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|
| Study Name: |
VX-770 Expanded Access Program |
| Study Type: |
Interventional |
| Intervention Category: |
CFTR Modulation |
| Study Sponsor: |
Vertex |
| Study Phase: |
Other |
| Recruitment Status: |
Recruiting |
| 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. |
| 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. |
| Sponsor Contact Information: |
Call Center, Expanded Access
(800) 745-4484
|
| 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:
Richardson, Stacey
Phone: (317) 948-7121
Email: stlyrich@iupui.edu
|
| MA |
-
Children's Hospital Boston (Pediatric), Boston, MA, 02115
Contact Information:
Leone Thakkallapalli, Erin
Phone: (617) 355-3197
Email: erin.leone@childrens.harvard.edu
|
| MI |
-
Wayne State University Harper University Hospital (Adult), Detroit, MI, 48201
Contact Information:
Cahill, James
Phone: (313) 745-2221
Email: jcahil@med.wayne.edu
|
| NY |
-
Children's Hospital of Buffalo (Pediatric), Buffalo, NY, 14222
Contact Information:
Caci, Nadine
Phone: (716) 878-7524
Email: ncaci@upa.chob.edu
-
Long Island Jewish Medical Center Adult Program (Adult), New Hyde Park, NY, 11040
Contact Information:
Wann, Li-Chen
Phone: (516) 712-9608
Email: LCWann@nshs.edu
-
Beth Israel Medical Center (Pediatric), New York, NY, 10003
Contact Information:
Grece, Carroll Anne
Phone: (212) 420-4196
Email: cgrece@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
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The Children's Medical Center of Dayton (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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