G551D Observational Study (GOAL)
This is a prospective, multi-center observational study. The study is designed to collect specimens from and perform functional measurements on cystic fibrosis subjects with the G551D CFTR mutation, many of whom are expected to be prescribed ivacaftor during the source of the study.
|Study Design:||Observational Model: Cohort
Time Perspective: Prospective
|Official Title:||G551D Observational Study (GOAL)|
- No Primary Outcomes are collected for this study [ Designated as safety issue: No ]
Biospecimen Retention: Samples With DNA
Blood, Sputum, Urine
|Study Start Date:||February 2012|
|Estimated Study Completion Date:||March 2014|
|Estimated Primary Completion Date:||March 2013 (Final data collection date for primary outcome measure)|
Specimens (blood, sputum, and urine) will be collected and spirometry and sweat chloride testing will be performed at Visit 1. Subjects who begin ivacaftor during the 12 month testing will be performed at Visit 1. Subjects who begin ivacaftor during the 12 month enrollment period of the study will have specimens and test performed within 30 days prior to ivacftor initiation (visit 2) and at 1 month (Visit 3), 3 months (Visit 4) and 6 months (Visit 5) after the start of ivacaftor administration. Subjects who do not begin administration of ivacaftor during the 12-month enrollment period of the study will have the same specimens and tests performed at a follow-up visit after the close of the 12-month enrollment period of the study (visit 1B).
Show 29 Study Locations
|Principal Investigator:||Steven M Rowe, MD||University of Alabama at Birmingham|