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Study Name: Ivacaftor in People with CF age 2 to 5 years with a CFTR Gating Mutation (VX 770-108)
Study Type: Interventional
Intervention Category: CFTR Modulation
Study Sponsor: Vertex
Study Phase: 3
Recruitment Status: Trial Completed
Study Drug(s): Kalydeco
Number of Participants Being Recruited: 28
Single / Multi-Center: Multi-Center
This is an open-label study designed to look at the safety, pharmacokinetics (PK), pharmacodynamics(PD), and effectiveness of ivacaftor in people with CF who are 2 through 5 years of age and have a CFTR gating mutation in at least 1 allele. Part A is designed to look at the safety and PK of multiple-dose administration of ivacaftor in children 2 through 5 years of age and to confirm the doses for Part B. Part B is designed to look at the safety, PK, PD, and efficacy of ivacaftor in children 2 through 5 years of age.

Ivacaftor is currently approved by the US FDA and the EMA under the trade name Kalydeco® for the treatment of cystic fibrosis in patients 6 years of age and older who have the G551D-CFTR mutation
Age: 2 Years - 5 Years
FEV1: Not Applicable
P. aeruginosa status: Not applicable
B. cepacia status: Not applicable
Other Primary Eligibility Requirements:

Eligible subjects must have a CFTR Gating Mutation in at least one allele
Note: Detailed eligibility criteria information may be available on If a specific trial listing for this trial is available, a link to the specific listing will be present in the "More Information" section below.
Sponsor Contact Information: Medical Monitor, Vertex
(617) 444-6777
Trial Specific Link on
Clinical Research Terms Glossary: Click here
The sites listed below are currently recruiting subjects. Please note that this list generally lists either the pediatric or adult program but not both even though both programs may be recruiting subjects. Also, this list does not include sites that may be participating in the study but are not yet open for recruitment. If you are interested in this study please contact your center for more information.
  • The Children's Hospital/University of Alabama at Birmingham (Pediatric), Birmingham, AL, 35233
    Contact Information:
        Hathorne, Heather
        Phone: (205) 638-9568
  • Children's Hospital Colorado (Pediatric), Aurora, CO, 80045
    Contact Information:
        Pardee, Churee
        Phone: (720) 777-6162
  • Emory University (Pediatric), Atlanta, GA, 30322
    Contact Information:
        Hunter, Eric
        Phone: (404) 785-3102
  • Riley Hospital for Children Indiana University Medical Center (Pediatric), Indianapolis, IN, 46202
    Contact Information:
        Bendy, Lisa
        Phone: (317) 948-7152
  • Children's Hospital Boston (Pediatric), Boston, MA, 02115
    Contact Information:
        Fowler, Robert
        Phone: (617) 355-1834
  • Massachusetts General Hospital (Pediatric), Boston, MA, 02114
    Contact Information:
        Doolittle, Caitlin
        Phone: (617) 726-3719
  • The Minnesota Cystic Fibrosis Center (Pediatric), Minneapolis, MN, 55455
    Contact Information:
        Grover, Patricia
        Phone: (612) 626-7425
  • The Children's Mercy Hospital (Pediatric and Adult), Kansas City, MO, 64108
    Contact Information:
        Schmoll, Candy
        Phone: (816) 701-1339
  • Children's Hospital of Pittsburgh of UPMC (Pediatric), Pittsburgh, PA, 15213
    Contact Information:
        Hartigan, Elizabeth
        Phone: (412) 692-7060
  • Intermountain Cystic Fibrosis Center (Pediatric), Salt Lake City, UT, 84132
    Contact Information:
        Vroom, Jane
        Phone: (801) 587-7458
  • Virginia Commonwealth University (Pediatric), Richmond, VA, 23235
    Contact Information:
        Ward, Matthew
        Phone: (804) 628-3093
  • Seattle Children's Hospital (Pediatric), Seattle, WA, 98105
    Contact Information:
        McNamara, Sharon
        Phone: (206) 987-3921 X1
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