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Observational Completed with Results
Observational Study in People with CF with the G551D mutation (GOAL-OB-11)
This was a multi-center observational trial to collect clinical information (including height, weight and lung function) as well as specimens (to store and use for future research) in people with CF who have the G551D mutation. The study was initiated at the same time that ivacaftor was approved for use in people with CF and the G551D mutation. The study sought to evaluate response to ivacaftor in the clinical care setting and to evaluate some new potential outcome measures. Additionally biospecimens (blood, urine, and sputum samples) were collected and stored for future research. Researchers for future cystic fibrosis research may use the banked specimens and the health information to develop tests that could be used to help identify additional drugs that can help people with CF. Some participants also had additional procedures performed such as sweat rate testing, ability to clear mucus from the lungs, induced sputum collection, or taking a gastrointestinal pH pill to begin to evaluate the usefulness of these measures for future studies.
Eligibility
See other primary eligibility criteria for more information.
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Age:
6 Years and Older -
Mutation(s):
One Copy F508del or No Copies F508del -
FEV1% Predicted:
No FEV1 Limit
For more information about the results of this study and where it was conducted, visit ClinicalTrials.gov.
Other Primary Eligibility Criteria
Must have the G551D-CFTR mutation on at least 1 allele (any known or unknown mutations allowed in second allele).
Study Results
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What We Learned:
Study results show that ivacaftor was associated with significant improvements in lung function, body weight and mass, and sweat chloride concentration.
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Primary Findings:
Effectiveness:
This study was conducted between February 2012 and January 2013. Clinical measures and biospecimens were collected from 153 CF patients aged six years and older with at least one G551D mutation and no prior exposure to ivacaftor. Of the 153 participants, 151 were prescribed and received ivacaftor and 133 were followed for 6 months after their first dose. Statistically significant improvements were observed from before taking ivacaftor to after 6 months of treatment in lung function (6.7% improvement in FEV1% predicted [p<0.001]), body weight and body mass, and sweat chloride (mean change from baseline of -53.6 mmol/L to (p<0.001). There were also significant improvements observed in hospitalization rate (P < 0.001) and Pseudomonas aeruginosa burden (P < 0.01) relative to historical control data. Additionally the experimental outcome measures of mucociliary clearance and gastrointestinal pH were also improved.
Safety:
Because this was an observational study no safety measures were evaluated.
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Citation:
Am J Respir Crit Care Med 2014;DOI 10.1164/rccm.201404-0703OC;190(2):175-184
For current information about the overall development status of this drug, please check the Drug Development Pipeline.
Study Design
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Study Type: ?more info
Observational -
Randomized Study: ?more info
No -
Placebo Controlled: ?more info
No -
Length of Participation:
6 months -
Number of Study Visits:
5
Additional Information
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Phase: ?more info
Not Applicable -
Study Sponsor: ?more info
Rowe, Steven -
Study Drugs:
N/A
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:
No FEV1 Limit
For more information about the results of this study and where it was conducted, visit ClinicalTrials.gov.
Other Primary Eligibility Criteria
Must have the G551D-CFTR mutation on at least 1 allele (any known or unknown mutations allowed in second allele).
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