4 results
To investigate whether peri-operative dosing using a population-based pharmacokinetic model (non-linear mixed effect modelling) in hemophilia patients leads to a significant reduction in clotting factor consumption in comparison to the standard…
The purpose of this study is to allow collection of safety and tolerability data in subjects benefitting from treatment with spartalizumab as a single agent or in combination with other study treatments in a pre-defined (Appendix Section 16.1)…
To evaluate the efficacy of rVWF (vonicog alfa) prophylaxis based on the annualized bleeding rate (ABR) of spontaneous (not related to trauma) bleeding episodes in adult and pediatric / adolescent subjects (aged 12 to <18 years) during the…
To characterize the safety, tolerability, and recommended phase 2 dose (RP2D) of each combination partner used with ruxolitinib (Part 1)To evaluate the preliminary efficacy of each novel ruxolitinib combination treatment arm (Parts 2 & 3)