KO-Men-017: Ziftomenib in NPM1 Mutated or KMT2A Rearranged Acute Myeloid Leukemia

Purpose of this Study

We are doing this study to find the most effective, safe dose of an experimental drug called ziftomenib (the study drug). We want to know how well it works when it combined with either standard non-intensive (venetoclax + azacitidine) therapy or standard intensive (7+3) therapy for adults who have acute myeloid leukemia.

Who Can Participate?

Eligibility

Adults ages 18+ who:
  • Diagnosed with AML
  • Have a documented NPM1-m or KMT2A-r mutation
  • Have no history of BCR-ABL mutation
For more information, contact the study team at erin.murphy@duke.edu.

Age Range

18-110

Sex/Genders

Male (cisgender)
Female (cisgender)
Non-binary or gender fluid
Transgender male
Transgender female
Looking for Healthy Participants
No

What is Involved?

Description

If you choose to join this study, you will participate in either the intensive or the non-intensive therapy groups. The study team will let you know which option is available to you when you are making your decision. If you participate in the intensive therapy portion of the study, you will get a random assignment (fair, equal chance) to 1 of 3 groups:
  • Group 1: If you are in this group, your study regimen will be 7+3 in combination with the study drug during induction, ziftomenib+cytarabine consolidation, and then the study drug for maintenance
  • Group 2: If you are in this group, your study regimen will be 7+3 in combination with the study drug during induction, ziftomenib+cytarabine consolidation, and then a placebo (inactive substance with no drug in it) for maintenance
  • Group 3: If you are in this group, your study regimen will be 7+3 in combination with placebo during induction, placebo+cytarabine consolidation, and then placebo maintenance for maintenance
If you participate in the non-intensive therapy portion of the study, you will get a random assignment (fair, equal chance) to 1 of 2 groups:
  • Group 1: If you are in this group, your study regimen will be venetoclax + azacitidine in combination with the study drug
  • Group 2: If you are in this group, your study regimen will be venetoclax + azacitidine in combination with a placebo
During the study, your doctor may decide that a stem cell transplant is the best treatment option for you. If you do receive a stem cell transplant as part of your standard treatment, you may then go on to receive maintenance therapy after the stem cell transplant is complete.

Locations

Duke University Hospital

Visit Timing

Weekdays

Compensation

No

Spanish Materials Available

No

Study Details

Full Title

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

Principal Investigator

Harry
Erba

Protocol Number

PRO00118403

NCT ID

NCT07007312

Phase

III

Enrollment Status

Pending Open to Enrollment
ClinicalTrials.gov