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Restore CFTR Protein Closed to Enrollment
Phase 3 study of long-term ivacaftor in babies who have a CFTR gating mutation (Vertex VX-770-126)
This open-label study is taking place at multiple care centers across the U.S. It will look at the safety and effectiveness of long-term ivacaftor in babies who have a CFTR gating mutation.
This study is open-label, meaning that all participants will receive the study drug. There is also an observational arm open to people who participated in the VX-770-124 study; people who volunteer for this arm will not receive the study drug. Researchers 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 for babies under two years old who have a CFTR gating mutation. It may require sweat tests and/or other measurements.
Eligibility
See other primary eligibility criteria for more information.
-
Age:
Less than 25 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
Subjects with CF who have 1 of the following 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.
Study Design
-
Study Type: ?more info
Interventional -
Randomized Study: ?more info
No -
Placebo Controlled: ?more info
No -
Length of Participation:
128 weeks -
Number of Study Visits:
18
Additional Information
-
Phase: ?more info
Phase Three -
Study Sponsor: ?more info
Vertex -
Study Drugs:
Study Sites
-
Closed to Enrollment
Alabama
The Children's Hospital Alabama, University of Alabama at Birmingham, Birmingham, AL 35233
-
Closed to Enrollment
California
Stanford University Medical Center, Palo Alto, CA 94304
-
Closed to Enrollment
Delaware
Alfred I. duPont Hospital for Children, Wilmington, DE 19803
-
Closed to Enrollment
Florida
The Nemours Children's Clinic - Orlando, Orlando, FL 32827
-
Closed to Enrollment
Georgia
Emory University, Atlanta, GA 30324
-
Closed to Enrollment
Illinois
Ann & Robert H. Lurie Children’s Hospital of Chicago, Chicago, IL 60611
-
Closed to Enrollment
Indiana
Riley Hospital for Children, Indianapolis, IN 46202
-
Closed to Enrollment
Maryland
Johns Hopkins University, Baltimore, MD 21287
-
Closed to Enrollment
Massachusetts
Boston Children's Hospital, Boston, MA 02115
-
Closed to Enrollment
Minnesota
Children’s Hospitals and Clinics of Minnesota, Minneapolis, MN 55404
-
Closed to Enrollment
Missouri
Children's Mercy Kansas City, Kansas City, MO 64108
-
Closed to Enrollment
Montana
Billings Clinic , Billings, MT 59101
-
Closed to Enrollment
Ohio
Nationwide Children's Hospital, Columbus, OH 43205
-
Closed to Enrollment
Pennsylvania
Children's Hospital of Philadelphia, Philadelphia, PA 19104
-
Closed to Enrollment
Texas
Baylor College of Medicine, Houston, TX 77030
-
Closed to Enrollment
Washington
Seattle Children's Hospital, Seattle, WA 98105
-
Closed to Enrollment
Wisconsin
University of Wisconsin, Madison, WI 53792
Eligibility
See other primary eligibility criteria for more information.
-
Age:
Less than 25 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
Subjects with CF who have 1 of the following 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.
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