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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)
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 Soon
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 Soon
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 Soon
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 Soon
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 Soon
Minnesota
Children’s Hospitals and Clinics of Minnesota, Minneapolis, MN 55404
Contact
Elizabeth Franck Thompson
Phone: +1 (612) 813-6347
Email: elizabeth.franckthompson@childrensmn.org
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Enrolling Soon
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 Soon
New York
Albany Medical College, Albany, NY 12208
Contact
Terese Evans
Phone: +1 (518) 262-5293
Email: evansmt@mail.amc.edu
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Enrolling Soon
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 Soon
Texas
University of Texas Southwestern / Children’s Health, Dallas, TX 75247
Contact
Daniyal Kamal
Phone: +1 (214) 456-5489
Email: Daniyal.Kamal@childrens.com
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Enrolling Soon
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 Soon
Utah
Intermountain 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 Soon
Wisconsin
University of Wisconsin, Madison, WI 53792
Contact
Melanie Nelson
Phone: +1 (608) 265-4617
Email: mnelson@pediatrics.wisc.edu
Clinical Studies 101
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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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How Do Clinical Trials Work
Each clinical trial has a study sponsor and a protocol. Potential drugs must move through four well-defined phases of testing in patients.
