92 results
Investigating red blood cell deformability changes, during treatment, measured with hyperoxia-hypoxia ektacytometry in sickle cell anemia patients, patients with HbSC disease and patients with HbS-beta-thalassemia.
This study has been transitioned to CTIS with ID 2024-515528-35-00 check the CTIS register for the current data. Primary objectiveTo determine whether individualized pharmacokinetic (PK)-guided dosing of emicizumab is non-inferior to conventional…
Our primary aim is to investigate the current bleeding tendency of patients with VWD.
To study the effect of apotransferrin administration in patients with β thalassemia intermedia on erythropoiesis as reflected by hemoglobin level or transfusion dependency. Secondary objectives are the effect of apotransferrin on the…
To investigate the reliability and feasibility of PK-guided prophylactic dosing of factor concentrates in hemophilia A and B patients (predictive performance).
1. To establish optimized approaches for the isolation and characterization of BOECs, CD34+-derived MKs, iPSC- ECs and iPSC-MKs.2. Dissect the cellular mechanisms that control endothelial and platelet (secretory) function, thereby understanding the…
The aim of this study is to identify genetic determinants that are associated with successful immune tolerance induction in patients with haemophilia A and inhibitors.
This study is being conducted to determine the safety and efficacy of the study drug BIVV001 when used as a once-a-week prophylaxis treatment or as an on-demand (as-needed) treatment for bleeding in patients 12 years and older with severe hemophilia…
Main objective: - To investigate pyruvate kinase thermal stability in haemoglobinopathies.Secondary objectives: - To investigate the possibility of stimulation of PK activity and thermal stability by use of allosteric activators- To investigate…
The aim of SPIRIT is to investigate the mechanisms of the immunological tolerance to FVIII in patients with hemophilia A aged younger than 18 years using NFT for prophylaxis.
To study the effect of crizanlizumab on the hemodynamics of the cerebral vasculature (CBF and CVR)
The purpose of this study is to identify a pathogenic or likely pathogenic variant in unresolved families with antithrombin deficiency.