4 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 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
To evaluate the efficacy and pharmacodynamics (PD) of ELX/TEZ/IVA
Part A: healthy volunteers Primary objective- To determine safety and tolerability of a single dose of VB5-845D-800CW in healthy volunteers.Secondary objective- To determine the pharmacokinetics of VB5-845D-800CW by measuring fluorescence in blood…