Page Title
Clinical Trial Finder
Restore CFTR Protein Completed with Results
Ivacaftor in People with CF who have the R117H-CFTR mutation (KONDUCT) (Vertex VX-770-110)
This study looked at the safety and effectiveness of ivacaftor in people with CF who have the R117H-CFTR mutation. Study participants were randomized to receive placebo or ivacaftor (150 mg every 12 hours) for 24 weeks. Ivacaftor is the first cystic fibrosis transmembrane regulator (CFTR) modulator to show an improvement in CFTR function and clinical benefit in people with CF. Ivacaftor was initially approved in the United States for the treatment of CF in patients 6 years of age and older who have a G551D mutation in the CFTR gene.
Eligibility
See other primary eligibility criteria for more information.
-
Age:
6 Years and Older -
Mutation(s):
One Copy F508del or No Copies F508del -
FEV1% Predicted:
40 to 105%
For more information about the results of this study and where it was conducted, visit ClinicalTrials.gov.
Other Primary Eligibility Criteria
Eligible patients must have the R117H mutation on at least one allele
Study Results
-
What We Learned:
Study results show that there was no significant improvement in lung function in the treatment group compared to the placebo group. The treatment group did show improvement in other outcome measures. Additionally, the frequency of adverse events was similar in both groups.
-
Primary Findings:
Effectiveness:
This multi-center study was conducted between July 2012 and October 2013. A total of 69 were enrolled and 94% of participants receiving ivacaftor and 100% of participants receiving placebo completed the study. The primary outcome was the absolute change from baseline in percent predicted FEV1 through week 24. This study did not show clinically significant improvement in percent predicted FEV1 with ivacaftor as compared to the placebo group (treatment difference = 2.1 %).
Ivacaftor treatment resulted in significant improvement in sweat chloride and quality of life scores (CFQ-R respiratory domain). Additionally, when the lung function data were analyzed by age-group, participants that were 18 years of age and older did have a statistically significant improvement of 5% in FEV1 percent predicted.
Safety:
The incidence of adverse events was similar between the ivacaftor and placebo groups in the study with most adverse events being mild or moderate.
-
Citation:
Lancet 2015;DOI 10.1016/S2213-2600(15)00201-5;3(7):524-533
For current information about the overall development status of this drug, please check the Drug Development Pipeline.
Study Design
-
Study Type: ?more info
Interventional -
Randomized Study: ?more info
Yes -
Placebo Controlled: ?more info
Yes -
Length of Participation:
28 weeks -
Number of Study Visits:
8
Additional Information
-
Phase: ?more info
Phase Three -
Study Sponsor: ?more info
Vertex -
Study Drugs:
Eligibility
See other primary eligibility criteria for more information.
-
Age:
6 Years and Older -
Mutation(s):
One Copy F508del or No Copies F508del -
FEV1% Predicted:
40 to 105%
For more information about the results of this study and where it was conducted, visit ClinicalTrials.gov.
Other Primary Eligibility Criteria
Eligible patients must have the R117H mutation on at least one allele
Sign up for clinical trial alerts
Get email updates about clinical trials that matter to you.
Check the Drug Development Pipeline
We’re attacking CF from every angle. Learn about the status of CF drugs in development.
Learn More