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

Status

Not yet recruiting

Phases

Phase 3

Study type

Interventional

Source

EU CTIS

Registry ID

CTIS2025-521314-25-00

Enrollment

280

Registered

2026-02-20

Start date

Unknown

Completion date

Unknown

Last updated

2026-02-23

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

NPM1-m and KMT2A-r Acute Myeloid Leukemia

Brief summary

Nonintensive Therapy Study - Overall survival (OS) Intensive Therapy Study (7+3) - EFS, defined as the time from randomization to treatment failurea, hematologic relapse following CR, or death from any cause, whichever comes first

Detailed description

Nonintensive Therapy Study - CR rate per the ELN 2022 criteria per Investigator assessment - Percentage of patients achieving centrally defined BM MRD negativity - Rates of CR+CRh per ELN 2022 per Investigator assessment, Intensive Therapy Study (7+3) - Percentage of patients achieving CR per ELN 2022 criteria with centrally defined BM MRD negativity (CRMRD-) - Overall survival (OS)

Interventions

DRUGDaunoblastin® 20 mg Pulver zur Herstellung einer Infusions- oder Injektionslösung

DRUGZiftomenib

DRUGVENETOCLAX

DRUGCYTARABINE

DRUGAZACITIDINE

DRUGZiftomenib Placebo

Eligibility

Sex/Gender

All

Age

18 Years to No maximum

Design outcomes

Primary

Measure	Time frame
Nonintensive Therapy Study - Overall survival (OS) Intensive Therapy Study (7+3) - EFS, defined as the time from randomization to treatment failurea, hematologic relapse following CR, or death from any cause, whichever comes first	—

Secondary

Measure	Time frame
Nonintensive Therapy Study - CR rate per the ELN 2022 criteria per Investigator assessment - Percentage of patients achieving centrally defined BM MRD negativity - Rates of CR+CRh per ELN 2022 per Investigator assessment, Intensive Therapy Study (7+3) - Percentage of patients achieving CR per ELN 2022 criteria with centrally defined BM MRD negativity (CRMRD-) - Overall survival (OS)	—

Outcome results

None listed

Source: EU CTIS · Data processed: Feb 24, 2026