ICM to Evaluate the Activation of p.Phe508del-CFTR by Lumacaftor in Combination With Ivacaftor
Status:
Completed
Trial end date:
2020-02-10
Target enrollment:
Participant gender:
Summary
The academic investigator - initiated trial will evaluate in a postapproval setting whether,
and if yes, to what extent and variability, the treatment with lumacaftor in combination with
ivacaftor reverses the p.Phe508del CFTR - mediated basic defect in p.Phe508del homozygous
subjects with cystic fibrosis under real life conditions.