(Study: Vertex IIS) Does Ivacaftor Alter Wild Type CFTR-Open Probability In The Sweat Gland Secretory Coil?
Status:
Completed
Trial end date:
2017-08-23
Target enrollment:
Participant gender:
Summary
Clinical studies of lumacaftor + ivacaftor (combo therapy) produced better FEV1 (forced
expiratory volume in 1 second) improvements than ivacaftor alone, without further improvement
in sweat chloride results.
To help understand why sweat chloride was unresponsive, the investigators will use a newly
developed sweat secretion test that provides accurate, in vivo readout of CFTR (cystic
fibrosis transmembrane conductance regulator) function in the sweat gland secretory coil.
The investigators devised a protocol to determine if short courses of ivacaftor (3.5 days)
will produce significant increases in WT (Wild-Type, i.e. normal) CFTR open probability by
measuring CFTR-dependent sweating (C-sweat) in subjects with WT CFTR.