Ziftomenib for the Treatment of Patients With NPM1 Mutated or KMT2A Rearranged Acute Myeloid Leukemia Not Eligible for Standard Therapy

Part of paid clinical trials in Columbus, Ohio.

Sponsor
Uma Borate
Study ID
NCT06930352
Phase
PHASE2
Status
Not Yet Recruiting

Notify me when recruiting opens

Save your spot on the interest list for this study. We'll keep your details with this study so our team can follow up when recruiting opens.

Not yet recruiting

Add your contact details and location so we can keep your interest tied to this study.

Conditions

Eligibility Criteria

Sex
ALL
Age
18 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • Biospecimen Collection — PROCEDURE
    Undergo blood sample collection
  • Bone Marrow Aspiration — PROCEDURE
    Undergo bone marrow biopsy and/or aspiration
  • Bone Marrow Biopsy — PROCEDURE
    Undergo bone marrow biopsy and/or aspiration
  • Cytarabine — DRUG
    Given cytarabine
  • Echocardiography Test — PROCEDURE
    Undergo ECHO
  • Hydroxyurea — DRUG
    Given hydroxyurea
  • Leukapheresis — PROCEDURE
    Undergo leukapheresis
  • Multigated Acquisition Scan — PROCEDURE
    Undergo MUGA
  • Questionnaire Administration — OTHER
    Ancillary studies
  • Ziftomenib — DRUG
    Given PO

Study Details

This phase II trial tests how well ziftomenib works in treating patients with NPM1 mutated or KMT2A rearranged acute myeloid leukemia (AML) and are not eligible to receive standard therapy. AML is often due to genetic changes in the cancer cells, including mutations in the NPM1 gene and rearrangements involving the KMT2A gene. These mutations result in activation of the menin pathway. Menin is a type of protein in the body that helps to regulate some of the naturally occurring processes in the body, but can also be involved in some types of cancers. Ziftomenib blocks this menin pathway and may prevent the cancer cells from continuing to grow. Giving ziftomenib may kill more cancer cells in patients with NPM1 mutated or KMT2A rearranged AML that are not eligible to receive standard therapy.

Key Dates

Start date
Apr 10, 2026
Status verified
Mar 2026
Primary completion
Dec 31, 2027
Completion
Dec 31, 2027

Study Design

Enrollment
70 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Treatment (ziftomenib)
    Patients receive ziftomenib PO QD on days 1-28 of each cycle. Cycles repeat every 28 days for up to 24 cycles in the absence of disease progression or unacceptable toxicity. Patients may also undergo cytoreduction therapy with hydroxyurea up to end of cycle 1, cytarabine within 7 days of starting treatment, or leukapheresis within 7 days of treatment to reduce white blood cell count to =\< 10,000/uL. Additionally, patients undergo ECHO or MUGA at screening and bone marrow biopsy and/or aspiration and blood sample collection throughout the study.

Primary Outcome Measure

Complete remission (CR) plus CR/response with hematologic improvement [ Time Frame: After 6 cycles of treatment (cycle length = 28 days) ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
Ohio State University Comprehensive Cancer CenterColumbusOhio43210
Uma M. Borate, MBBS, MD, MSc
[email protected]
614-685-9828
Uma M. Borate, MBBS, MD, MSc (PRINCIPAL_INVESTIGATOR)

Find similar trials in Columbus, OH

By condition
Leukemia (other)
By specialty
OncologyBlood cancer
By research site
Ohio State University Comprehensive Cancer Center· Columbus, OH

Related Studies