The CF Foundation recently provided an update on the following research trial.
GOAL e2 Lung Clearance Index Study in children ages 3-5 who have a CFTR gating mutation
This is a Phase 4 prospective observational trial. It will evaluate Lung Clearance Index (LCI) as an outcome measure for young children with CF.
The recent approval of Kalydeco for children ages 2 to 5 with at least one gating mutation provides an unprecedented opportunity for researchers to evaluate if this measurement could be used in future studies for this age group. The study will measure LCI in children before and after taking ivacaftor.
This trial is for children with CF ages 3 to 5 years at the time of enrollment who have at least one gating mutation. The trial will last 6 months.
- CFF Link: https://tools.cff.org/Display/dsp_ClinicalResearchHTML.cfm?id=430&IDH=9154082
- ClinicalTrials.gov Link: https://clinicaltrials.gov/ct2/show/NCT01521338