Interesting new clinical trial for 661/Kalydeco

Was checking to see if next generation details were at clinicaltrials.gov and didn't find but discovered this study, which I hadn't been aware of: https://clinicaltrials.gov/ct2/show/...lmonary&rank=1 It is a small study for 661/Kalydeco, not the Phase 3 for homozygotes going on now, but a Phase 2 looking at "extrapulmonary systems." Primary end point is mucociliary clearance, and secondary endpoints include (beyond FEV), change in gastrointestinal pH, change in sweat chloride, change in nasal potential difference. This is interesting to me because the FDA at the Orcambi discussed (if I recall correctly) that FEV isn't a great marker for whether the drug works. And if they are able to find other markers to show it works, it could mean more can benefit (i.e., heterozygotes or non df508), because it can show other benefits beyond FEV to establish it is working. And this would be true for other drugs in the pipeline throughout the biotech field. fwiw
Source: Cystic Fibrosis Adults Forum - Category: Respiratory Medicine Authors: Tags: Adults Source Type: forums