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

NCT07623616 | Recruiting Phase 2

• 2 other identifiers: KO-MEN-J001jRCT2031250550
• Study Type: interventional
• Target: 6
• Locations: 1 country
• Sites: 20

Brief 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

Conditions

Acute Myeloid Leukemia (AML)

Outcome Measures

Primary Outcomes (1)

• CR+CRh rate

  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

  Best overall response assessed every 28 days from first dose until disease progression or withdrawall, an average of 16weeks

• CR rate

  Best overall response assessed every 28 days from first dose until disease progression or withdrawall, an average of 16weeks

• MRD-negative CR rate

  Best overall response assessed every 28 days from first dose until disease progression or withdrawall, an average of 16weeks

• CRc (CR+ CRh + CRi) rate

  Best overall response assessed every 28 days from first dose until disease progression or withdrawall, an average of 16weeks

• MRD-negative CRc (CRcMRD-) rate

  Best overall response assessed every 28 days from first dose until disease progression or withdrawall, an average of 16weeks

Study Arms (1)

ziftomenib

EXPERIMENTAL

Oral adminitration once daily

Drug: ziftomenib

Interventions

ziftomenib DRUG

Oral adminitration once daily

ziftomenib

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

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

Sponsors & Collaborators

• Kyowa Kirin Co., Ltd.: lead

Study Sites (20)

Chiba Aoba Municipal Hospital

Chiba, Japan

RECRUITING

Gifu Municipal Hospital

Gifu, Japan

RECRUITING

Hyogo Medical University Hospital

Hyōgo, Japan

NOT YET RECRUITING

Mito Medical Center

Ibaraki, Japan

RECRUITING

Imamura General Hospital

Kagoshima, Japan

RECRUITING

Kanagawa Cancer Center

Kanagawa, Japan

RECRUITING

Kyoto University Hospital

Kyoto, Japan

NOT YET RECRUITING

Tohoku University Hospital

Miyagi, Japan

RECRUITING

Matsumoto National Hospital

Nagano, Japan

RECRUITING

Nagasaki University Hospital

Nagasaki, Japan

RECRUITING

Kurashiki Central Hospital

Okayama, Japan

RECRUITING

Okayama University Hospital

Okayama, Japan

RECRUITING

Kansai Medical University Hospital

Osaka, Japan

RECRUITING

Osaka Metropolitan University Hospital

Osaka, Japan

RECRUITING

Jichi Medical University Saitama Medical Center

Saitama, Japan

NOT YET RECRUITING

Dokkyo Medical University Hospital

Tochigi, Japan

RECRUITING

Jichi Medical University Hospital

Tochigi, Japan

RECRUITING

Keio University Hospital

Tokyo, Japan

NOT YET RECRUITING

Nippon Medical School Hospital

Tokyo, Japan

NOT YET RECRUITING

Tokyo Metropolitan Cancer and Infectious Diseases Center Komagome Hospital

Tokyo, Japan

RECRUITING

MeSH Terms

Conditions

Leukemia, Myeloid, Acute

Condition Hierarchy (Ancestors)

Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases

Central Study Contacts

Kyowa Kirin, Inc.

CONTACT

1-609-919-1100; kkd.clintrial.82@kyowakirin.com

Kyowa Kirin Co., Ltd.

CONTACT

clinical.info.jp@kyowakirin.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 8, 2026
• First Posted: June 3, 2026
• Study Start: April 23, 2026
• Primary Completion (Estimated): March 1, 2027
• Study Completion (Estimated): December 1, 2028
• Last Updated: June 3, 2026

Record last verified: 2026-05

Data Sharing

• IPD Sharing: Will share

Locations

JP (20)