drugset / Trial / NCT02325362
Effect of Miglustat on the Nasal Potential Difference in Patients With Cystic Fibrosis Homozygous for the F508del Mutation
Phase 2/3
Completed
16 enrolled
Assistance Publique - Hôpitaux de Paris
Actelion · collabCRCM (Centres de Ressources et de Compétences de la Mucoviscidose) · collabURC-CIC Paris Descartes Necker Cochin · collab
RandomizedCrossoverTriple-blindTreatment
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.
Timeline
- Start
- 2015-03-17
- Primary completion
- 2017-04-03
- Completion
- 2017-04-03
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Miglustat | Small molecule | — | — |