Safety, Pharmacokinetics, and Preliminary Efficacy of BYON4413 in Acute Myeloid Leukemia and Myelodysplastic Neoplasms.
- Conditions
- Relapsed / Refractory MDSRelapsed / Refractory AML
- Interventions
- Drug: BYON4413
- Registration Number
- NCT06359002
- Lead Sponsor
- Byondis B.V.
- Brief 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.
- 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.
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 100
-
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.
- 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 & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SEQUENTIAL
- Arm && Interventions
Group Intervention Description Dose Escalation BYON4413 Escalating dose cohorts of BYON4413 for patients with AML or MDS.
- Primary Outcome Measures
Name Time Method Incidence of dose-limiting toxicities (dose escalation) 21 days Composite Complete Remission Rate (expansion) Up to 24 months CR + CRh + CRi according ELN 2022 criteria
- Secondary Outcome Measures
Name Time Method Incidence and severity of adverse events Up to 24 months Relapse-free survival Up to 24 months Maximum Plasma Concentration (Cmax) BYON4413 Up to 24 months Percentage of blasts in bone marrow change from baseline Up to 24 months Number of patients with dose modifications Up to 24 months Time to Cmax (Tmax) BYON4413 Up to 24 months Percentage of patients with confirmed anti-BYON4413 antibodies Up to 24 months Objective response rate Up to 24 months CR + CRh + CRi + MLFS + PR according ELN 2022 criteria
Time to response Up to 24 months Overall survival Up to 24 months Percentage of blasts in peripheral blood change from baseline Up to 24 months Event-free survival Up to 24 months Rate of early death Within 3 treatment cycles Area under the curve (AUC) BYON4413 Up to 24 months Composite Complete Remission Rate (dose escalation) Up to 24 months CR + CRh + CRi according ELN 2022 criteria
Duration of response Up to 24 months
Trial Locations
- Locations (9)
Het Ziekenhuisnetwerk Antwerpen
🇧🇪Antwerpen, Belgium
UZ Leuven
🇧🇪Leuven, Belgium
Universitair Medisch Centrum Groningen
🇳🇱Groningen, Netherlands
Institut Catala d'Oncologia
🇪🇸Badalona, Spain
Hospital Clinic de Barcelona
🇪🇸Barcelona, Spain
Hospital de la Santa Creu i Sant Pau
🇪🇸Barcelona, Spain
Hospital San Pedro de Alcantara
🇪🇸Caceres, Spain
Hospital Universitario de Salamanca
🇪🇸Salamanca, Spain
Hospital Universitario y Politecnico La Fe
🇪🇸Valencia, Spain