G551D Observational Study- Expanded to Additional Genotypes and Extended for Long Therm Follow up (GOAL-e2) (GOAL- e2)
Children's Health | Diseases of the Respiratory Systems
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.
- Ages6 years and older
- Trial withCystic Fibrosis Foundation
- Start Date05/28/2014
- End Date01/30/2016
- Last Updated06/22/2018
- Study HIC#1401013233