3 results
Primary objective:Determine the incidence rate of inhibitor formation in PUPs with severe and moderately severe hemophilia A during the first 50 exposure days (EDs) to starting with a once weekly prophylactic regimen together with the minimization…
1) Compare a group of bilateral users to a unilateral control group (between-subject design)2) Compare unilateral use to bilateral use after sequential implantation (within-subject design)3) Compare simultaneous bilateral implantation to sequential…
To characterize the PK of eltrombopag at steady state after oral administration in pediatric patients with SAA.Secondary (key only, see protocol page 40-41 for all objectives): Safety and tolerability. Efficacy (overall response rate ORR).