4 results
PRIMARY• To determine the efficacy on study Day 28 of the IT-combination in inducing a clinical response in patients with severe acute GVHD refractory to first line therapy with intermediate dose corticosteroids. SECONDARY• To evaluate the overall…
The primary objective is to determine the effect of a 8-week inspiratory muscle training program on respiratory muscle function in nemaline myopathy patients. The secondary objective is to determine respiratory muscle function in nemaline myopathy…
Primary Objective:To evaluate the efficacy of VX-445 in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are homozygous for the F508del mutation (F/F)Secondary Objectives:* To evaluate the…
Primary Objective:To evaluate the efficacy of VX-445 in triple combination (TC) with tezacaftor (TEZ) andivacaftor (IVA) in subjects with cystic fibrosis (CF) who are heterozygous for F508del and aminimal function mutation (F/MF subjects)Secondary…