Bioavailability and Bioequivalence Study of Ziftomenib in Participants With Relapsed/Refractory AML or MDS

November 5, 2026 updated by: Kura Oncology, Inc.

Relative Bioavailability and Bioequivalence Study of Ziftomenib Drug Products in Participants With Relapsed/Refractory AML With NPM1 Mutation, or With Relapsed/Refractory AML or MDS With Mutations Associated With MEIS1 Overexpression

This study will evaluate the pharmacokinetics (PK) of ziftomenib drug products to inform relative bioavailability (rBA) and bioequivalence (BE) assessments.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Not yet recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Detailed Description

The purpose of this study is to measure rBA or BE of various ziftomenib drug products in participants with R/R NPM1-m AML, or with R/R AML or MDS with a mutation associated with MEIS1 overexpression.

Study details include:

  • The study is composed of two parts.
  • Participant study duration will be a minimum of approximately 4 months including screening, 2 cycles of treatment, and safety follow-up.
  • The visit frequency will be weekly in Part 1.
  • The required treatment duration will be approximately 2 cycles of treatment (56 days) in Part 1 with the option for participants deriving clinical benefit to continue treatment in Part 2.
  • The visit frequency will be monthly in Part 2.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Estimated)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
126

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 1

Expanded Access

Expanded Access

A way for patients with serious diseases or conditions who cannot participate in a clinical trial to gain access to a medical product that has not been approved by the U.S. Food and Drug Administration (FDA). Also called compassionate use. There are different expanded access types.
Available

Available

  • Available: Expanded access is currently available for this investigational treatment, and patients who are not participants in the clinical study may be able to gain access to the drug, biologic, or medical device being studied.
  • No longer available: Expanded access was available for this intervention previously but is not currently available and will not be available in the future.
  • Temporarily not available: Expanded access is not currently available for this intervention but is expected to be available in the future.
  • Approved for marketing: The intervention has been approved by the U.S. Food and Drug Administration for use by the public.
 outside the clinical trial. See expanded access record.

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  1. Age ≥18 years.
  2. Relapsed/refractory NPM1-mutated AML, or relapsed/refractory AML or MDS with a mutation associated with MEIS1 overexpression; disease confirmed in blood and/or bone marrow, as applicable, and not a candidate for currently approved standard-of-care regimens or hematopoietic stem cell transplantation (HSCT).
  3. Female participants must not be pregnant or breastfeeding.
  4. Male participants with partners of childbearing potential must agree to abstinence or highly effective contraception and refrain from sperm donation during treatment and for at least 90 days after the last dose.
  5. Signed informed consent.
  6. Eastern Cooperative Oncology Group (ECOG) performance status ≤2 and life expectancy ≥2 months.
  7. Adequate organ function: estimated glomerular filtration rate ≥30 mL/min/1.73 m²; bilirubin ≤1.5 × the upper limit of normal (ULN), unless attributable to Gilbert's syndrome or leukemic disease; and AST and ALT ≤3 × ULN.
  8. Peripheral white blood cell count ≤25,000/µL before Day 1 of Part 1; hydroxyurea or cytarabine may be used to control the count.

Exclusion Criteria:

  1. Clinically active central nervous system (CNS) leukemia; participants with controlled CNS leukemia receiving intrathecal therapy at study entry may be eligible.
  2. Grade ≥2 acute graft-versus-host disease (GVHD), moderate/severe limited chronic GVHD, or extensive chronic GVHD.
  3. Active HIV, hepatitis C, or hepatitis B infection, as defined in the protocol; participants with controlled disease are not excluded.
  4. Active, uncontrolled systemic fungal, bacterial, viral, or other infection.
  5. Significant cardiovascular disease, including unstable angina, uncontrolled hypertension or arrhythmia, qualifying heart failure, ischemic or severe valvular heart disease, or stroke, transient ischemic attack, or myocardial infarction within the preceding 6 months.
  6. Mean QT interval corrected using Fridericia's formula (QTcF) >480 ms on triplicate electrocardiograms obtained within 5 minutes of each other.

The full inclusion and exclusion criteria, including applicable definitions are presented in the study protocol.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Crossover Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: Cohort A

Sequential administration of oral formulations (Part 1) followed by optional treatment continuation (Part 2)
Drug: Ziftomenib

Oral Formulation 1

Drug: Ziftomenib

Oral Formulation 2

Drug: Ziftomenib

Oral Formulation 3

Drug: Ziftomenib

Oral Formulation 4
Experimental: Cohort B

Sequential administration of oral formulations (Part 1) followed by optional treatment continuation (Part 2)
Drug: Ziftomenib

Oral Formulation 1

Drug: Ziftomenib

Oral Formulation 2

Drug: Ziftomenib

Oral Formulation 3

Drug: Ziftomenib

Oral Formulation 4
Experimental: Cohort C

Sequential administration of oral formulations (Part 1) followed by optional treatment continuation (Part 2)
Drug: Ziftomenib

Oral Formulation 1

Drug: Ziftomenib

Oral Formulation 2

Drug: Ziftomenib

Oral Formulation 3

Drug: Ziftomenib

Oral Formulation 4

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
To determine the plasma PK parameters of ziftomenib following oral (PO) administration (Part 1: Cohorts A, B and C)
Time Frame: During the first 56 days of ziftomenib

Maximum plasma concentration at steady state (Cmax,ss)
During the first 56 days of ziftomenib
To determine the plasma PK parameters of ziftomenib following oral (PO) administration (Part 1: Cohorts A and B)
Time Frame: During the first 56 days of ziftomenib

Plasma concentration observed at end of dosing interval at steady state (Cτ)
During the first 56 days of ziftomenib
To determine the plasma PK parameters of ziftomenib following oral (PO) administration (Part 1: Cohorts A, B and C)
Time Frame: During the first 56 days of ziftomenib

Area under the concentration-time curve from time 0 to dosing interval at steady state (AUC0-τ,ss)
During the first 56 days of ziftomenib
To determine the plasma PK parameters of ziftomenib following oral (PO) administration (Part 1: Cohorts A and B)
Time Frame: During the first 56 days of ziftomenib

Time to Cmax,ss (Tmax,ss)
During the first 56 days of ziftomenib
To determine the plasma PK parameters of ziftomenib following oral (PO) administration (Part 1: Cohorts A and B)
Time Frame: During the first 56 days of ziftomenib

Cavg,ss
During the first 56 days of ziftomenib

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
To determine the plasma PK parameters of ziftomenib following oral (PO) administration (Part 1: Cohort C)
Time Frame: During the first 56 days of ziftomenib

Cτ
During the first 56 days of ziftomenib
To determine the plasma PK parameters of ziftomenib following oral (PO) administration (Part 1: Cohort C)
Time Frame: During the first 56 days of ziftomenib

Tmax,ss
During the first 56 days of ziftomenib
To determine the plasma PK parameters of ziftomenib following oral (PO) administration (Part 1: Cohort C)
Time Frame: During the first 56 days of ziftomenib

Cavg,ss
During the first 56 days of ziftomenib
To assess safety and tolerability of ziftomenib (Parts 1 and 2)
Time Frame: Day 1 of study intervention through 28 days after the last dose
  1. Incidence and severity of AEs per National Cancer Institute-Common Terminology Criteria for Adverse Events (NCI CTCAE) V6.0
  2. Incidence of SAEs
Day 1 of study intervention through 28 days after the last dose
To assess discontinuation of study treatment due to Adverse Events (Parts 1 and 2)
Time Frame: Day 1 of study intervention through 28 days after the last dose

Discontinuation of study treatment due to AEs
Day 1 of study intervention through 28 days after the last dose
To assess deaths while receiving treatment (Parts 1 and 2)
Time Frame: Day 1 of study intervention through 28 days after the last dose

Deaths on treatment
Day 1 of study intervention through 28 days after the last dose

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Kura Oncology, Inc.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Estimated)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
October 30, 2026

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
February 28, 2029

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
February 28, 2029

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
October 21, 2026

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
November 5, 2026

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
November 10, 2026

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
November 10, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
November 5, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
October 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • KO-MEN-018
  • 2025 (U.S. NIH Grant/Contract)
  • 2025-524332-19-00 (Ctis)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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