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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
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.
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 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: Call Center, Expanded Access
(800) 745-4484
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.
  • Providence Alaska Medical Center (Pediatric and Adult), Anchorage, AK, 99508
    Contact Information:
        Roberts, Vicki
        Phone: (907) 212-3299
  • The Children's Hospital/University of Alabama at Birmingham (Pediatric), Birmingham, AL, 35233
    Contact Information:
        Hathorne, Heather
        Phone: (205) 638-9568
  • Miller Children's Hospital (Pediatric), Long Beach, CA, 90801
    Contact Information:
        O'Donnell, Nan
        Phone: (562) 933-5607
  • Ann and Robert H. Lurie Children's Hospital of Chicago (Pediatric), Chicago, IL, 60611
    Contact Information:
        Singh, Noopur
        Phone: (312) 227-6861
  • Indiana University (Adult), 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
  • Wayne State University Harper University Hospital (Adult), Detroit, MI, 48201
    Contact Information:
        Driscoll, Debra
        Phone: (313) 745-2221
  • Women and Children's Hospital of Buffalo (Pediatric), Buffalo, NY, 14222
    Contact Information:
        Caci, Nadine
        Phone: (716) 878-7524
  • Beth Israel Medical Center (Pediatric), New York, NY, 10003
    Contact Information:
        Grece, Carroll Anne
        Phone: (212) 420-4196
  • Beth Israel Medical Center (Pediatric), New York, NY, 10003
    Contact Information:
        DiBenedetto, Donna
        Phone: (212) 420-4100
  • University Hospitals Case Medical Center/Rainbow Babies and Children's Hospital (Pediatric), Cleveland, OH, 44106
    Contact Information:
        Ksenich, Roberta (Bobbi)
        Phone: (216) 844-5733
  • Dayton Children's Hospital (Pediatric), Dayton, OH, 45404
    Contact Information:
        Bartosik, Sandy
        Phone: (937) 641-4004
  • Hershey Medical Center Pennsylvania State University (Pediatric), Hershey, PA, 17033
    Contact Information:
        Kitch, Diane
        Phone: (717) 531-5646
  • Children's Hospital of Philadelphia University of Pennsylvania (Pediatric), Philadelphia, PA, 19104
    Contact Information:
        Kubrak, Christina
        Phone: (267) 426-5135
  • Froedtert & Medical College of Wisconsin (Adult), Milwaukee, WI, 53201
    Contact Information:
        Harris, Rachel
        Phone: (414) 955-7030
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