Evaluating BYON4413 for treating acute myeloid leukemia and myelodysplastic neoplasms.
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 study is testing a new drug called BYON4413 to see if it can safely help people with relapsed or hard-to-treat acute myeloid leukemia and myelodysplastic syndromes.
Quick facts
| Phase | Phase 1 |
|---|---|
| Study type | Interventional |
| Enrollment | 100 (estimated) |
| Ages | 18 Years and up |
| Sex | All |
| Sponsor | Byondis B.V. Industry-sponsored |
| Locations | 9 sites (Antwerpen and 8 other locations) |
| Trial ID | NCT06359002 on ClinicalTrials.gov |
What this trial studies
This clinical trial is the first-in-human evaluation of BYON4413, focusing on its safety, pharmacokinetics, and preliminary efficacy in patients with relapsed or refractory acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS). The study is divided into two parts: the first part involves a dose escalation to determine the maximum tolerated dose, while the second part assesses the anti-leukemia activity and safety of the drug at the recommended dose. Patients will be monitored for safety and response to treatment throughout the trial.
Who should consider this trial
Good fit: Ideal candidates are patients with relapsed or refractory AML or MDS who have progressed on standard therapies and have no established alternative treatments.
Not a fit: Patients who have previously received CD123-targeting therapies or have active central nervous system AML may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could provide a new therapeutic option for patients with relapsed or refractory AML and MDS.
How similar studies have performed: While this approach is novel in the context of BYON4413, similar studies targeting CD123 in hematologic malignancies have shown promise.
Eligibility criteria
Show full inclusion / exclusion criteria
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.
Where this trial is running
Antwerpen and 8 other locations
- Het Ziekenhuisnetwerk Antwerpen — Antwerpen, Belgium (Recruiting)
- UZ Leuven — Leuven, Belgium (Recruiting)
- Universitair Medisch Centrum Groningen — Groningen, Netherlands (Recruiting)
- Institut Catala d'Oncologia — Badalona, Spain (Recruiting)
- Hospital Clinic de Barcelona — Barcelona, Spain (Recruiting)
- Hospital de la Santa Creu i Sant Pau — Barcelona, Spain (Recruiting)
- Hospital San Pedro de Alcantara — Caceres, Spain (Recruiting)
- Hospital Universitario de Salamanca — Salamanca, Spain (Recruiting)
- Hospital Universitario y Politecnico La Fe — Valencia, Spain (Recruiting)
Study contacts
- Study coordinator: Glenn van Wigcheren
- Email: clinicaltrials@byondis.com
- Phone: +31 24 679 5100
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.