A Phase 2 Clinical Study of Ziftomenib in Patients With Relapsed or Refractory NPM1-Mutated Acute Myeloid Leukemia

Recruiting now · Phase 2

Conditions studied: Acute Myeloid Leukemia (AML)

In brief

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

Key facts

Study ID

NCT07623616

Run by

Kyowa Kirin Co., Ltd.

People needed

6

Starts

2026-04-23

Expected to finish

2028-12-01

Last updated by the study team

2026-06-03

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• 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.
• 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.

Where it is running

• Tokyo Metropolitan Cancer and Infectious Diseases Center Komagome Hospital — Tokyo, Japan (enrolling)
• Gifu Municipal Hospital — Gifu, Japan (enrolling)
• Kansai Medical University Hospital — Osaka, Japan (enrolling)
• Osaka Metropolitan University Hospital — Osaka, Japan (enrolling)
• Dokkyo Medical University Hospital — Tochigi, Japan (enrolling)
• Jichi Medical University Hospital — Tochigi, Japan (enrolling)
• Chiba Aoba Municipal Hospital — Chiba, Japan (enrolling)
• Mito Medical Center — Ibaraki, Japan (enrolling)
• Imamura General Hospital — Kagoshima, Japan (enrolling)
• Kanagawa Cancer Center — Kanagawa, Japan (enrolling)
• Tohoku University Hospital — Miyagi, Japan (enrolling)
• Matsumoto National Hospital — Nagano, Japan (enrolling)
• Nagasaki University Hospital — Nagasaki, Japan (enrolling)
• Kurashiki Central Hospital — Okayama, Japan (enrolling)
• Okayama University Hospital — Okayama, Japan (enrolling)
• Hyogo Medical University Hospital — Hyōgo, Japan
• Nippon Medical School Hospital — Tokyo, Japan
• Jichi Medical University Saitama Medical Center — Saitama, Japan
• Keio University Hospital — Tokyo, Japan
• Kyoto University Hospital — Kyoto, Japan

Full record on ClinicalTrials.gov

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