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Clinical Trial Finder
Restore CFTR Function Enrolling
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.
Study Design
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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
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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
Arkansas
Arkansas Children's Hospital, Little Rock, AR 72202
Contact
Sheila Stroupe
Phone: +1 (501) 364-2375
Email: StroupeSL@archildrens.org
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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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Closed to Enrollment
Colorado
Children's Hospital Colorado, Aurora, CO 80045
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Enrolling
Connecticut
Yale University School of Medicine, New Haven, CT 06520
Contact
Catalina Guzman
Phone: +1 (203) 785-4971
Email: catalina.guzman@yale.edu
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Enrolling Soon
Delaware
Alfred I. duPont Hospital for Children, Wilmington, DE 19803
Contact
Kimberly Renner
Phone: +1 (302) 651-6536
Email: Kimberly.renner@nemours.org
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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 Soon
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
Minnesota
Children’s Hospitals and Clinics of Minnesota, Minneapolis, MN 55404
Contact
Camerone Bey
Phone: +1 (612) 813-7628
Email: Camerone.bey@childrensmn.org
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Enrolling
Missouri
SSM Health Cardinal Glennon Children’s Hospital, St. Louis, MO 63104
Contact
Freda Branch
Phone: +1 (314) 678-5468
Email: freda.branch@health.slu.edu
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Enrolling
Missouri
Children's Mercy Kansas City, KCMO, MO 64108
Contact
Candy Schmoll
Phone: +1 (816) 302-3538
Email: cschmoll@cmh.edu
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Enrolling Soon
New York
University of Rochester Medical Center Strong Memorial, Rochester, NY 14642
Contact
Barb Johnson
Phone: +1 (585) 276-4123
Email: Barbara_Johnson@URMC.Rochester.edu
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Enrolling Soon
North Carolina
University of North Carolina at Chapel Hill, Chapel Hill, NC 27599
Contact
Margret Powell
Phone: +1 (984) 974-2962
Email: margret_powell@med.unc.edu
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Enrolling
North Carolina
Wake Forest University Baptist Medical Center, Winston-Salem, NC 27103
Contact
Patricia Whalen
Phone: +1 (336) 713-9190
Email: pwhalen@wakehealth.edu
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Enrolling
Ohio
Cincinnati Children's Hospital Medical Center, Cincinnati, OH 45229
Contact
Kelly Thornton
Phone: +1 (513) 636-0604
Email: Kelly.Thornton@cchmc.org
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Enrolling
Pennsylvania
Children's Hospital of Philadelphia, Philadelphia, PA 19104
Contact
Erin Donnelly
Phone: +1 (267) 426-9626
Email: Donnellye4@email.chop.edu
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Closed to Enrollment
Texas
University of Texas Southwestern / Children’s Health, Dallas, TX 75207
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Enrolling
Texas
Cook Children's Medical Center, Fort Worth, TX 76104
Contact
Heather Urbanek
Phone: +1 (682) 885-1244
Email: Heather.Urbanek@cookchildrens.org
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Enrolling
Utah
Primary Children's Cystic Fibrosis Center, Salt Lake City, UT 84113
Contact
Jane Vroom
Phone: +1 (801) 587-7458
Email: jane.vroom@hsc.utah.edu
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Enrolling Soon
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
Related Topics
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Questions to Ask When Considering a Specific Trial
Clinical trials that test potential drugs and therapies in people with cystic fibrosis are a major part of CF research. They take place at Cystic Fibrosis Foundation-accredited care centers all over the United States and enroll people with CF of all ages.
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Patient Safety Is a Priority
Nothing is more important than patient safety in developing new treatments for cystic fibrosis. The U.S. government requires that all clinical trials are supervised for safety, and the Cystic Fibrosis Foundation adds additional measures to protect study volunteers.
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Trikafta™
The following is a collection of information and news about Trikafta™, the triple-combination modulator therapy that was approved by the U.S. Food and Drug Administration on Oct. 21, 2019.
