4 results
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…
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…
To demonstrate in a phase III study that the addition of the monoclonal anti-T cell antibody alemtuzumab added to standard CHOP chemotherapy followed by autologous stam cell transplantation will increase the response, event-free survival,…
The three key outcome measurements and challenges of this proof of concept study are to achieve a decrease in ongoing HO bone formation, decrease in numbers of disease *flare-up* episodes and to prevent the continuation of FOP induced contractures.…