Ziftomenib for relapsed/refractory NPM1-mutated AML in Japanese adults

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This Phase 2, interventional trial evaluates the safety, efficacy, and pharmacokinetics of ziftomenib in adults with relapsed or refractory NPM1-mutated AML who are treated in Japan. Participants must have limited or no standard therapy options and an ECOG performance status of 0–2. The study includes careful monitoring of blood counts, organ function, and potential side effects to determine how well the drug works and how it behaves in the body. Eligible patients will undergo informed consent and receive ziftomenib with predefined dosing and assessments at participating Japanese centers.

Why it matters

Who can join: the full rules

Inclusion Criteria:

* 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 Criteria:

* 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's running

• Chiba Aoba Municipal Hospital
  Chiba, Japan Recruiting
• Gifu Municipal Hospital
  Gifu, Japan Recruiting
• Mito Medical Center
  Ibaraki, Japan Recruiting
• Imamura General Hospital
  Kagoshima, Japan Recruiting
• Kanagawa Cancer Center
  Kanagawa, Japan Recruiting
• Tohoku University Hospital
  Miyagi, Japan Recruiting

Study contacts

• Study coordinator: Kyowa Kirin, Inc.
• Email: kkd.clintrial.82@kyowakirin.com
• Phone: 1-609-919-1100

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.