Ziftomenib for Acute Myeloid Leukemia
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests a new treatment called ziftomenib for individuals with acute myeloid leukemia (AML) involving certain genetic changes. Ziftomenib blocks a protein pathway that aids cancer cell growth. The trial aims to determine if ziftomenib can effectively target and kill these cancer cells, particularly for those unable to receive standard treatments. It may suit someone with AML who has specific genetic mutations (NPM1 or KMT2A) and cannot undergo typical cancer treatments. As a Phase 2 trial, the research focuses on measuring the treatment's effectiveness in an initial, smaller group of participants.
Do I need to stop my current medications to join the trial?
The trial does not specify if you need to stop all current medications, but you cannot take drugs that strongly affect a liver enzyme called CYP3A4, except for certain antibiotics, antifungals, and antivirals. You can continue using hydroxyurea or cytarabine to control white blood cell counts.
Is there any evidence suggesting that ziftomenib is likely to be safe for humans?
Research shows that ziftomenib is generally well-tolerated by patients. In earlier studies, only a small number (3%) of patients stopped taking ziftomenib due to side effects, indicating that most patients manage the treatment well. The studies also found that side effects were not severe and could be controlled. No reports of severe differentiation syndrome, a condition associated with some cancer treatments, were noted. Overall, the safety data for ziftomenib appears promising.12345
Why do researchers think this study treatment might be promising?
Ziftomenib is unique because it targets the menin-MLL1 protein interaction, which is a novel approach in treating acute myeloid leukemia (AML). Unlike standard treatments, such as chemotherapy and targeted therapies that focus on broader cellular destruction or specific genetic mutations, Ziftomenib specifically disrupts this protein interaction, potentially leading to more effective treatment with fewer side effects. Researchers are excited about Ziftomenib because this targeted mechanism could offer a new avenue for tackling AML, especially in cases that are resistant to other treatments.
What evidence suggests that ziftomenib might be an effective treatment for acute myeloid leukemia?
Research has shown that ziftomenib effectively treats acute myeloid leukemia (AML) with certain genetic changes. In earlier studies, ziftomenib led to strong and lasting improvements in patients with NPM1-mutant AML, a specific genetic mutation in leukemia cells. It blocks the menin pathway, crucial for cancer cell growth. Another study found a 94% response rate when ziftomenib was combined with standard chemotherapy in newly diagnosed patients with NPM1-mutated and KMT2A-rearranged AML. These findings suggest that ziftomenib could be a promising treatment option for this type of leukemia. Participants in this trial will receive ziftomenib as the investigational treatment.678910
Who Is on the Research Team?
Uma M Borate, MBBS, MD, MSc
Principal Investigator
Ohio State University Comprehensive Cancer Center
Are You a Good Fit for This Trial?
This trial is for patients with acute myeloid leukemia (AML) who have specific genetic changes (NPM1 mutations or KMT2A rearrangements) and cannot receive standard therapy. It's important that participants haven't had certain treatments before, are not pregnant, and do not have other serious medical conditions.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive ziftomenib orally once daily on days 1-28 of each cycle. Cycles repeat every 28 days for up to 24 cycles in the absence of disease progression or unacceptable toxicity.
Follow-up
Participants are monitored for safety and effectiveness after treatment completion, with follow-up every 6 months for up to 24 months.
What Are the Treatments Tested in This Trial?
Interventions
- Ziftomenib
Trial Overview
The trial is testing ziftomenib, a drug designed to block the menin pathway which may be involved in AML growth due to NPM1 or KMT2A gene changes. The study includes collecting biospecimens, bone marrow procedures, heart tests, and administering additional drugs like cytarabine.
How Is the Trial Designed?
1
Treatment groups
Experimental Treatment
Patients receive ziftomenib PO QD on days 1-28 of each cycle. Cycles repeat every 28 days for up to 24 cycles in the absence of disease progression or unacceptable toxicity. Patients may also undergo cytoreduction therapy with hydroxyurea up to end of cycle 1, cytarabine within 7 days of starting treatment, or leukapheresis within 7 days of treatment to reduce white blood cell count to =\< 10,000/uL. Additionally, patients undergo ECHO or MUGA at screening and bone marrow biopsy and/or aspiration and blood sample collection throughout the study.
Find a Clinic Near You
Who Is Running the Clinical Trial?
Uma Borate
Lead Sponsor
Kura Oncology
Collaborator
Citations
Final Trial Data Show Success for Ziftomenib in NPM1 AML
Ziftomenib showed significant efficacy in relapsed/refractory NPM1-mutant AML, achieving deep, durable responses in the KOMET-001 trial. The ...
2.
physicianresources.roswellpark.org
physicianresources.roswellpark.org/news/final-landmark-clinical-trial-data-demonstrates-deep-durable-responses-to-menin-inhibition-in-acute-leukemia-patientsFinal Landmark Clinical Trial Data Demonstrates Deep ...
“Ziftomenib is a potent menin inhibitor with proven efficacy for the treatment of adult patients with NPM1-mutant acute myeloid leukemia who ...
Ziftomenib in Relapsed or Refractory NPM1-Mutated AML
In this trial, the median time to response was 1.9 months, with some patients requiring up to 3.7 months of continuous therapy to achieve ...
4.
onclive.com
onclive.com/view/ziftomenib-with-7-3-shows-early-promise-in-newly-diagnosed-npm1-mutated-kmt2a-rearranged-amlZiftomenib With 7+3 Shows Early Promise in Newly ...
Ziftomenib combined with 7+3 chemotherapy shows a 94% objective response rate in newly diagnosed NPM1-mutated and KMT2A-rearranged AML patients.
5.
ir.kuraoncology.com
ir.kuraoncology.com/news-releases/news-release-details/kura-oncology-and-kyowa-kirin-announce-presentations-ziftomenibPress Release
Ziftomenib in Combination with Venetoclax and Azacitidine in Newly Diagnosed NPM1-m Acute Myeloid Leukemia: Phase 1b Results from KOMET-007
P504: UPDATED DATA FOR ZIFTOMENIB IN PATIENTS ...
Ziftomenib continues to demonstrate significant clinical activity in heavily pretreated and co-mutated R/R NPM1m AML pts.
Ziftomenib in relapsed or refractory acute myeloid ...
Ziftomenib showed promising clinical activity with manageable toxicity in heavily pretreated patients with relapsed or refractory acute myeloid ...
Ziftomenib in Relapsed / Refractory (R/R) NPM1-mutant ...
aNo patients had Grade 4–5 differentiation syndrome. Safety & Tolerability of Ziftomenib in R/R NPM1-m AML (Safety Population). *All 3 patients were ...
NCT06001788 | Safety and Tolerability of Ziftomenib ...
Safety and Tolerability of Ziftomenib Combinations in Patients With Relapsed/Refractory Acute Myeloid Leukemia. ClinicalTrials.gov ID NCT06001788. Sponsor ...
Ziftomenib in relapsed/refractory (R/R) NPM1-mutant acute ...
Ziftomenib was well tolerated with limited myelosuppression and only 3% ziftomenib-related discontinuations. Taken together, these data support ...
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