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Restore CFTR Function Enrolling
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.
Study Design
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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
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Phase: ?more info
Phase Three -
Study Sponsor: ?more info
Vertex -
Study Drugs:
Study Sites
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Enrolling
Alabama
The Children's Hospital Alabama, University of Alabama at Birmingham, Birmingham, AL 35233
Contact
Heather Hathorne
Phone: +1 (205) 638-9568
Email: hhathorne@peds.uab.edu
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Enrolling
California
Stanford University Medical Center, Palo Alto, CA 94304
Contact
Colleen Dunn
Phone: +1 (650) 736-0388
Email: cedunn@stanford.edu
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Enrolling
Georgia
Children’s Healthcare of Atlanta and Emory University, Atlanta, GA 30322
Contact
Eric Hunter
Phone: +1 (404) 727-7398
Email: eahunte@emory.edu
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Enrolling
Illinois
Ann & Robert H. Lurie Children’s Hospital of Chicago, Chicago, IL 60611
Contact
Larissa Rugg
Phone: +1 (312) 227-6865
Email: lrugg@luriechildrens.org
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Enrolling
Indiana
Riley Hospital for Children, Indianapolis, IN 46202
Contact
Lisa Bendy
Phone: +1 (317) 278-7152
Email: lbendy@iupui.edu
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Enrolling
Maryland
Johns Hopkins University, Baltimore, MD 21287
Contact
Britany Zeglin
Phone: +1 (443) 287-8983
Email: bzeglin1@jhmi.edu
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Enrolling
Massachusetts
Boston Children's Hospital, Boston, MA 02115
Contact
Robert Fowler
Phone: +1 (617) 355-1834
Email: Robert.fowler@childrens.harvard.edu
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Enrolling
Missouri
Children's Mercy Kansas City, Kansas City, MO 64108
Contact
Candy Schmoll
Phone: +1 (816) 302-3538
Email: cschmoll@cmh.edu
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Enrolling
Ohio
Nationwide Children's Hospital, Columbus, OH 43205
Contact
Diana Gilmore
Phone: +1 (614) 722-4752
Email: diana.gilmore@nationwidechildrens.org
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Enrolling
Pennsylvania
Children's Hospital of Philadelphia, Philadelphia, PA 19104
Contact
Christina Kubrak
Phone: +1 (267) 426-5135
Email: kubrak@email.chop.edu
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Enrolling
Texas
Baylor College of Medicine, Houston, TX 77030
Contact
Nicoline Schaap
Phone: +1 (832) 822-4252
Email: nxschaap@texaschildrens.org
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Enrolling
Washington
Seattle Children's Hospital, Seattle, WA 98105
Contact
Sharon McNamara
Phone: +1 (206) 987-3921
Email: sharon.mcnamara@seattlechildrens.org
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Enrolling
Wisconsin
University of Wisconsin, Madison, WI 53792
Contact
Melanie Nelson
Phone: +1 (608) 265-4617
Email: mnelson@pediatrics.wisc.edu
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