test limits. Your heart must pump out at least 50% of the blood it holds with each beat.
- If able to become pregnant, have a negative pregnancy test. Women who can become pregnant and men must agree to use birth control or avoid sex during treatment and for 6 months afterward.
- Be able to understand the study and sign a consent form.
Who can't join
- AML came back or worsened while taking a menin inhibitor, a type of cancer drug, before transplant.
- Have a history of another cancer, unless it meets the study's rules for low-risk or stable cancer. Certain early cervical cancers and skin cancers are allowed.
- Have active hepatitis B or C, or an infection affecting the body that is not controlled.
- Have current or past severe heart failure, reduced heart pumping or filling function, or certain dangerous heart rhythms.
- Have long-QT syndrome, an abnormal heart timing test, or other factors that raise the risk of dangerous heart rhythms.
- Have trouble swallowing or a gut condition that limits taking in or absorbing pills.
- Have uncontrolled blood pressure above 180 for the top number or 100 for the bottom number, or another uncontrolled illness that would prevent following study rules.
- Be pregnant or breastfeeding.
Simplified for readability. The official criteria below are what the study team uses.
Official eligibility criteria
Inclusion Criteria: * 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 * 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. Exclusion Criteria: * 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) * 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.
Study sites (2)
MA
- Massachusetts General Hospital · Boston, MA 02114Zachariah DeFilipp, MD · Study contact · (617) 726-5765 · zdefilipp@mgh.harvard.eduMore trials in Boston
OH
- Ohio State University Wexner Medical Center- James Cancer Hospital · Columbus, OH 43210Hannah Choe, MD · Study contact · 614-293-7526 · Hannah.Choe@osumc.eduHannah Choe, MD · Principal investigatorMore trials in Columbus