Safety, Pharmacokinetics, and Preliminary Efficacy of BYON4413 in Acute Myeloid Leukemia and Myelodysplastic Neoplasms.

January 16, 2026 updated by: Byondis B.V.

A First-in-human Dose Escalation and Expansion Trial With the Antibody-drug Conjugate BYON4413 to Evaluate Safety, Pharmacokinetics, and Preliminary Efficacy in Patients With Relapsed/Refractory Acute Myeloid Leukemia or Myelodysplastic Neoplasms.

This is the first-in-human trial with BYON4413 to evaluate safety, PK, immunogenicity, and anti-leukemia activity of BYON4413 in patients with AML or MDS.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Active, not 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

This trial includes two parts. Part 1 is a dose escalation study in which the maximum tolerated dose and recommended dose for expansion of BYON4413 will be determined. Part 2 is an expansion study to evaluate the anti-leukemia activity and safety of BYON4413.

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 (Actual)

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.
16

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

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Locations

  • Belgium
      • Antwerp, Belgium
        • Het Ziekenhuisnetwerk Antwerpen
      • Leuven, Belgium
        • UZ Leuven
  • Netherlands
      • Groningen, Netherlands
        • Universitair Medisch Centrum Groningen
  • Spain
      • Badalona, Spain
        • Institut Catala d'Oncologia
      • Barcelona, Spain
        • Hospital Clinic De Barcelona
      • Barcelona, Spain
        • Hospital De La Santa Creu I Sant Pau
      • Cáceres, Spain
        • Hospital San Pedro de Alcantara
      • Salamanca, Spain
        • Hospital Universitario de Salamanca
      • Valencia, Spain
        • Hospital Universitario y Politécnico La Fe

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

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • Patients who have progressed on standard therapy or have no established alternative treatment, with a diagnosis of:

    • R/R AML (WHO 2022) OR
    • MDS (WHO 2022) with ≥10% blasts in BM and have received ≥3 cycles of HMA
  • Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1 or 2;
  • Adequate baseline organ function.

Exclusion Criteria:

  • Having been treated with any CD123-targeting therapies;
  • Having received allogeneic hematopoietic stem cell transplantation within 100 days prior to start Cycle 1 Day 1;
  • Having treatment-related toxicities from prior anti-leukemia therapies that have not resolved to CTCAE Grade ≤ 1;
  • Having active central nervous system AML or AML of the APL/M3 subtype;
  • History of keratitis;
  • History of specified lung or renal disease;
  • Having clinically significant cardiovascular disease;
  • Known infection of Hepatitis B, C or E.

Key inclusion and exclusion criteria details are listed here, additional requirements may apply.

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: N/A
  • Interventional Model: Sequential 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: Dose Escalation
Escalating dose cohorts of BYON4413 for patients with AML or MDS.
Drug: BYON4413
BYON4413 will be administered by IV infusion.

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
Incidence of dose-limiting toxicities (dose escalation)
Time Frame: 21 days
21 days
Composite Complete Remission Rate (expansion)
Time Frame: Up to 24 months
CR + CRh + CRi according ELN 2022 criteria
Up to 24 months

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
Time to response
Time Frame: Up to 24 months
Up to 24 months
Duration of response
Time Frame: Up to 24 months
Up to 24 months
Overall survival
Time Frame: Up to 24 months
Up to 24 months
Incidence and severity of adverse events
Time Frame: Up to 24 months
Up to 24 months
Number of patients with dose modifications
Time Frame: Up to 24 months
Up to 24 months
Rate of early death
Time Frame: Within 3 treatment cycles
Within 3 treatment cycles
Maximum Plasma Concentration (Cmax) BYON4413
Time Frame: Up to 24 months
Up to 24 months
Time to Cmax (Tmax) BYON4413
Time Frame: Up to 24 months
Up to 24 months
Area under the curve (AUC) BYON4413
Time Frame: Up to 24 months
Up to 24 months
Percentage of patients with confirmed anti-BYON4413 antibodies
Time Frame: Up to 24 months
Up to 24 months
Composite Complete Remission Rate (dose escalation)
Time Frame: Up to 24 months
CR + CRh + CRi according ELN 2022 criteria
Up to 24 months
Percentage of blasts in bone marrow change from baseline
Time Frame: Up to 24 months
Up to 24 months
Percentage of blasts in peripheral blood change from baseline
Time Frame: Up to 24 months
Up to 24 months
Objective response rate
Time Frame: Up to 24 months
CR + CRh + CRi + MLFS + PR according ELN 2022 criteria
Up to 24 months
Relapse-free survival
Time Frame: Up to 24 months
Up to 24 months
Event-free survival
Time Frame: Up to 24 months
Up to 24 months

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.
Byondis B.V.

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 (Actual)

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.
June 25, 2024

Primary Completion (Actual)

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.
September 30, 2025

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 1, 2026

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).
March 26, 2024

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.
April 5, 2024

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.
April 11, 2024

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.
January 20, 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.
January 16, 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.
January 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.
  • BYON4413.001
  • 2023-507781-13-00 (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

No

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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