Investigational Therapy That Seeks to Correct Basic Defect in CF Enters Phase 2 Trial

June 15, 2007

Vertex Pharmaceuticals Incorporated recently announced the beginning of a Phase 2 clinical trial of an investigational therapy known as VX-770. The drug, which is given orally, is designed to address a core defect in CF. The defect causes thick mucus to accumulate in the lungs and causes a host of serious problems including inflammation and infections. This potential therapy is aimed at correcting the defect and may benefit other organs in addition to the lungs.

The trial will involve approximately 36 adult volunteers with cystic fibrosis from different CF care centers across the country. A Phase 1 clinical trial of VX-770, which was completed in 2006, demonstrated that the drug could achieve desired levels in the blood. The Phase 2 clinical trial will study the safety and tolerability of the drug compared with a placebo, and how well the body absorbs the drug. Currently, the drug is being studied only in individuals with CF who carry at least one G551D mutation in their CFTR genes.

For more details regarding this clinical trial, click here.

The CF Foundation encourages people with CF to learn more about participating in clinical trials. People with cystic fibrosis play a critical role in the development of new treatments and the search for a cure. For more information about clinical trials in general, click here or call (877) 8CF-JOIN (877-823-5646).