G551D Observational Study- Expanded to Additional Genotypes and Extended for Long Therm Follow up (GOAL-e2) (GOAL- e2)

What is the purpose of this trial?

The primary objective of this study is to obtain specimens for the CFFT Specimen Biorepository and to record clinical parameters from cystic fibrosis patients with the G551D, R117H, and non-G551D gating CFTR mutations, some of whom may be treated with ivacaftor (a CFTR potentiator drug). The secondary objective of the study is to conduct four specific sub-studies to explore the impact of treatment with ivacaftor on various experimental outcome measures directly and indirectly relevant to CFTR modulation.

Participation Guidelines

Ages: 6 years and older

Gender: Both

Cystic Fibrosis Foundation

Start Date: 05/29/2014

End Date: 01/31/2016

Last Updated: 06/22/2018

Study HIC#: 1401013233

Get Involved

For more information about this study, contact:
Catalina Guzman
+1 203-836-7172

If you would prefer to contact a member of the Help us Discover team about this trial and other similar trials, please email helpusdiscover@yale.edu or call 1-877-978-8348.

Trial Image


Marie Egan

Principal Investigator