6 results
Primary ObjectiveTo evaluate the efficacy of lumacaftor in combination with ivacaftor throughat Week 24 in subjects with cystic fibrosis (CF) who are homozygous for the F508del CFTR mutation on the CF transmembrane conductance regulator (CFTR)…
Primary Objective* To evaluate the efficacy and safety of tofacitinib in subjects in stable remission on 10 mg BID who decrease the dose to and remain on 5 mg BID (*5 mg BID dose group*) compared to subjects remaining on 10 mg BID. Secondary…
Primary Objective:To evaluate the efficacy of ELX/TEZ/IVA in subjects 6 through 11 years of age with CF, heterozygous for F508del and a MF mutation (F/MF)Secondary Objectives:• To evaluate the PD of ELX/TEZ/IVA• To evaluate the safety of ELX/TEZ/IVA
Primary:Part A: To assess the safety and tolerability of single ascending doses of DMT in healthy subjects, when given by IV infusion. Part B: To assess the safety and tolerability of fixed repeated doses of DMT in healthy subjects, when given three…
To evaluate the efficacy and pharmacodynamics (PD) of ELX/TEZ/IVA
To evaluate the efficacy of VX-121/TEZ/D-IVA in CF subjects who are homozygous forF508del, heterozygous for F508del and a gating (F/G) or residual function (F/RF) mutation, orhave at least 1 other TCR CFTR mutation and no F508del mutation