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N/A Completed N=8 Basic Science

(Study: Vertex IIS) Does Ivacaftor Alter Wild Type CFTR-Open Probability In The Sweat Gland Secretory Coil?

Source: ClinicalTrials.gov NCT02310789 ↗
Enrolled (actual)
8
Serious AEs
0.0%
Results posted
Apr 2018
Primary outcomePrimary: Change in Cystic Fibrosis Transmembrane Conductance Regulator (CFTR)-Dependent Sweat Rate — 16.45; 6.60 percent change

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.

Outcome Measures

OutcomeResultp-value
PRIMARY
Change in Cystic Fibrosis Transmembrane Conductance Regulator (CFTR)-Dependent Sweat Rate
16.45; 6.60
SECONDARY
Change Sweat Chloride Production
25.1; 24.9

Eligibility Criteria

Inclusion Criteria

  • Healthy adults without a Cystic Fibrosis (CF) mutation
  • Carriers with a known CF mutation

Exclusion Criteria

  • Documented liver disease
  • 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.
View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT02310789). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.

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