Skip to Main Content

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.

  • Trial with
    Seattle Children's Hospital
  • Start Date
  • End Date

I'm interested in volunteering

If you would prefer to contact a member of the Help us Discover team about this trial and other similar trials, please email or call 877.978.8343

  • Last Updated
  • Study HIC