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.
Email a research coordinator to express interest in participating in this study.
Eligibility
-
Age:
1 Years to 2 Years -
Mutation(s):
Two Copies F508del -
FEV1% Predicted:
No FEV1 Limit
For a more comprehensive list of eligibility criteria and details on this study, 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
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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
-
Enrolling
Arkansas
Arkansas Children's Hospital, Little Rock, AR 72202
Contact
Sheila Stroupe
Phone: +1 (501) 364-2375
Email: StroupeSL@archildrens.org
-
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
-
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
-
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
-
Enrolling
Indiana
Riley Hospital for Children, Indianapolis, IN 46202
Contact
Lisa Bendy
Phone: +1 (317) 278-7152
Email: lbendy@iupui.edu
-
Enrolling
Maryland
Johns Hopkins University, Baltimore, MD 21287
Contact
Britany Zeglin
Phone: +1 (443) 287-8983
Email: bzeglin1@jhmi.edu
-
Enrolling Soon
Massachusetts
Boston Children's Hospital, Boston, MA 02115
Contact
Robert Fowler
Phone: +1 (617) 355-1834
Email: Robert.fowler@childrens.harvard.edu
-
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
-
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
-
Enrolling
Missouri
Children's Mercy Kansas City, KCMO, MO 64108
Contact
Candy Schmoll
Phone: +1 (816) 302-3538
Email: cschmoll@cmh.edu
-
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
-
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
-
Enrolling
Ohio
Cincinnati Children's Hospital Medical Center, Cincinnati, OH 45229
Contact
Kelly Thornton
Phone: +1 (513) 636-0604
Email: Kelly.Thornton@cchmc.org
-
Enrolling
Pennsylvania
Children's Hospital of Philadelphia, Philadelphia, PA 19104
Contact
Erin Donnelly
Phone: +1 (267) 426-9626
Email: Donnellye4@email.chop.edu
-
Closed to Enrollment
Texas
University of Texas Southwestern / Children’s Health, Dallas, TX 75207
-
Enrolling
Texas
Cook Children's Medical Center, Fort Worth, TX 76104
Contact
Heather Urbanek
Phone: +1 (682) 885-1244
Email: Heather.Urbanek@cookchildrens.org
-
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
-
Enrolling Soon
Washington
Seattle Children's Hospital, Seattle, WA 98105
Contact
Sharon McNamara
Phone: +1 (206) 987-3921
Email: sharon.mcnamara@seattlechildrens.org
-
Enrolling
Wisconsin
University of Wisconsin, Madison, WI 53792
Contact
Melanie Nelson
Phone: +1 (608) 265-4617
Email: mnelson@pediatrics.wisc.edu
Email a research coordinator to express interest in participating in this study.
Eligibility
-
Age:
1 Years to 2 Years -
Mutation(s):
Two Copies F508del -
FEV1% Predicted:
No FEV1 Limit
For a more comprehensive list of eligibility criteria and details on this study, visit ClinicalTrials.gov.
Related Topics
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Questions to Ask When Considering a Specific Trial
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Patient Safety Is a Priority
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