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Ivacaftor in People with CF age 2 to 5 years with a CFTR Gating Mutation (Vertex VX-770-108)
This was an open-label study designed to look at the safety and effectiveness of ivacaftor (Kalydeco®) in children with CF who had at least one copy of a CFTR gating mutation. Participants in Part A received either 50 mg or 75 mg of ivacaftor (based on their weight) twice daily for four days to confirm that the doses were safe and resulted in drug levels anticipated to be effective. Part B started after Part A was complete and the dose levels were confirmed. Participants in Part B received either 50 mg or 75 mg of ivacaftor (based on their weight) twice daily for 24 weeks.
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
Study Results
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What We Learned:
Ivacaftor at doses of 50mg and 75mg (adjusted for weight) appeared to be generally well-tolerated and resulted in decreased sweat chloride in children aged 2-5 years. 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:
Part A of this study recruited participants between January 8, 2013 and March 1, 2013 and Part B, between June 28, 2013 and September 26, 2013. Part A enrolled 9 participants, all of whom completed the treatment period and confirmed the dose levels of 50 mg and 75 mg (based on participant weight) were appropriate for Part B. Part B enrolled 34 participants, of which 33 completed the 24-week treatment period. Part B demonstrated that the decrease in sweat chloride in these young children was similar in magnitude to what was observed in the older participants included in the phase 3 studies of ivacaftor (STRIVE and ENVISION).
Safety:
Ivacaftor at doses of 50mg and 75mg appeared to be generally well-tolerated in children aged 2-5 years. Elevated liver function enzymes were noted for 15% of participants. The rate of elevated liver function enzymes in this 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.
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Citation:
Lancet ;DOI 10.1016/S2213-2600(15)00545-7
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:
32 weeks -
Number of Study Visits:
11
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
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