Ziftomenib for relapsed or hard-to-treat NPM1-mutated acute myeloid leukemia in Japan
Tests ziftomenib in Japanese adults whose NPM1-mutated acute myeloid leukemia returned or resisted treatment.
Plain-language title generated from the registry record
Registry title: A Phase 2 Clinical Study of Ziftomenib in Patients With Relapsed or Refractory NPM1-Mutated Acute Myeloid Leukemia
Key facts
- Where
- Chiba, Gifu, Hyōgo, Ibaraki, Kagoshima, Kanagawa, Kyoto, Miyagi, Nagano, Nagasaki, Okayama, Osaka, Saitama, Tochigi, and Tokyo, JapanSee the 20 sites
- Phase
- Phase 2
Where in Japan
- Chiba Aoba Municipal HospitalChiba, JapanTaking people
- Gifu Municipal HospitalGifu, JapanTaking people
- Hyogo Medical University HospitalHyōgo, JapanNot yet recruiting
- Mito Medical CenterIbaraki, JapanTaking people
- Imamura General HospitalKagoshima, JapanTaking people
- Kanagawa Cancer CenterKanagawa, JapanTaking people
- Kyoto University HospitalKyoto, JapanNot yet recruiting
- Tohoku University HospitalMiyagi, JapanTaking people
- Matsumoto National HospitalNagano, JapanTaking people
- Nagasaki University HospitalNagasaki, JapanTaking people
- Kurashiki Central HospitalOkayama, JapanTaking people
- Okayama University HospitalOkayama, JapanTaking people
- Kansai Medical University HospitalOsaka, JapanTaking people
- Osaka Metropolitan University HospitalOsaka, JapanTaking people
- Jichi Medical University Saitama Medical CenterSaitama, JapanNot yet recruiting
- Dokkyo Medical University HospitalTochigi, JapanTaking people
- Jichi Medical University HospitalTochigi, JapanTaking people
- Keio University HospitalTokyo, JapanNot yet recruiting
- Nippon Medical School HospitalTokyo, JapanNot yet recruiting
- Tokyo Metropolitan Cancer and Infectious Diseases Center Komagome HospitalTokyo, JapanTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Kyowa Kirin Co., Ltd.
- Trial code
- NCT07623616
- Record updated
- 3 June 2026
- Original description
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 leukemiaRead full original descriptionShow less
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
Inclusion: 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; 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.
Exclusion: Prior hematopoietic stem cell transplantation (HSCT) without adequate hematologic recovery; Prior treatment with a menin inhibitor; 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; Major surgery within 4 weeks prior to first dose; Active Grade ≥ 2 acute graft-versus-host disease or moderate/severe chronic graft-versus-host disease; 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; 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; Donor lymphocyte infusion < 30 days prior to study entry; Diagnosis of acute promyelocytic leukemia; Clinically active central nervous system (CNS) leukemia; Requirement for strong CYP3A4 inducers; Women who are pregnant or lactating.