Actively Recruiting
A Phase 2, Multicenter, Open-Label Study of Ziftomenib Monotherapy in Japanese Patients With Relapsed or Refractory Acute Myeloid Leukemia With NPM1 Mutation
Led by Kyowa Kirin Co., Ltd. · Updated on 2026-06-03
6
Participants Needed
20
Research Sites
91 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating ziftomenib in Japanese patients with relapsed or refractory acute myeloid leukemia (AML) that has an NPM1 mutation. This Phase 2 study aims to assess the drug's effectiveness, safety, and how it behaves in the body. It is the first trial to administer ziftomenib to this patient group in Japan, with the goal of understanding its potential benefits for patients who have limited treatment options. Participants will take ziftomenib orally once daily. The study will monitor responses every 28 days, continuing treatment until the disease progresses or the patient withdraws. Several secondary measures will assess deeper remission rates, transfusion needs, and duration of response. The study also includes detailed monitoring of adverse events, drug levels in the blood, and patient health indicators during treatment and after discontinuation. During the trial, participants will have regular assessments including disease response evaluations every 28 days and safety checks. Follow-up will continue for up to one year after treatment ends to track overall survival and late effects. Participants will be monitored for side effects, changes in laboratory values, heart function, and overall physical status. The study duration varies but includes ongoing treatment and post-treatment observation to gather comprehensive data on ziftomenib's impact.
CONDITIONS
Brief Title
A Phase 2 Clinical Study of Ziftomenib in Patients With Relapsed or Refractory NPM1-Mutated Acute Myeloid Leukemia
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Voluntary written informed consent and willingness to comply with all study procedures
- Age 18 years or older
- Confirmed diagnosis of acute myeloid leukemia (AML)
- Relapsed or refractory AML with NPM1 mutation
- No standard care available expected to provide clinical benefit, or ineligible for or declined standard therapy
- ECOG performance status of 0 to 2
- White blood cell count of 30,000/mm³ or less at screening (hydroxyurea allowed for cytoreduction)
- Adequate organ function as required by protocol
- Women of childbearing potential must agree to use highly effective contraception during the study and for at least 187 days after the last dose
- Males with female partners of childbearing potential must agree to use highly effective contraception during the study and for at least 97 days after the last dose
You will not qualify if you...
- Diagnosis of acute promyelocytic leukemia
- Donor lymphocyte infusion within 30 days before study entry
- Active central nervous system leukemia
- Prior hematopoietic stem cell transplant without adequate blood recovery
- Active Grade 2 or higher acute graft-versus-host disease or moderate/severe chronic graft-versus-host disease
- Previous treatment with a menin inhibitor
- Chemotherapy, immunotherapy, radiotherapy, or investigational therapy within 14 days or 5 half-lives before first dose
- Unresolved side effects from prior therapy greater than Grade 1
- Need for strong CYP3A4 inducers
- Active or uncontrolled infection including hepatitis B, hepatitis C, or HIV
- Conditions causing serious infection risk or significant immune deficiency
- Cardiovascular disease or QTcF interval over 480 ms
- Interstitial lung disease
- Major surgery within 4 weeks before first dose
- Pregnant or breastfeeding women
- Any medical, psychiatric, or social condition interfering with participation or safety as judged by investigators
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
Duration - Repeated 28-day cycles until disease progression or withdrawal, averaging 16 weeks
Participants receive oral ziftomenib once daily to treat relapsed or refractory acute myeloid leukemia with NPM1 mutation.
Visits every 28 days for assessment
Duration - Up to 1 year after study treatment completion
Participants are monitored for safety and overall survival after treatment completion.
Visits every 90 days after treatment completion
Trial Site Locations
Total: 20 locations
1
Chiba Aoba Municipal Hospital
Chiba, Japan
Actively Recruiting
2
Gifu Municipal Hospital
Gifu, Japan
Actively Recruiting
3
Hyogo Medical University Hospital
Hyōgo, Japan
Not Yet Recruiting
4
Mito Medical Center
Ibaraki, Japan
Actively Recruiting
5
Imamura General Hospital
Kagoshima, Japan
Actively Recruiting
6
Kanagawa Cancer Center
Kanagawa, Japan
Actively Recruiting
7
Kyoto University Hospital
Kyoto, Japan
Not Yet Recruiting
8
Tohoku University Hospital
Miyagi, Japan
Actively Recruiting
9
Matsumoto National Hospital
Nagano, Japan
Actively Recruiting
10
Nagasaki University Hospital
Nagasaki, Japan
Actively Recruiting
11
Kurashiki Central Hospital
Okayama, Japan
Actively Recruiting
12
Okayama University Hospital
Okayama, Japan
Actively Recruiting
13
Kansai Medical University Hospital
Osaka, Japan
Actively Recruiting
14
Osaka Metropolitan University Hospital
Osaka, Japan
Actively Recruiting
15
Jichi Medical University Saitama Medical Center
Saitama, Japan
Not Yet Recruiting
16
Dokkyo Medical University Hospital
Tochigi, Japan
Actively Recruiting
17
Jichi Medical University Hospital
Tochigi, Japan
Actively Recruiting
18
Keio University Hospital
Tokyo, Japan
Not Yet Recruiting
19
Nippon Medical School Hospital
Tokyo, Japan
Not Yet Recruiting
20
Tokyo Metropolitan Cancer and Infectious Diseases Center Komagome Hospital
Tokyo, Japan
Actively Recruiting
Research Team
K
Kyowa Kirin, Inc.
K
Kyowa Kirin Co., Ltd.
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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