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Study Name: GOAL-e2 Expansion Study
Study Type: Observational
Intervention Category: Not Applicable
Study Sponsor: Rowe, Steven

Funding for this trial has been provided in full or in part by Cystic Fibrosis Foundation Therapeutics, Inc.
Study Phase: Other
Recruitment Status: Recruiting
Study Drug(s): Not Applicable
Number of Participants Being Recruited: 90
Single / Multi-Center: Multi-Center
The purpose of this study is to collect samples from people who have either the R117H or other non-G551D gating CFTR mutations to be kept for future research. Samples of blood, sputum, and urine may be collected for this study along with pulmonary function testing.
Age: >= 6 Years
FEV1: >= Percent Predicted
P. aeruginosa status: Not applicable
B. cepacia status: Not applicable
Other Primary Eligibility Requirements:

Must have following CFTR mutations:
For Cohort 2:
a) R117H on at least 1 allele
b) Any known or unknown mutation on the second allele except G551D

For Cohort 3:
a) A Non-G551D gating mutation on one allele: (G178R, S549N, S549R, G551S, G970R, G1244E, S1251N, S1255P, G1349D)
b) Any known or unknown mutation on the second allele except G551D OR R117H

Cannot be participating in the VX-770-105, VX-770-106, VX-770-108, VX-770-110, VX-770-111, VX-770-112, or VX-770-113 study, VX-770 Extended Access Program or use of ivacaftor within 6 months prior to Visit 1.
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: Sagel, Scott
(720) 777-5546
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
  • Stanford University Medical Center (Pediatric), Palo Alto, CA, 94304
    Contact Information:
        Dunn, Colleen
        Phone: (650) 736-0388
  • Children's Hospital Colorado (Pediatric), Aurora, CO, 80045
    Contact Information:
        Pardee, Churee
        Phone: (720) 777-6162
  • National Jewish Health (Adult), Denver, CO, 80206
    Contact Information:
        Jones, Marion
        Phone: (303) 398-1265
  • Yale University School of Medicine (Pediatric), New Haven, CT, 06520
    Contact Information:
        Young, Jane
        Phone: (203) 785-5852
  • University of Chicago (Adult), Chicago, IL, 60637
    Contact Information:
        Holland, Spring
        Phone: (773) 834-4053
  • Indiana University (Adult), Indianapolis, IN, 46202
    Contact Information:
        Bendy, Lisa
        Phone: (317) 948-7152
  • Riley Hospital for Children Indiana University Medical Center (Pediatric), Indianapolis, IN, 46202
    Contact Information:
        Bendy, Lisa
        Phone: (317) 948-7152
  • University of Kentucky (Pediatric), Lexington, KY, 40536
    Contact Information:
        Watts, Tammy
        Phone: (859) 257-5087
  • 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
  • Johns Hopkins University (Pediatric), Baltimore, MD, 21287
    Contact Information:
        Callahan, Karen
        Phone: (443) 287-8983
  • University of Michigan Health System (Pediatric), Ann Arbor, MI, 48109
    Contact Information:
        Kruse, Dawn
        Phone: (734) 615-3266
  • Children's Hospital of Michigan (Pediatric), Detroit, MI, 48201
    Contact Information:
        Van Wagnen, Catherine
        Phone: (313) 745-4737
  • Helen DeVos Women and Children's Center (Pediatric), Grand Rapids, MI, 49503
    Contact Information:
        Gile, Cynthia
        Phone: (616) 391-6148
  • 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
  • University of North Carolina at Chapel Hill (Pediatric), Chapel Hill, NC, 27599
    Contact Information:
        Barlow, Carol
        Phone: (919) 966-9146
  • Dartmouth Hitchcock Medical Center (Pediatric), Lebanon, NH, 03756
    Contact Information:
        Moulton, Lisa
        Phone: (603) 650-8178
        Email: Lisa.A.Moulton@Hitchcock.ORG
  • Women and Children's Hospital of Buffalo (Pediatric), Buffalo, NY, 14222
    Contact Information:
        Caci, Nadine
        Phone: (716) 878-7524
  • Cincinnati Children's Hospital Medical Center (Pediatric), Cincinnati, OH, 45229
    Contact Information:
        Decker, Beth
        Phone: (513) 803-4325
  • University Hospitals Case Medical Center/Rainbow Babies and Children's Hospital (Pediatric), Cleveland, OH, 44106
    Contact Information:
        Ksenich, Roberta (Bobbi)
        Phone: (216) 844-5733
  • Nationwide Children's Hospital (Pediatric), Columbus, OH, 43205
    Contact Information:
        Gilmore, Diana
        Phone: (614) 722-4752
  • Toldeo Children's Hospital (Pediatric), Toledo, OH, 43606
    Contact Information:
        Houser, Kelly
        Phone: (419) 291-4630
  • Oregon Health Sciences University (Pediatric), Portland, OR, 97201
    Contact Information:
        Simmons, Kim
        Phone: (503) 494-7807
  • Children's Hospital of Philadelphia University of Pennsylvania (Pediatric), Philadelphia, PA, 19104
    Contact Information:
        Kubrak, Christina
        Phone: (267) 426-5135
  • Children's Hospital of Pittsburgh of UPMC (Pediatric), Pittsburgh, PA, 15213
    Contact Information:
        Hartigan, Elizabeth
        Phone: (412) 692-7060
  • Medical University of South Carolina (Pediatric), Charleston, SC, 29403
    Contact Information:
        Warden, Ashley
        Phone: (843) 792-4349
  • Vanderbilt Children's Hospital Vanderbilt University Medical Center (Pediatric), Nashville, TN, 37232
    Contact Information:
        Berry, Pamela
        Phone: (615) 936-8308
        Email: pamela.berry@Vanderbilt.Edu
  • University of Texas Southwestern Medical Center/ St. Paul University Hospital (Adult), Dallas, TX, 75390
    Contact Information:
        Keller, Ashley
        Phone: (214) 648-2817
  • Baylor College of Medicine (Pediatric), Houston, TX, 77030
    Contact Information:
        Schaap, Nicoline
        Phone: (832) 822-4252
  • Intermountain Cystic Fibrosis Center (Adult), Salt Lake City, UT, 84132
    Contact Information:
        Packer, Kristyn
        Phone: (801) 585-0401
  • Froedtert & Medical College of Wisconsin (Adult), Milwaukee, WI, 53201
    Contact Information:
        Harris, Rachel
        Phone: (414) 955-7030
  • West Virginia University CF Center (Pediatric and Adult), Morgantown, WV, 26505
    Contact Information:
        Collins, Sue
        Phone: (304) 293-7348
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