Effect of Miglustat on the Nasal Potential Difference in Patients With Cystic Fibrosis Homozygous for the F508del Mutation
Status:
Completed
Trial end date:
2017-04-03
Target enrollment:
Participant gender:
Summary
The purpose of this study is to demonstrate that Miglustat restores the function of the
cystic fibrosis transmembrane conductance regulator (CFTR) in adult patients with cystic
fibrosis homozygous for the F508del mutation.
Phase:
Phase 2/Phase 3
Details
Lead Sponsor:
Assistance Publique - Hôpitaux de Paris
Collaborators:
Actelion CRCM (Centres de Ressources et de Compétences de la Mucoviscidose)