Relapsed / Refractory AMLPhase 1NCT06359002
Safety, Pharmacokinetics, and Preliminary Efficacy of BYON4413 in Acute Myeloid Leukemia and Myelodysplastic Neoplasms.
A Phase 1 study testing BYON4413 in people with Relapsed / Refractory AML and Relapsed / Refractory MDS, run by Byondis B.V. at 9 sites in 3 countries.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Relapsed / Refractory AML, Relapsed / Refractory MDS
- What is being tested?
- BYON4413
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 16 participants
- Where
- 9 sitesin 3 countries
- Study dates
- 25 Jun 2024to Feb 2026
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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;
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- 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.
Trial locations and contacts
9 sites in 3 countries.
9 sites in 3 countries
Spain6 sites
Institut Catala d'Oncologia
Badalona
Hospital Clinic de Barcelona
Barcelona
Hospital de la Santa Creu i Sant Pau
Barcelona
Hospital San Pedro de Alcantara
Cáceres
Hospital Universitario de Salamanca
Salamanca
Hospital Universitario y Politecnico La Fe
Valencia
About this study
Official title: 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.
Read the 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.
- Being tested
- BYON4413
- Keywords
- BYON4413, CD123, Hematologic Malignancies, Antibody Drug Conjugate, AML, MDS
- Medical subject headings
- Leukemia, Myeloid, Acute, Recurrence, Hematologic Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BYON4413Drug
BYON4413 will be administered by IV infusion.
| Study group | Role | What they receive |
|---|---|---|
| Dose Escalation | Experimental | Escalating dose cohorts of BYON4413 for patients with AML or MDS. Receives: Drug: BYON4413 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of dose-limiting toxicities (dose escalation)
Measured: 21 days
Composite Complete Remission Rate (expansion)
CR + CRh + CRi according ELN 2022 criteria
Measured: Up to 24 months
Secondary outcomes (16)
Incidence and severity of adverse events
Measured: Up to 24 months
Number of patients with dose modifications
Measured: Up to 24 months
Rate of early death
Measured: Within 3 treatment cycles
Maximum Plasma Concentration (Cmax) BYON4413
Measured: Up to 24 months
Time to Cmax (Tmax) BYON4413
Measured: Up to 24 months
Area under the curve (AUC) BYON4413
Measured: Up to 24 months
Percentage of patients with confirmed anti-BYON4413 antibodies
Measured: Up to 24 months
Composite Complete Remission Rate (dose escalation)
CR + CRh + CRi according ELN 2022 criteria
Measured: Up to 24 months
Percentage of blasts in bone marrow change from baseline
Measured: Up to 24 months
Percentage of blasts in peripheral blood change from baseline
Measured: Up to 24 months
Objective response rate
CR + CRh + CRi + MLFS + PR according ELN 2022 criteria
Measured: Up to 24 months
Duration of response
Measured: Up to 24 months
Relapse-free survival
Measured: Up to 24 months
Event-free survival
Measured: Up to 24 months
Time to response
Measured: Up to 24 months
Overall survival
Measured: Up to 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Apr 2024
- Study start25 Jun 2024
- Primary completion30 Sep 2025
- Study completionFeb 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Relapsed / Refractory AMLStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 16 participants (actual)
Sponsor and investigators
- Lead sponsor
- Byondis B.V.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06359002
- Sponsor's protocol ID
- BYON4413.001
- Other IDs
- 2023-507781-13-00, 2023
- Status last verified
- Jan 2026
- First submitted
- 26 Mar 2024
- Last updated
- 20 Jan 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 20 Jan 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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.
- ClinicalTrials.gov ID
- NCT06359002
- Acronym / sponsor ID
- BYON4413.001
- Keywords
- BYON4413, CD123, Hematologic Malignancies, Antibody Drug Conjugate, AML, MDS
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 16 participants (actual)
- Lead sponsor
- Byondis B.V.Industry (pharma, biotech, devices)
- Study start
- 25 Jun 2024 (actual)
- Primary completion
- 30 Sep 2025 (actual)
- Study completion
- Feb 2026 (estimated)
- First posted
- 11 Apr 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Dose Escalation | Experimental | Drug: BYON4413Escalating dose cohorts of BYON4413 for patients with AML or MDS. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Patients who have progressed on standard therapy or have no established alternative treatment, with a diagnosis of:
- Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1 or 2;
- Adequate baseline organ function.
Key exclusion criteria (9 listed)
- 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;
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| BYON4413BYON4413 will be administered by IV infusion. | Drug | Dose Escalation |
Primary outcome
Incidence of dose-limiting toxicities (dose escalation)
Time frame: 21 days
Composite Complete Remission Rate (expansion)
Time frame: Up to 24 months
CR + CRh + CRi according ELN 2022 criteria
16 secondary outcomes
Incidence and severity of adverse events
Time frame: Up to 24 months
Number of patients with dose modifications
Time frame: Up to 24 months
Rate of early death
Time frame: Within 3 treatment cycles
Maximum Plasma Concentration (Cmax) BYON4413
Time frame: Up to 24 months
Time to Cmax (Tmax) BYON4413
Time frame: Up to 24 months
Area under the curve (AUC) BYON4413
Time frame: Up to 24 months
Percentage of patients with confirmed anti-BYON4413 antibodies
Time frame: Up to 24 months
Composite Complete Remission Rate (dose escalation)
Time frame: Up to 24 months
Percentage of blasts in bone marrow change from baseline
Time frame: Up to 24 months
Percentage of blasts in peripheral blood change from baseline
Time frame: Up to 24 months
Objective response rate
Time frame: Up to 24 months
Duration of response
Time frame: Up to 24 months
Relapse-free survival
Time frame: Up to 24 months
Event-free survival
Time frame: Up to 24 months
Time to response
Time frame: Up to 24 months
Overall survival
Time frame: Up to 24 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Jan 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
9 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing BYON4413 in people with Relapsed / Refractory AML and Relapsed / Refractory MDS, run by Byondis B.V. at 9 sites in 3 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 20 Jan 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 9 sites in Spain, Belgium, Netherlands. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of dose-limiting toxicities (dose escalation) (time frame: 21 days). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 Jan 2026.