2 results
Approved WMOPending
To evaluate the efficacy of VX-661 in combination with ivacaftor through 24 weeks of treatment in subjects with cystic fibrosis (CF) who are homozygous for the F508del mutation on the CF transmembrane conductance regulator (CFTR) gene.
Approved WMOCompleted
This trial is, along with establishment of the safety, tolerability and pharmacokinetics and -dynamics of CDZ173 in the target population, designed to select the optimal dose to normalize the function of the PI3K protein in patients with APDS/PASLIā¦