Ziftomenib for Treating Acute Myeloid Leukemia with NPM1 Mutation or KMT2A Rearrangement in Patients Not Eligible for Standard Therapy

Sponsor: Uma Borate

Sponsor score: 0
Inactive

ClinicalTrials ID:NCT06930352

Provider View

This study is testing a new drug, ziftomenib, to see if it can help treat patients with a specific type of acute myeloid leukemia (AML) who cannot receive standard treatments. The drug works by blocking a protein pathway that helps cancer cells grow.

Patient Parameters

Program Overview

This phase II study is exploring how well ziftomenib works for patients with acute myeloid leukemia (AML) that has specific genetic changes, either in the NPM1 gene or involving the KMT2A gene. These changes can activate a protein pathway called menin, which helps cancer cells grow. Ziftomenib aims to block this pathway, potentially stopping the cancer from growing. The study focuses on patients who cannot receive standard treatments for AML.

Description

The main goal of this study is to see if ziftomenib can help patients with certain genetic types of AML achieve complete remission, meaning no signs of cancer, after six cycles of treatment. Researchers will also look at other outcomes, such as how long patients remain free from needing blood transfusions, the overall response rate to the treatment, and how long the response lasts. They will also assess patients' overall survival and quality of life during the study.

Patients will take ziftomenib by mouth once a day for 28 days in each cycle, with up to 24 cycles in total, unless the cancer progresses or side effects become too severe. Some patients may receive additional treatments to manage high white blood cell counts at the start of the study. Throughout the study, patients will undergo various tests, including heart scans and bone marrow biopsies, to monitor their health and the treatment's effects.

After finishing the study treatment, patients will have follow-up visits every six months for up to two years to check on their health and any long-term effects of the treatment.

Eligibility Criteria

Inclusion Criteria

  • Must provide signed consent to participate.
  • Must have a confirmed diagnosis of acute myeloid leukemia (AML) with specific genetic changes: KMT2A rearrangement or NPM1 mutation.
  • Must be ineligible or unwilling to receive standard AML treatments due to age (75 or older) or other health conditions.
  • Must have a white blood cell count of 10,000/uL or lower, with options to manage higher counts at the start of the study.
  • Women of childbearing potential must agree to use effective birth control during the study and for 180 days after the last dose.
  • Men must agree to use effective birth control with partners during the study and for 90 days after the last dose.

Exclusion Criteria

  • Cannot have acute promyelocytic leukemia or chronic myelogenous leukemia in blast crisis.
  • Cannot have active leukemia in the central nervous system.
  • Cannot have received prior AML treatment, except for specific medications to control white blood cell counts.
  • Cannot be taking certain medications that interfere with the study drug.
  • Cannot have active infections like HIV, hepatitis B, or hepatitis C unless controlled.
  • Cannot have conditions that increase the risk of serious infections.
  • Cannot have a heart condition that affects the heart's electrical activity.
  • Cannot have psychiatric conditions that prevent understanding the study.
  • Cannot be pregnant or breastfeeding, and women must have a negative pregnancy test before starting treatment.
  • Cannot require dual antiplatelet therapy.

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