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NCT07355335PHASE1NOT_YET_RECRUITING

Ziftomenib + Mezigdomide in Adolesc. and Adults w/ R/R AML

A Phase 1 Study of Menin-KMT2A Inhibitor Ziftomenib (KO-539) in Combination With Cereblon E3 Ligase Modulator Mezigdomide (CC-92480) in Adolescents and Adults With Relapsed or Refractory Acute Myeloid Leukemia (AML)

Sponsor: Massachusetts General Hospital + Kura Oncology, Inc., Bristol-Myers Squibb

No open prediction endpoints

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Key Facts

Study type
INTERVENTIONAL
Conditions
KMT2A-rearranged, NPM1-mutant Refractory or Relapsed AML
Interventions
Ziftomenib, Mezigdomide
Enrollment
24 participants
Primary completion
Jul 2027
Study completion
Jan 2028
First posted
Jan 2026
Last updated
Jan 2026

Primary Endpoints (CT.gov)

Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]

Time frame: From the start of treatment until participant withdrawal, death, or removal from study, whichever comes first, assessed up to 2 years after initial dose of study treatment.

Recommended phase 2 dose (RP2D) of mezigdomide in combination with ziftomenib.

Time frame: From start of treatment to end of 12 cycles (each cycle is 28 days).

Secondary Endpoints

Complete remission/complete remission with partial hematologic recovery (CR/CRh) and overall response rate (ORR)

2-year overall and relapse-free survival rates

Eligibility Criteria

Inclusion Criteria: * Age ≥16 years during dose escalation portion of study, patients must weigh ≥40 kg. * Age \>12 years during dose expansion portion of study, patients must weight ≥40 kg. * Diagnosis of AML per the WHO Classification of Hematolymphoid Tumors (5th Edition) with documented KMT2A rearrangement or NPM1 cytoplasmic-type (NPM1c) mutation. KMT2A-rearrangements must be confirmed by FISH or RNA-based fusion calling by a CLIA-certified laboratory. This study will only enroll KMT2A gene rearrangements in which there is a translocation between the N-terminal portion of KMT2A and a fusion partner, and will not include KMT2A partial tandem duplications (PTDs) or other structural alterations of KMT2A. NPM1c mutations must be confirmed by DNA sequencing in a CLIA-certified laboratory. Patients with myeloid sarcoma are eligible only if concurrent bone marrow involvement is present. Patients must have at least 5% bone marrow disease by morphology at the time of study entry. * Patien

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✦ Analyst Commentary

Expert commentary on why this trial matters and what to watch for.

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Source

Open on ClinicalTrials.gov