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Restore CFTR Protein Closed to Enrollment
Phase 3 study of ivacaftor in babies who have a CFTR gating mutation (Vertex VX15-770-124)
This two-part, open-label study is taking place at multiple care centers across the U.S. It will look at the safety and effectiveness of ivacaftor, as well as how the body processes the drug, in babies who have a CFTR gating mutation.
This is a two-part study. Both parts will look at the safety of ivacaftor (Kalydeco) in babies with CF. Part A will last for 10 weeks and look at how the body processes the drug. Part B will last 6 months and will look at the effectiveness of the drug. This study is open-label, meaning that all study participants will receive the study drug. Researchers will study how the body processes ivacaftor (Kalydeco) by measuring the amount of the drug in the bloodstream. They will study the drug’s safety by tracking adverse events and other measures. They will also study the drug's effectiveness by measuring sweat chloride concentration.This study is currently enrolling babies less than 12 months old who have a CFTR gating mutation. It may require blood draws, sweat tests, electrocardiograms (ECGs), eye exams and/or other measurements.
Eligibility
See other primary eligibility criteria for more information.
-
Age:
Less than 13 Months -
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 1 of the following 9 CFTR mutations on at least 1 allele: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D. Subjects who have an R117H-CFTR mutation will be eligible in regions where ivacaftor is approved for use in subjects 2 through 5 years of age with an R117H-CFTR mutation. Subjects who have an ivacaftor-responsive CFTR mutation on at least 1 allele will be
considered eligible for enrollment in Part A/B Cohort 8 (consistent with the approved
mutations in the region).
Study Design
-
Study Type: ?more info
Interventional -
Randomized Study: ?more info
No -
Placebo Controlled: ?more info
No -
Length of Participation:
24 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:
Less than 13 Months -
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 1 of the following 9 CFTR mutations on at least 1 allele: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D. Subjects who have an R117H-CFTR mutation will be eligible in regions where ivacaftor is approved for use in subjects 2 through 5 years of age with an R117H-CFTR mutation. Subjects who have an ivacaftor-responsive CFTR mutation on at least 1 allele will be
considered eligible for enrollment in Part A/B Cohort 8 (consistent with the approved
mutations in the region).
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