5 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.
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-…
Part 1PrimaryTo assess dose limiting toxicities (DLTs) of EDR to select an RP3D for the combination to be used in Part 2 of this study.Secondary• To evaluate the overall safety profile of EDR to select an RP3D for the combination to be used in Part…
Efficacy ObjectivesThe primary efficacy objective:• To evaluate the efficacy of GDC-0199 and rituximab (GDC-0199+R) compared with bendamustine and rituximab (BR) in patients with relapsed or refractory chronic lymphocytic leukemia (CLL) as measured…
To investigate the reliability and feasibility of PK-guided prophylactic dosing of factor concentrates in hemophilia A and B patients (predictive performance).