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Restore CFTR Protein Completed with Results
Roll-over study of ivacaftor in children with CF who have a CFTR gating mutation (Vertex VX-770-109)
This study evaluated the long-term safety of ivacaftor (Kalydeco®) in children with CF. This study was for children who have a CFTR gating mutation and received at least one dose of ivacaftor in the VX11-770-108 study.
Participants were given the option to enroll in either an ivacaftor group or an observational group. All participants in this study enrolled in the ivacaftor group and none in the observational group. In the ivacaftor group, participants received ivacaftor every 12 hours for 84 weeks. The doses given were dependent on the child’s weight (50mg every 12 hours for participants 2 to <6 years of age and <14kg; 75mg every 12 hours for participants 2 to <6 years of age and ≥14kg; 150mg every 12 hours for participants ≥6 years of age). Doses of ivacaftor were adjusted at each visit if necessary as age or weight changed.
Eligibility
See other primary eligibility criteria for more information.
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Age:
2 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
Eligible subjects must have a CFTR Gating Mutation in at least one allele and have participated in VX 770-108 Study.
Study Results
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What We Learned:
This study found that ivacaftor appeared to be generally well-tolerated at doses of 50mg and 75mg (adjusted for weight). It resulted in decreased sweat chloride in children aged 2-5 years, similar to what was seen in the VX11-770-108 study. Frequent monitoring of liver function should occur in young children using this drug, particularly those with a history of elevated liver function enzymes.
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Primary Findings:
Effectiveness:
Safety:
This study was conducted between February 2014 and December 2015. In this study, 33 participants were enrolled and 28 completed (n=9 ivacaftor 50mg; n=24 ivacaftor 75mg). Of the 5 that did not complete the study, one stopped participating due to an adverse event.
The primary endpoint was safety and tolerability from the beginning of the study to week 97. Researchers assessed the number of participants who experienced an adverse event that developed during treatment (TEAEs) or a serious adverse (SAEs) event.
Ivacaftor appeared to be generally well-tolerated at doses of 50mg and 75mg, similar to what was seen in the VX11-770-108 study. The rate of elevated liver function enzymes in the 50mg group indicated that frequent monitoring of liver function should occur in young children using this drug, particularly those with a history of elevated liver function enzymes.
Additionally, ivacaftor reduced sweat chloride over the 84 weeks (-2.4 ivacaftor 50mg and -11.2 ivacaftor 75mg). Overall, from before the start of the 108 study to the end of the 109 study, sweat chloride reduced by -46.5 at the 50mg dose and -58.1 in the 75mg dose. Ivacaftor also improved the weight, height, and BMI of the participants.
These results have been provided from Clinicaltrials.gov.
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Citation:
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
Interventional -
Randomized Study: ?more info
No -
Placebo Controlled: ?more info
No -
Length of Participation:
88 weeks -
Number of Study Visits:
7
Additional Information
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Phase: ?more info
Phase Three -
Study Sponsor: ?more info
Vertex -
Study Drugs:
Eligibility
See other primary eligibility criteria for more information.
-
Age:
2 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
Eligible subjects must have a CFTR Gating Mutation in at least one allele and have participated in VX 770-108 Study.
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