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Study Name: VX-770 (901) Expanded Access Program
Study Type: Interventional
Intervention Category: CFTR Modulation
Study Sponsor: Vertex
Study Phase: Other
Recruitment Status: Trial Completed
Study Drug(s): Kalydeco
Number of Participants Being Recruited: 50
Single / Multi-Center: Multi-Center
STUDY BACKGROUND INFORMATION:
VX-770 is a small molecule compound being developed by Vertex Pharmaceuticals Incorporated (Vertex) for the treatment of CF that targets the underlying defect in CF. In Phase 3 studies of VX-770 in patients with CF and a G551D CFTR mutation, improvements in CFTR function (measured by reduction in sweat chloride concentration) and improvements in lung function were observed. VX-770 is available through an expanded access program to people with CF who have at least one copy of the G551D-CFTR mutation, are in critical medical need, and who are not eligible for participation in other Vertex-sponsored studies.
ELIGIBILITY
Age: >= 6 Years
FEV1: 1 - 40 Percent Predicted
P. aeruginosa status: Not applicable
B. cepacia status: Not applicable
Other Primary Eligibility Requirements:

Have the G551D-CFTR mutation in at least one allele.
Clarification of FEV1 requirement: Highest FEV1 in the 6 months prior to screening is <=40% of predicted value or patient is documented to be active on the lung transplant wait list.
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.
FOR MORE INFORMATION:
Sponsor Contact Information: Call Center, Expanded Access
(800) 745-4484
Trial Specific Link on ClinicalTrials.gov: http://www.clinicaltrials.gov/ct2/show/NCT01381289?term=vertex+and+cystic+fibrosis&rank=1
Clinical Research Terms Glossary: Click here
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.
AK
  • Providence Alaska Medical Center (Pediatric and Adult), Anchorage, AK, 99508
    Contact Information:
        Roberts, Vicki
        Phone: (907) 212-3299
        Email: vicki.roberts@providence.org
AL
  • The Children's Hospital/University of Alabama at Birmingham (Pediatric), Birmingham, AL, 35233
    Contact Information:
        Hathorne, Heather
        Phone: (205) 638-9568
        Email: hhathorne@peds.uab.edu
CA
  • Miller Children's Hospital (Pediatric), Long Beach, CA, 90801
    Contact Information:
        O'Donnell, Nan
        Phone: (562) 933-5607
        Email: nodonnell@memorialcare.org
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
IN
  • Indiana University (Adult), Indianapolis, IN, 46202
    Contact Information:
        Puntenney, Paula
        Phone: (317) 274-1441
        Email: ppuntenn@iu.edu
MA
  • Children's Hospital Boston (Pediatric), Boston, MA, 02115
    Contact Information:
        Fowler, Robert
        Phone: (617) 355-1834
        Email: Robert.fowler@childrens.harvard.edu
MI
  • Wayne State University Harper University Hospital (Adult), Detroit, MI, 48201
    Contact Information:
        Van Wagnen, Catherine
        Phone: (313) 745-4737
        Email: cvanwag@med.wayne.edu
NY
  • Women and Children's Hospital of Buffalo (Pediatric), Buffalo, NY, 14222
    Contact Information:
        Caci, Nadine
        Phone: (716) 878-7524
        Email: ncaci@upa.chob.edu
  • Beth Israel Medical Center (Pediatric), New York, NY, 10003
    Contact Information:
        Grece, Carroll Anne
        Phone: (212) 420-4196
        Email: cgrece@chpnet.org
  • Beth Israel Medical Center (Pediatric), New York, NY, 10003
    Contact Information:
        DiBenedetto, Donna
        Phone: (212) 420-4100
        Email: ddibenedetto@chpnet.org
OH
  • University Hospitals Case Medical Center/Rainbow Babies and Children's Hospital (Pediatric), Cleveland, OH, 44106
    Contact Information:
        Ksenich, Roberta (Bobbi)
        Phone: (216) 844-5733
        Email: Roberta.Ksenich@UHHospitals.org
  • Dayton Children's Hospital (Pediatric), Dayton, OH, 45404
    Contact Information:
        Bartosik, Sandy
        Phone: (937) 641-4004
        Email: bartosiks@childrensdayton.org
PA
  • Hershey Medical Center Pennsylvania State University (Pediatric), Hershey, PA, 17033
    Contact Information:
        Kitch, Diane
        Phone: (717) 531-5646
        Email: dkitch@psu.edu
  • 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
WI
  • Froedtert & Medical College of Wisconsin (Adult), Milwaukee, WI, 53201
    Contact Information:
        Harris, Rachel
        Phone: (414) 955-7030
        Email: racharris@mcw.edu
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