6 results
The primary objective is to evaluate the safety of ReFacto AF in previously untreated patients (no prior exposure to factor products or any blood products) of less than 6 years of age.
This research project aims to investigate whether hyperoxic interval training improves whole body insulin resistance, endothelial dysfunction, cardiovascular fitness, lipid metabolism and glycemic control in the treatment of deconditioned non-…
DDAVP treatment combined with FVIII clotting factor concentrates in patients with mild hemophilia A.
Primary objectives:To assess the proportion of non-severe hemophilia A patients within FVIII target levels with the DDAVP and FVIII concentrate combination treatment in the first 72 hours after the start of combination treatment, without adding off-…
Primary:To compare the efficacy of asciminib versus Investigator selected TKI with respect to the proportion of patients that are in Major Molecular Response at Week 48. To compare the efficacy of asciminib versus Investigator selected TKI, within…
Primary: To compare the Major Molecular Response (MMR) rate at 24 weeks of ABL001 versus bosutinibSecondary: To compare additional parameters of the efficacy of ABL001 versus bosutinib. Safety, tolerability.
To investigate the reliability and feasibility of PK-guided prophylactic dosing of factor concentrates in hemophilia A and B patients (predictive performance).