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|
| Study Name: |
Ivacaftor (Kalydeco) in People with CF who have a Non G551D Gating Mutation (KONNECTION) |
| Study Type: |
Interventional |
| Intervention Category: |
CFTR Modulation |
| Study Sponsor: |
Vertex |
| Study Phase: |
3 |
| Recruitment Status: |
Recruiting |
| Study Drug(s): |
Kalydeco |
| Number of Participants Being Recruited: |
40 |
| Single / Multi-Center: |
Multi-Center |
| STUDY BACKGROUND INFORMATION: |
| Ivacaftor (Trade Name Kalydeco) is the first CFTR modulator to show an improvement in CFTR function and clinical benefit in patients with CF. Ivacaftor was initially approved in the United States for the treatment of CF in people 6 years of age and older who have a G551D mutation in the CFTR gene. This trial will look at the safety and effectiveness of ivacaftor in people with CF who are 6 years of age and older and have a non G551D gating mutation (any 1 of the following: G178R, G551S, S549N, S549R, G970R, G1244E, S1251N, S1255P, or G1349D). |
| Age: |
>= 6 Years
|
| FEV1: |
40 - 105 Percent Predicted
|
| P. aeruginosa status: |
Not applicable |
| B. cepacia status: |
Not applicable |
Other Primary Eligibility Requirements:
Eligible subjects must have at least 1 allele of the following CFTR gating mutations: G178R, S549N, S549R, G551S, G970R, G1244E, S1251N, S1255P, G1349. Eligible subjects cannot have the G551 mutation in either allele.
|
| Note: Detailed eligibility criteria information may be available on clinicaltrials.gov. If a specific
trial listing for this trial is available, a link to the specific clinicaltrials.gov listing will be present in the "More Information" section below. |
| Sponsor Contact Information: |
Medical Monitor, Vertex
(617) 444-6777
medicalinfo@vrtx.com
|
| PARTICIPATING SITES: |
| 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. |
| FL |
-
Tampa General Hospital (Adult), Tampa, FL, 33606
Contact Information:
Roberson, Suzanne
Phone: (813) 844-7179
Email: sroberson@tgh.org
|
| GA |
-
Emory University (Pediatric), Atlanta, GA, 30322
Contact Information:
Hunter, Eric
Phone: (404) 727-9960
Email: eahunte@emory.edu
|
| IL |
-
Ann and Robert H. Lurie Children's Hospital of Chicago (Pediatric), Chicago, IL, 60611
Contact Information:
Singh, Noopur
Phone: (312) 227-6861
Email: nosingh@luriechildrens.org
|
| MA |
|
|
| MI |
-
University of Michigan Health System (Pediatric), Ann Arbor, MI, 48109
Contact Information:
Kruse, Dawn
Phone: (734) 615-3266
Email: dmkruse@umich.edu
|
| MN |
-
Children's Hospitals and Clinics (Pediatric), Minneapolis, MN, 55404
Contact Information:
Johnson, Mahrya
Phone: (612) 813-6384
Email: mahrya.johnson@childrensmn.org
|
| MO |
-
Washington University School of Medicine (Adult), St. Louis, MO, 63110
Contact Information:
Burks, Patricia
Phone: (314) 454-5175
Email: Burks_P@kids.wustl.edu
|
| PA |
-
Children's Hospital of Philadelphia University of Pennsylvania (Pediatric), Philadelphia, PA, 19104
Contact Information:
Kubrak, Christina
Phone: (267) 426-5135
Email: kubrak@email.chop.edu
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Children's Hospital of Pittsburgh of UPMC (Pediatric), Pittsburgh, PA, 15213
Contact Information:
Hartigan, Elizabeth
Phone: (412) 692-7060
Email: elizabeth.hartigan@chp.edu
|
| TX |
-
Baylor College of Medicine (Pediatric), Houston, TX, 77030
Contact Information:
Schaap, Nicoline
Phone: (832) 822-4252
Email: nxschaap@texaschildrens.org
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