Current Studies

Genetic Modifier Study: Cystic Fibrosis Twin and Sibling

The purpose of this multi-center study is to understand more about Cystic Fibrosis (CF) and how we could better treat people that have it. This research is being done to determine the genes and other factors that are responsible for variation among persons with CF. We are interested in twins and siblings because they share similar genes and a similar environment. There will be no cost to the patient for any tests done as part of this study. Participating in this study is strictly on a volunteer basis. Patients will not be compensated for their participation.

Coordinating Center:
Johns Hopkins Medicine

Sponsor:
National Institute of Health

Principal Investigator at ETCH:
John S. Rogers, M.D.

Contact: If you and your sibling would like more information or would like to participate, please contact us at 1-865-637-8481, ext. 240.

VX08-770-105: An Open-Label Rollover Study to Evaluate the Long-Term Safety and Efficacy of VX-770 in Subjects with Cystic Fibrosis

VX-770 is a new compound, called a "potentiator," designed to act upon a non-functional but properly localized CFTR protein to help to open the chloride channel in CF cells. Phase 3 trials have been completed in pediatric and adult people with CF who have one copy of the G551D mutation in their CF gene. There were no safety issues in these trials and the treatment groups met the primary endpoint for improved lung function and secondary endpoints of reduced pulmonary exacerbations, increase in weight, and increase in quality of life measures.

Patients with CF who have completed VX08-770-102 (Study 102) and VX08-770-103 (Study 103) studies are eligible to participate in this open-label study to further evaluate the safety and efficacy of long-term VX-770 treatment.

Sponsor:
Vertex Pharmaceuticals, Inc.

Principal Investigator at ETCH:
John S. Rogers, M.D.

Cystic Fibrosis Patient Registry
The Cystic Fibrosis (CF) Patient Registry is a data collection effort sponsored by the Cystic Fibrosis Foundation. The goal of the Patient Registry is to collect data on individuals with CF to better understand the illness and ultimately improve the care and survival of those with CF. The Patient Registry was established as a means to monitor important trends in the CF population and to improve understanding, treatment, and survival.

There will be no cost to participate in the registry. Participating in this study is strictly on a volunteer basis. Patients will not be compensated for their participation.

Sponsor:
Cystic Fibrosis Foundation (CFF)

Principal Investigator at ETCH:
John S. Rogers, M.D.

Contact: If you would like more information or would like to participate, please contact us at 1-865-637-8481, ext. 240.