Clinical Trials List
2025-12-19 - 2031-11-30
Phase III
Not yet recruiting1
Recruiting4
Phase 3 Randomized, Double-blind, Placebo-controlled Studies Assessing Ziftomenib in Combination With Either Standard of Care Nonintensive (Venetoclax+Azacitidine) or Intensive (7+3) Therapy in Patients With Untreated NPM1 Mutated or KMT2A Rearranged Acute Myeloid Leukemia
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Trial Applicant
Pharmaceutical Research Associates Taiwan Inc.
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Sponsor
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Trial scale
Multi-Regional Multi-Center
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Update
2026/07/02
Investigators and Locations
Co-Principal Investigator
- Wen-Chien Chou 無
- MING YAO 無
- HSIN-AN HOU 無
- Huai-Hsuan Huang 無
- Chien-Chin Lin 無
- Chieh-Lung Cheng 無
- 田豐銘 無
- YAO CHI-YUAN 無
- 李思慧 無
- 楊鎰聰 無
The Actual Total Number of Participants Enrolled
0 Not yet recruiting
Co-Principal Investigator
- Tsung -Chih Chen 無
- CHENG-HSIEN LIN 無
- 滕傑林 無
- PO-WEI LIAO 無
- 鄧齡喬 無
The Actual Total Number of Participants Enrolled
0 Recruiting
Co-Principal Investigator
- Ta-Chih Liu 無
- 吳敬炫 無
The Actual Total Number of Participants Enrolled
0 Recruiting
Co-Principal Investigator
The Actual Total Number of Participants Enrolled
0 Recruiting
Co-Principal Investigator
The Actual Total Number of Participants Enrolled
0 Recruiting
Condition/Disease
Objectives
Test Drug
Active Ingredient
Dosage Form
Dosage
Endpoints
Primary endpoint:
• Overall survival (OS)
• CR rate as assessed by the trial administrator according to the European Leukemia Network (ELN) 2022 criteria
Intensive treatment trial (7+3)
Primary endpoint:
• EFS, defined as the time elapsed from randomization to treatment failure, hematological relapse after CR, or death from any cause, whichever occurs first
• Percentage of patients who achieved CR according to the ELN 2022 criteria and exhibited central laboratory-defined BM MRD negativity (CRMRD-) at the end of 2 treatment cycles
Inclution Criteria
The following criteria apply to both the Nonintensive Therapy Study and the Intensive Therapy Study unless otherwise noted:
Age ≥18 years at time of signing the informed consent form.
Diagnosis of AML per the 2022 WHO Classification of Hematolymphoid Tumors (5th Edition).
Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1, or 2.
Adequate liver and kidney function according to protocol requirements.
A female of childbearing potential must agree to use adequate contraception from the time of screening through 180 days following the last dose of study intervention. A male with a female partner of childbearing potential must agree to use abstinence or adequate contraception from the time of screening through 90 days following the last dose of study intervention.
NONINTENSIVE THERAPY STUDY ONLY (VEN+AZA):
Documented NPM1-m.
Patients considered ineligible for Intensive Therapy defined by the following:
i. Age ≥75, OR
ii. Age <75 with an ECOG performance status of 2 or cardiac, renal, or hepatic impairment per protocol criteria.
INTENSIVE THERAPY STUDY ONLY (7+3):
Documented NPM1-m or KMT2A-r (KMT2A-r patients with a partial tandem duplication are not eligible).
Documented FLT3 wild-type or ITD ratio <0.05 OR ineligible to receive FLT3-targeted therapy (medically ineligible or mutation in which FLT3 inhibition is not SOC). Lack of access to an FLT3 inhibitor is not considered "ineligible" for FLT3-targeted therapy.
Ejection fraction of ≥50%.
Fit for Intensive Therapy per Investigator opinion.
Exclusion Criteria
Prior therapy for AML (except hydroxyurea or leukapheresis for WBC control).
Diagnosis of acute promyelocytic leukemia (APL), blast phase chronic myeloid leukemia, or isolated myeloid sarcoma.
Known history of BCR-ABL mutation.
History of other active concurrent malignancies prior to study entry except:
Basal cell skin cancer or localized squamous cell cancer of the skin
Previous malignancy confined and locally resected (or treated with other modalities) with curative intent
Prostate or breast cancer receiving adjuvant hormonal therapy.
Active central nervous system (CNS) involvement by AML.
Clinical signs/symptoms of leukostasis or white blood cells (WBC) >25×10^9/L prior to start of ziftomenib/placebo. Note: Hydroxyurea and/or leukapheresis are permitted to meet this criterion.
Known uncontrolled HIV infection or known active hepatitis B virus, hepatitis C virus infection, or other uncontrolled infection.
Uncontrolled intercurrent illness including but not limited to, cardiac illness as defined in the protocol.
Women who are pregnant or lactating.
The Estimated Number of Participants
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Taiwan
35 participants
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Global
1320 participants
