(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: 0
• Participant gender: All

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.

• Phase: N/A
• Accepts Healthy Volunteers?: Accepts Healthy Volunteers

Details

• Lead Sponsor: Richard Barry Moss
• Treatments: Adrenergic Agents; Ivacaftor; Pilocarpine

Criteria

Inclusion Criteria:

• Healthy adults without a Cystic Fibrosis (CF) mutation

• Carriers with a known CF mutation

Exclusion Criteria:

1. Documented liver disease

2. Participants should not be taking:

• medicines that are strong CYP3A (Cytochrome P450, family 3, subfamily A) inducers, such as:

• the antibiotics rifampin and rifabutin;

• seizure medications (phenobarbital, carbamazepine, or phenytoin); and

• the herbal supplement St. John's Wort, substantially decreases exposure of ivacaftor and may diminish effectiveness.