4 results
7.2 Primary ObjectiveThe primary objective is to assess the incidence of FVIII inhibitory antibodies(>=0.6 Bethesda units [BU] using the Nijmegen modification of the Bethesda assay).7.3 Secondary Objectives1. To evaluate the PK parameters of…
To determine if emixustat hydrochloride (emixustat) reduces the rate of progression of macular atrophy (MA) compared to placebo in subjects with Stargardt disease (STGD)1. To evaluate the safety and tolerability of emixustat compared to placebo2. To…
Primary ObjectiveThe primary objective is to compare the annualized rates of bleeding episodes (ABR)between subjects receiving a prophylactic regimen of BAX 855 with an on-demandtreatment regimen.Secondary ObjectivesThe key secondary objective is to…
To assess whether the use of the personalized risk assessment tool (PERSARC) is (cost)effective in reducing decisional conflict and increasing informed choices in high-grade extremity STS compared to usual care (co-primary outcomes). In addition, we…