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Completed

NCT Number: NCT01262352

Study of the Effect of Ivacaftor on Lung Clearance Index in Subjects With Cystic Fibrosis and the G551D Mutation

The purpose of this study is to evaluate the effect of ivacaftor (VX-770) on lung clearance index (LCI) in subjects aged 6 years and older with cystic fibrosis (CF) who have the G551D-CFTR mutation on at least 1 allele.

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Key information

Conditions

Cystic Fibrosis Congenital, Hereditary, and Neonatal Diseases and Abnormalities Digestive System Diseases Genetic Diseases, Inborn Infant, Newborn, Diseases Lung Diseases Pancreatic Diseases Respiratory Tract Diseases

Age range

6 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Toronto, Ontario, Canada

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About this study

Currently, limited objective measures are available to quantify lung function in CF patients with mild lung disease. Lung clearance index (LCI) derived from inert gas multiple-breath washout (MBW) testing hold considerable promise to evaluate early lung disease as studies have detected abnormalities in a high percentage of CF patients with normal spirometry in both infants and children.

This study explored the effect of ivacaftor on LCI and the efficacy of ivacaftor on other clinical and biomarker endpoints of CF lung disease in subjects aged 6 years and older with CF who have the G551D-CFTR mutation on at least 1 allele.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Male or female subjects with confirmed diagnosis of CF
  • Must have the G551D-CFTR mutation in at least 1 allele
  • FEV1 >90% of predicted normal for age, gender, and height

Exclusion criteria

  • Ongoing participation in another therapeutic clinical study or prior participation in an investigational drug study within the 30 days prior to screening
  • Use of inhaled hypertonic saline treatment within 2 weeks of the Period 1, Day 1 visit

Treatment and study plan

Ivacaftor

Drug

150 mg tablet, oral use, twice daily every 12 hours (q12h)

Placebo

Drug

Tablet, oral use, twice daily every 12 hours (q12h)

Primary outcomes

  1. Absolute Change From Baseline in Lung Clearance Index (LCI)

    Time frame: Baseline through Day 29

    Lung clearance index (LCI) is a measure of ventilation inhomogeneity that is derived from a multiple-breath washout test. The LCI was calculated as the number of lung volume turnovers (cumulative expired volume divided by the functional residual capacity [FRC]) required to reduce end-tidal SF6 concentration to 1/40th of the starting value.

Secondary outcomes

  1. Absolute Change From Baseline in Percent Predicted FEV1

    Time frame: Baseline through Day 29

    Spirometry (as measured by FEV1) is a standardized assessment to evaluate lung function that is the most widely used endpoint in cystic fibrosis studies.

  2. Change From Baseline in Sweat Chloride

    Time frame: Baseline through Day 29

    The sweat chloride (quantitative pilocarpine iontophoresis) test is a standard diagnostic tool for cystic fibrosis (CF), serving as an indicator of cystic fibrosis transmembrane conductance regulator (CFTR) activity.

  3. Change From Baseline in CF Questionnaire-Revised (CFQ-R) Score (Respiratory Domain Score, Pooled)

    Time frame: Baseline through Day 29

    The CFQ-R is a health-related quality of life measure for subjects with cystic fibrosis. Each domain is scored from 0 (worst) to 100 (best). A difference of at least 4 points in the respiratory domain score of the CFQ-R is considered a minimal clinically important difference (MCID). The primary analytical focus was the respiratory health domain, which was analyzed by combining all self-response questionnaire versions from different age groups (e.g., Adult/Adolescent and Child versions).

Sponsors and collaborators

Lead sponsor

Vertex Pharmaceuticals Incorporated

Industry

Collaborators

  • Cystic Fibrosis Foundation

Registry information

Official study title

A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Crossover Study to Evaluate the Effect of VX-770 on Lung Clearance Index in Subjects With Cystic Fibrosis, the G551D Mutation, and FEV1 >90% Predicted

Important dates

Study start
2011
Primary completion
2011
Study completion
2011
First posted
Dec 17, 2010
Registry last updated
Feb 11, 2013

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

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This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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