Clinical trial NCT01262352: Study of the Effect of Ivacaftor on Lung Clearance Index in Subjects With Cystic Fibrosis and the G551D Mutation
Official 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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Cystic Fibrosis
Lead sponsor: Vertex Pharmaceuticals Incorporated
Sponsor class: INDUSTRY
Collaborators: name: Cystic Fibrosis Foundation
First posted: 2010-12-17
Enrollment: 21
Sex: ALL
Minimum age (years): 6
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: CROSSOVER
Primary purpose: TREATMENT

Brief summary: 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.

Detailed description: 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.

Eligibility criteria: 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

Primary outcomes:
measure: Absolute Change From Baseline in Lung Clearance Index (LCI)
timeFrame: Baseline through Day 29
Secondary outcomes:
measure: Absolute Change From Baseline in Percent Predicted FEV1
timeFrame: Baseline through Day 29
measure: Change From Baseline in Sweat Chloride
timeFrame: Baseline through Day 29
measure: Change From Baseline in CF Questionnaire-Revised (CFQ-R) Score (Respiratory Domain Score, Pooled)
timeFrame: Baseline through Day 29
Other outcomes:
Locations:
city: Stanford
country: United States
state: California
city: Iowa City
country: United States
state: Iowa
city: Durham
country: United States
state: North Carolina
city: Pittsburgh
country: United States
state: Pennsylvania
city: Toronto
country: Canada
state: Ontario
city: Belfast
country: United Kingdom
city: Edinburgh
country: United Kingdom
city: London
country: United Kingdom
Study officials:
affiliation: Royal Brompton Hospital and Imperial College
name: Jane Davies
role: PRINCIPAL_INVESTIGATOR
affiliation: The Hospital for Sick Children
name: Felix Ratjen
role: PRINCIPAL_INVESTIGATOR
References:
citation: Davies J, Sheridan H, Bell N, Cunningham S, Davis SD, Elborn JS, Milla CE, Starner TD, Weiner DJ, Lee PS, Ratjen F. Assessment of clinical response to ivacaftor with lung clearance index in cystic fibrosis patients with a G551D-CFTR mutation and preserved spirometry: a randomised controlled trial. Lancet Respir Med. 2013 Oct;1(8):630-638. doi: 10.1016/S2213-2600(13)70182-6. Epub 2013 Sep 10.