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Observational Completed with Results
Study of Lung Clearance Index (LCI) in children 3 to 5 years old before and after taking ivacaftor (GOAL e2) (GOAL-OB-11 Cohort 4)
This study evaluated lung function in young children with CF before and after taking ivacaftor. This study was for children ages 3 to 5 years old who have at least one CFTR gating mutation.
This observational study was initiated at the same time that ivacaftor was approved for use in young children (aged 2 to 5 years old). The study evaluated whether ivacaftor would impact Lung Clearance Index (LCI), a way to measure lung function in young children. Lung function, measured by LCI, was evaluated in participants at 1 month and 6 months after starting ivacaftor treatment.
Eligibility
See other primary eligibility criteria for more information.
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Age:
3 Years to 5 Years -
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
At least one allele with a gating mutation
Study Results
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What We Learned:
Study results show that ivacaftor was associated with significant improvement in lung function, as measured by LCI. Ivacaftor was also associated with a reduction in sweat chloride concentration.
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Primary Findings:
Effectiveness:
This study was conducted between March 2015 and June 2016. In this study, 5 participants were enrolled (aged 3 to 5 years old). An identical study conducted in Toronto enrolled 4 participants. Study results from the two studies were combined for analysis.
The primary objective was change in lung function, measured by the relative change in LCI over the course of the study. LCI was significantly improved after 1 month of treatment with ivacaftor (median change from baseline of −23.6%). These improvements were sustained over 6 months (median change from baseline of −24.6%). Additionally, sweat chloride was significantly reduced (mean change from baseline of -46 mmol/L (p<0.001).
Because this was an observational study, no safety measures were evaluated.
Safety:
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Citation:
Am J Respir Crit Care Med 2018;198(4)
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
Phase Four/Post-Approval -
Study Sponsor: ?more info
Rowe, Steven -
Study Drugs:
N/A
Eligibility
See other primary eligibility criteria for more information.
-
Age:
3 Years to 5 Years -
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
At least one allele with a gating mutation
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