Acute Myeloid Leukemia (AML)Phase 2NCT07623616
A Phase 2 Clinical Study of Ziftomenib in Patients With Relapsed or Refractory NPM1-Mutated Acute Myeloid Leukemia
A Phase 2 study testing ziftomenib in people with Acute Myeloid Leukemia (AML), run by Kyowa Kirin Co., Ltd. at 20 sites in Japan.
Trial summary
This is the first study to administer ziftomenib to Japanese patients. In this study, the efficacy, safety, and pharmacokinetics of ziftomenib will be evaluated in patients with relapsed or refractory NPM1-mutated acute myeloid leukemia Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Acute Myeloid Leukemia (AML)
- 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
- 6 participants
- Where
- 20 sitesJapan
- Study dates
- 23 Apr 2026to Dec 2028
- Phase
- Phase 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Voluntary written informed consent and willingness to comply with all study procedures
- Age ≥ 18 years
- Confirmed diagnosis of acute myeloid leukemia (AML)
- Patients with R/R AML with NPM1-m
- No available standard of care expected to provide clinical benefit, ineligible for or declined standard therapy.
- ECOG performance status 0-2.
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- White blood cell count ≤ 30,000/mm³ at screening (hydroxyurea permitted for cytoreduction).
- Adequate organ function according to protocol requirements.
- Women of childbearing potential must be willing to use a highly effective method of contraception throughout the study and for at least 187 days after the last dose of study treatment.
- Males with female partners of childbearing potential must agree to use a highly effective method of contraception throughout the study and for at least 97 days after the last dose of study treatment.
Who cannot join
- Diagnosis of acute promyelocytic leukemia.
- Donor lymphocyte infusion < 30 days prior to study entry.
- Clinically active central nervous system (CNS) leukemia.
- Prior hematopoietic stem cell transplantation (HSCT) without adequate hematologic recovery.
- Active Grade ≥ 2 acute graft-versus-host disease or moderate/severe chronic graft-versus-host disease.
- Prior treatment with a menin inhibitor.
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- Receipt of chemotherapy, immunotherapy, radiotherapy, or investigational therapy within 14 days or 5 half-lives prior to first dose.
- Unresolved toxicities from prior therapy > Grade 1.
- Requirement for strong CYP3A4 inducers.
- Active or uncontrolled infection, including hepatitis B, hepatitis C, or HIV.
- Conditions predisposing to serious or life-threatening infection or significant immunodeficiency.
- Cardiovascular disease or QTcF > 480 ms.
- Interstitial lung disease.
- Major surgery within 4 weeks prior to first dose.
- Women who are pregnant or lactating
- Any medical, psychiatric, or social condition that may interfere with study participation or safety, or that makes the patient unsuitable in the investigator's judgment.
Trial locations and contacts
20 sites in 1 country. Contact the study team or a site near you to ask about taking part.
Kyowa Kirin Co., Ltd. contact
20 sites in 1 country
Japan20 sites, 20 recruiting
- Recruiting
Chiba Aoba Municipal Hospital
Chiba
- Recruiting
Gifu Municipal Hospital
Gifu
- Not yet recruiting
Hyogo Medical University Hospital
Hyōgo
- Recruiting
Mito Medical Center
Ibaraki
- Recruiting
Imamura General Hospital
Kagoshima
- Recruiting
Kanagawa Cancer Center
Kanagawa
- Not yet recruiting
Kyoto University Hospital
Kyoto
- Recruiting
Tohoku University Hospital
Miyagi
- Recruiting
Matsumoto National Hospital
Nagano
- Recruiting
Nagasaki University Hospital
Nagasaki
- Recruiting
Kurashiki Central Hospital
Okayama
- Recruiting
Okayama University Hospital
Okayama
- Recruiting
Kansai Medical University Hospital
Osaka
- Recruiting
Osaka Metropolitan University Hospital
Osaka
- Not yet recruiting
Jichi Medical University Saitama Medical Center
Saitama
- Recruiting
Dokkyo Medical University Hospital
Tochigi
- Recruiting
Jichi Medical University Hospital
Tochigi
- Not yet recruiting
Keio University Hospital
Tokyo
- Not yet recruiting
Nippon Medical School Hospital
Tokyo
- Recruiting
Tokyo Metropolitan Cancer and Infectious Diseases Center Komagome Hospital
Tokyo
About this study
Official title: A Phase 2, Multicenter, Open-Label Study of Ziftomenib Monotherapy in Japanese Patients With Relapsed or Refractory Acute Myeloid Leukemia With NPM1 Mutation
This is the first study to administer ziftomenib to Japanese patients. In this study, the efficacy, safety, and pharmacokinetics of ziftomenib will be evaluated in patients with relapsed or refractory NPM1-mutated acute myeloid leukemia
- Conditions
- Acute Myeloid Leukemia (AML)
- Being tested
- ziftomenib
- 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
Oral adminitration once daily
| Study group | Role | What they receive |
|---|---|---|
| ziftomenib | Experimental | Oral adminitration once daily Receives: Drug: ziftomenib |
What is being measured
The primary outcome is the main question the study is designed to answer.
CR+CRh rate
Measured: Best overall response assessed every 28 days from first dose until disease progression or withdrawall, an average of 16weeks
Secondary outcomes (23)
MRD-negative CR+CRh (CR+CRhMRD-) rate
Measured: Best overall response assessed every 28 days from first dose until disease progression or withdrawall, an average of 16weeks
CR rate
Measured: Best overall response assessed every 28 days from first dose until disease progression or withdrawall, an average of 16weeks
MRD-negative CR rate
Measured: Best overall response assessed every 28 days from first dose until disease progression or withdrawall, an average of 16weeks
CRc (CR+ CRh + CRi) rate
Measured: Best overall response assessed every 28 days from first dose until disease progression or withdrawall, an average of 16weeks
MRD-negative CRc (CRcMRD-) rate
Measured: Best overall response assessed every 28 days from first dose until disease progression or withdrawall, an average of 16weeks
ORR (CR + CRh + CRi + MLFS + PR)
Measured: Best overall response assessed every 28 days from first dose until disease progression or withdrawall, an average of 16weeks
Transfusion independence rate
Measured: From the day after first dose through the last dose before initiation of subsequent therapy (including hematopoietic stem cell transplantation)l, an average of 16weeks
Duration of CR+CRh
Measured: Every 28 days from first dose until disease progression or withdrawall, an average of 16weeks
Time to CR+CRh
Measured: Every 28 days from first dose until disease progression or withdrawall, an average of 16weeks
Time to CR
Measured: Every 28 days from first dose until disease progression or withdrawall, an average of 16weeks
Time to CRc
Measured: Every 28 days from first dose until disease progression or withdrawall, an average of 16weeks
Time to CR, CRh, Cri, MLFS or PR
Measured: Every 28 days from first dose until disease progression or withdrawall, an average of 16weeks
EFS
Measured: Every 28 days from first dose until disease progression or withdrawall, an average of 16weeks
OS
Measured: During the treatment and every 90 days after study treatment completion (approximately up to 1 year after study treatment completion)
Incidence and severity of adverse events
Measured: During treatment and up to approximately 28 days after treatment discontinuation
Incidence of serious adverse events
Measured: During treatment and up to approximately 28 days after treatment discontinuation
Death during treatment with ziftomenib
Measured: During the treatment
Discontinuation of ziftomenib due to adverse events
Measured: During the treatment
Clinically significant changes in clinical laboratory values, vital signs, and ECG parameters
Measured: During treatment and up to end of the treatment assessment
Clinically significant decrease in ECOG PS
Measured: During treatment and up to end of the treatment assessment
Area under the plasma drug concentration time curve over a dosing interval (AUC0-τ)
AUC0-τ of ziftomenib and its metabolites
Measured: Cycle 1 Day 1, and Cycle 2 Day 1 (each cycle is 28 days)
Maximum plasma concentration (Cmax)
Cmax of ziftomenib and its metabolites
Measured: Cycle 1 Day 1, and Cycle 2 Day 1 (each cycle is 28 days)
Time to observed maximum plasma concentration (Tmax)
Tmax of ziftomenib and its metabolites
Measured: Cycle 1 Day 1, and Cycle 2 Day 1 (each cycle is 28 days)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Jun 2026
- Study start23 Apr 2026
- Primary completionMar 2027 (estimated)
- Study completionDec 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 Leukemia (AML)Studies of the same condition, open and under way.
- More trials run by Kyowa Kirin Co., Ltd.The 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 6 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Kyowa Kirin Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07623616
- Sponsor's protocol ID
- KO-MEN-J001
- Other IDs
- jRCT2031250550
- Status last verified
- May 2026
- First submitted
- 8 Apr 2026
- Last updated
- 3 Jun 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: The datasets generated and/or analyzed during the study sponsored by Kyowa Kirin will be available in the Vivli repository, https://vivli.org/ourmember/kyowa-kirin/ as long as conditions of data disclosure specified in the policy section of the Vivli website are satisfied.
Study at a glance
From the registry record, last updated by the sponsor on 3 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2, Multicenter, Open-Label Study of Ziftomenib Monotherapy in Japanese Patients With Relapsed or Refractory Acute Myeloid Leukemia With NPM1 Mutation
- ClinicalTrials.gov ID
- NCT07623616
- Acronym / sponsor ID
- KO-MEN-J001
- Conditions
- Acute Myeloid Leukemia (AML)
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 6 participants (estimated)
- Lead sponsor
- Kyowa Kirin Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- 23 Apr 2026 (actual)
- Primary completion
- Mar 2027 (estimated)
- Study completion
- Dec 2028 (estimated)
- First posted
- 3 Jun 2026 (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.
This is the first study to administer ziftomenib to Japanese patients. In this study, the efficacy, safety, and pharmacokinetics of ziftomenib will be evaluated in patients with relapsed or refractory NPM1-mutated acute myeloid leukemia
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: ziftomenibOral adminitration once daily |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- Voluntary written informed consent and willingness to comply with all study procedures
- Age ≥ 18 years
- Confirmed diagnosis of acute myeloid leukemia (AML)
- Patients with R/R AML with NPM1-m
- No available standard of care expected to provide clinical benefit, ineligible for or declined standard therapy.
Key exclusion criteria (16 listed)
- Diagnosis of acute promyelocytic leukemia.
- Donor lymphocyte infusion < 30 days prior to study entry.
- Clinically active central nervous system (CNS) leukemia.
- Prior hematopoietic stem cell transplantation (HSCT) without adequate hematologic recovery.
- Active Grade ≥ 2 acute graft-versus-host disease or moderate/severe chronic graft-versus-host disease.
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 |
|---|---|---|
| ziftomenibOral adminitration once daily | Drug | ziftomenib |
Primary outcome
CR+CRh rate
Time frame: Best overall response assessed every 28 days from first dose until disease progression or withdrawall, an average of 16weeks
23 secondary outcomes
MRD-negative CR+CRh (CR+CRhMRD-) rate
Time frame: Best overall response assessed every 28 days from first dose until disease progression or withdrawall, an average of 16weeks
CR rate
Time frame: Best overall response assessed every 28 days from first dose until disease progression or withdrawall, an average of 16weeks
MRD-negative CR rate
Time frame: Best overall response assessed every 28 days from first dose until disease progression or withdrawall, an average of 16weeks
CRc (CR+ CRh + CRi) rate
Time frame: Best overall response assessed every 28 days from first dose until disease progression or withdrawall, an average of 16weeks
MRD-negative CRc (CRcMRD-) rate
Time frame: Best overall response assessed every 28 days from first dose until disease progression or withdrawall, an average of 16weeks
ORR (CR + CRh + CRi + MLFS + PR)
Time frame: Best overall response assessed every 28 days from first dose until disease progression or withdrawall, an average of 16weeks
Transfusion independence rate
Time frame: From the day after first dose through the last dose before initiation of subsequent therapy (including hematopoietic stem cell transplantation)l, an average of 16weeks
Duration of CR+CRh
Time frame: Every 28 days from first dose until disease progression or withdrawall, an average of 16weeks
Time to CR+CRh
Time frame: Every 28 days from first dose until disease progression or withdrawall, an average of 16weeks
Time to CR
Time frame: Every 28 days from first dose until disease progression or withdrawall, an average of 16weeks
Time to CRc
Time frame: Every 28 days from first dose until disease progression or withdrawall, an average of 16weeks
Time to CR, CRh, Cri, MLFS or PR
Time frame: Every 28 days from first dose until disease progression or withdrawall, an average of 16weeks
EFS
Time frame: Every 28 days from first dose until disease progression or withdrawall, an average of 16weeks
OS
Time frame: During the treatment and every 90 days after study treatment completion (approximately up to 1 year after study treatment completion)
Incidence and severity of adverse events
Time frame: During treatment and up to approximately 28 days after treatment discontinuation
Incidence of serious adverse events
Time frame: During treatment and up to approximately 28 days after treatment discontinuation
Death during treatment with ziftomenib
Time frame: During the treatment
Discontinuation of ziftomenib due to adverse events
Time frame: During the treatment
Clinically significant changes in clinical laboratory values, vital signs, and ECG parameters
Time frame: During treatment and up to end of the treatment assessment
Clinically significant decrease in ECOG PS
Time frame: During treatment and up to end of the treatment assessment
Area under the plasma drug concentration time curve over a dosing interval (AUC0-τ)
Time frame: Cycle 1 Day 1, and Cycle 2 Day 1 (each cycle is 28 days)
Maximum plasma concentration (Cmax)
Time frame: Cycle 1 Day 1, and Cycle 2 Day 1 (each cycle is 28 days)
Time to observed maximum plasma concentration (Tmax)
Time frame: Cycle 1 Day 1, and Cycle 2 Day 1 (each cycle is 28 days)
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 3 Jun 2026.
- Individual participant dataSharing planned: Yes. The datasets generated and/or analyzed during the study sponsored by Kyowa Kirin will be available in the Vivli repository, https://vivli.org/ourmember/kyowa-kirin/ as long as conditions of data disclosure specified in the policy section of the Vivli website are satisfied.
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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.
Kyowa Kirin Co., Ltd. contact
20 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing ziftomenib in people with Acute Myeloid Leukemia (AML), run by Kyowa Kirin Co., Ltd. at 20 sites in Japan.
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 10 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 3 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 20 sites in Japan. 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 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 Kyowa Kirin, Inc. (1-609-919-1100, kkd.clintrial.82@kyowakirin.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
CR+CRh rate (time frame: Best overall response assessed every 28 days from first dose until disease progression or withdrawall, an average of 16weeks). 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 3 Jun 2026.