4 results
Primary: To evaluate the efficacy of tisagenlecleucel therapy as measured by overall response rate by investigator assessment.Secondary: Duration of response, event free survival, relapse free survival, overall survival, safety, kinetics,…
To demonstrate the efficacy of repeated daily doses of 40 µg/kg/day and 120 µg/kg/day A4250 in children with progressive familial intrahepatic cholestasis (PFIC) Types 1 and 2, as determined by the following:* Proportion of patients experiencing at…
Primary Objective:Part A:To evaluate the efficacy of losmapimod for the treatment of FSHD by demonstrating slowing of disease progression assessed by reachable workspace quantification of total relative surface area (RSA) Q1-Q5 with 500 g wrist…
To compare tisagenlecleucel treatment strategy to SOC treatment strategy with respect to delaying the composite event of disease progression / stable disease at or after the week 12 assessment; or death at any time.