A Phase 1/2 Study of VX-445 in Healthy Subjects and Subjects With Cystic Fibrosis

Project type/s Clinical trial
Project status Currently underway

Cystic Fibrosis is a disease with a genetic origin, which results in defects in the function of a protein, known as CFTR. This protein plays a critical role in the production of sweat, digestive fluids, and mucous from lungs and other organs, and individuals carrying a faulty CFTR manifest abnormal secretion of mucus in these tissues resulting in severe problems in the lung and other organs, ultimately reducing life expectancy. In recent years few drugs have been developed for restoring the abnormal CFTR function in individuals with selective mutations, showing large benefits in reducing the symptoms and burden of CF disease by treating the underlying cause of this disease.

This project represents a first-in-human study of the efficacy and safety of VX-445 —a fourth next-generation drug for correcting the function of CFTR protein, which has been recently developed by Vertex Pharmaceuticals.

Team Members

  • Dr Lucy Burr - Team Role: Principal Investigator
  • Megan Martin - Team Role: Project Coordinator