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Clinical Trial Finder
Restore CFTR Protein Closed to Enrollment
Phase 3 study of lumacaftor/ivacaftor (Orkambi®) in babies with two copies of the F508del CFTR mutation (Part B) (Vertex VX-16-809-122 Part B)
This study is taking place at multiple care centers across the U.S. It will evaluate the safety of the drug lumacaftor/ivacaftor (Orkambi®) and its effect on the body. It is for babies ages 1 to less than 2 years old who have cystic fibrosis and two copies of the F508del CFTR mutation.
This study is open-label, meaning all participants will receive the lumacaftor/ivacaftor study drug. Researchers will assess the safety of the drug lumacaftor/ivacaftor by monitoring for adverse events. Researchers will also measure how long the drugs stay in the body.This study may require sweat tests, blood draws, and/or other measurements.
Eligibility
-
Age:
1 Years to 2 Years -
Mutation(s):
Two Copies F508del -
FEV1% Predicted:
No FEV1 Limit
For more information about the results of this study and where it was conducted, visit ClinicalTrials.gov.
Study Design
-
Study Type: ?more info
Interventional -
Randomized Study: ?more info
No -
Placebo Controlled: ?more info
No -
Length of Participation:
38 weeks -
Number of Study Visits:
11
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
Arkansas
Arkansas Children's Hospital, Little Rock, AR 72202
-
Closed to Enrollment
Colorado
Children's Hospital Colorado, Aurora, CO 80045
-
Closed to Enrollment
Connecticut
Yale University School of Medicine, New Haven, CT 06520
-
Closed to Enrollment
Delaware
Alfred I. duPont Hospital for Children, Wilmington, DE 19803
-
Closed to Enrollment
Georgia
Children’s Healthcare of Atlanta and Emory University, Atlanta, GA 30322
-
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
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
SSM Health Cardinal Glennon Children’s Hospital, St. Louis, MO 63104
-
Closed to Enrollment
Missouri
Children's Mercy Kansas City, Kansas City, MO 64108
-
Closed to Enrollment
North Carolina
University of North Carolina at Chapel Hill, Chapel Hill, NC 27599
-
Closed to Enrollment
North Carolina
Wake Forest University Baptist Medical Center, Winston-Salem, NC 27157
-
Closed to Enrollment
Ohio
Cincinnati Children's Hospital Medical Center, Cincinnati, OH 45229
-
Closed to Enrollment
Pennsylvania
Children's Hospital of Philadelphia, Philadelphia, PA 19104
-
Closed to Enrollment
Texas
University of Texas Southwestern / Children’s Health, Dallas, TX 75207
-
Closed to Enrollment
Texas
Cook Children's Medical Center, Fort Worth, TX 76104
-
Closed to Enrollment
Utah
Primary Children's Cystic Fibrosis Center, Salt Lake City, UT 84113
-
Closed to Enrollment
Washington
Seattle Children's Hospital, Seattle, WA 98105
-
Closed to Enrollment
Wisconsin
University of Wisconsin, Madison, WI 53792
Eligibility
-
Age:
1 Years to 2 Years -
Mutation(s):
Two Copies F508del -
FEV1% Predicted:
No FEV1 Limit
For more information about the results of this study and where it was conducted, visit ClinicalTrials.gov.
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