G551D Observational Study- Expanded to Additional Genotypes and Extended for Long Therm Follow up (GOAL-e2)



Status:Active, not recruiting
Conditions:Pulmonary
Therapuetic Areas:Pulmonary / Respiratory Diseases
Healthy:No
Age Range:6 - Any
Updated:5/3/2018
Start Date:January 2014
End Date:January 2019

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G551D Observational Study (GOAL)-Expanded to Additional Genotypes and Extended for Long Term Follow up (GOAL-e2)

The goal of this research study is to collect blood and urine samples from people who have
either the R117H type of CF or the non-G551D gating type of CF to be kept for future
research.We will also use some of the collected blood to measure the number of neutrophils.

- Blood Collection: Blood will be collected to be kept for research in the future and to
measure the number of blood cells called neutrophils. A small needle will be used to
collect blood from a vein in your arm. About 7-9 teaspoons of blood would be collected.

- Sweat Collection: Your sweat will be collected with a special sweat collection machine
to test the amount of salt in your sweat.

- Spirometry: You will be asked to take a test that measures how well your lungs are
working. You will be asked to take a deep breath and then blow into a mouthpiece as hard
as possible and for as long as possible. This is the same test that is done when you
come to clinic.

- Urine Collection: Urine will also be collected to be kept for research in the future.
You will be asked to pee in a cup.

- Medical Information: We are asking you to share your medical information with study
researchers. Your medical information will not contain any of your personal
identification information, like your name and address.

Optional:

• Induced Sputum Collection: If you say "yes" to collecting a sputum sample, you will be
asked to breathe in a salt water solution to help you cough out sputum. If you cannot breathe
in the salt water solution to cough out sputum for the last study visit, you will be asked to
cough mucus into a cup

Inclusion Criteria for Core Study:

1. Male or female ≥ 6 years of age at Visit 1. :

2. Must have a clinical diagnosis of cystic fibrosis and the following CFTR mutations:

1. For Cohort 1 (Closed to enrollment June 30, 2012):

G551D on at least 1 allele Any known or unknown mutations allowed on second
allele.

2. For Cohort 2:

R117H on at least 1 allele Any known or unknown mutation on the second allele
except G551D

3. For Cohort 3:

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

3. Enrolled in the Cystic Fibrosis Foundation Patient Registry (with the exception of
Canadian sites). (Patients may enroll in the Registry at Visit 1 if not previously
enrolled.)

4. Clinically stable with no significant changes in health status within the 14 days
prior to Visit 1.

5. Written informed consent (and assent when applicable) obtained from subject or
subject's legal representative.

Exclusion Criteria for Core Study

1. Participation 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.

2. Any upper or lower respiratory symptoms requiring treatment with oral, inhaled or IV
antibiotics within the 2 weeks prior to Visit 1.

3. History of solid organ transplantation.

4. Presence of a condition or abnormality that in the opinion of the investigator would
compromise the safety of the patient or the quality of the data.
We found this trial at
36
sites
500 S State St
Ann Arbor, Michigan 48109
(734) 764-1817
Principal Investigator: Samya Nasr, MD
Phone: 734-615-3266
University of Michigan The University of Michigan was founded in 1817 as one of the...
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3400 N Charles St
Baltimore, Maryland 21205
410-516-8000
Principal Investigator: Pamela Zeitlin, MD
Phone: 410-955-9782
Johns Hopkins University The Johns Hopkins University opened in 1876, with the inauguration of its...
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1720 2nd Ave S
Birmingham, Alabama 35233
(205) 934-4011 
Principal Investigator: Steven M Rowe, MD
Phone: 205-639-5970
University of Alabama at Birmingham The University of Alabama at Birmingham (UAB) traces its roots...
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185 Cambridge Street
Boston, Massachusetts 02114
617-724-5200
Principal Investigator: Leonard Sicilian, MD
Phone: 617-726-3719
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Boston, MA
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Chapel Hill, North Carolina 27599
(919) 962-2211
Principal Investigator: Scott Donaldson, MD
Phone: 919-966-9198
University of North Carolina at Chapel Hill Carolina’s vibrant people and programs attest to the...
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5801 South Ellis Avenue
Chicago, Illinois 60637
 773.702.1234
Principal Investigator: Edward Naureckas, MD
Phone: 773-834-4053
University of Chicago One of the world's premier academic and research institutions, the University of...
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3333 Burnet Avenue # Mlc3008
Cincinnati, Ohio 45229
 1-513-636-4200 
Principal Investigator: John P Clancy, MD
Phone: 513-803-7429
Cincinnati Children's Hospital Medical Center Patients and families from across the region and around the...
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529 West Markham Street
Little Rock, Arkansas 72205
(501) 686-7000
Principal Investigator: Paula Anderson, MD
Phone: 501-416-7019
University of Arkansas for Medical Sciences The University of Arkansas for Medical Sciences (UAMS) in...
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Minneapolis, Minnesota 55455
(612) 625-5000
Principal Investigator: Joanne Billings, MD
Phone: 612-625-7995
Univ of Minnesota With a flagship campus in the heart of the Twin Cities, and...
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South 34th Street
Philadelphia, Pennsylvania 19104
 215-590-1000
Principal Investigator: Ronald Rubenstein, MD
Phone: 267-426-9626
Children's Hospital of Philadelphia Since its start in 1855 as the nation's first hospital devoted...
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Aurora, Colorado 80045
Principal Investigator: Scott Sagel, MD
Phone: 720-777-6162
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Boston, Massachusetts 02115
Principal Investigator: Ahmet Uluer, MD
Phone: 617-355-1834
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Buffalo, New York 14222
Principal Investigator: Carla Frederick, MD
Phone: 716-878-1069
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171 Ashley Avenue
Charleston, South Carolina 29425
843-792-1414
Principal Investigator: Isabel Virella-Lowell, MD
Phone: 843-792-5168
Medical University of South Carolina The Medical University of South Carolina (MUSC) has grown from...
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Cleveland, Ohio 44115
Principal Investigator: Michael Konstan, MD
Phone: 216-844-5733
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Columbus, Ohio 43205
Principal Investigator: Karen McCoy, MD
Phone: 614-722-2607
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Dallas, Texas 75390
Principal Investigator: Raksha Jain, MD
Phone: 214-648-2817
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1400 Jackson St
Denver, Colorado 80206
(303) 388-4461
Principal Investigator: Jennifer Taylor-Cousar, MD
Phone: 303-270-2517
National Jewish Health National Jewish Health is known worldwide for treatment of patients with respiratory,...
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3901 Beaubien St
Detroit, Michigan 48201
(313) 745-5437
Principal Investigator: Ibrahim Abdulhamid, MD
Phone: 313-745-4737
Children's Hospital of Michigan Since 1886, the Children's Hospital of Michigan has been dedicated to...
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Grand Rapids, Michigan 49503
Principal Investigator: Susan Millard, MD
Phone: 616-391-6148
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Houston, Texas
Principal Investigator: Peter Hiatt, MD
Phone: 832-822-4772
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Indianapolis, Indiana 46202
Principal Investigator: Gregory Montgomery, MD
Phone: 317-948-7121
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Indianapolis, Indiana
Principal Investigator: Cynthia Brown, MD
Phone: 317-274-1441
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Kansas City, Missouri 64108
Principal Investigator: Phillip Black, MD
Phone: 816-701-5239
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Lebanon, New Hampshire
Principal Investigator: Alix Ashare, MD
Phone: 603-650-8178
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Lexington, Kentucky
859) 257-9000
Principal Investigator: Michael Anstead, MD
Phone: 859-257-9357
University of Kentucky The University of Kentucky is a public, land grant university dedicated to...
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9200 W Wisconsin Ave
Milwaukee, Wisconsin 53226
(414) 805-3666
Principal Investigator: Julie Biller, MD
Phone: 414-955-7030
Froedtert and the Medical College of Wisconsin Froedtert Health combines with the Medical College of...
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Morgantown, West Virginia 26506
Principal Investigator: Kathryn Moffett, MD
Phone: 304-293-2081
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Nashville, Tennessee 37232
Principal Investigator: Rebekah Brown, MD
Phone: 615-936-1826
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New Haven, Connecticut 06520
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Palo Alto, California 94304
Principal Investigator: Carlos Milla, MD
Phone: 650-723-4670
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1919 E Thomas Rd
Phoenix, Arizona 85006
(602) 933-1000
Principal Investigator: Gerald Gong, MD
Phone: 602-933-0343
Phoenix Children's Hospital Phoenix Children's Hospital has provided hope, healing, and the best healthcare for...
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Pittsburgh, Pennsylvania 15213
Principal Investigator: Joseph Pilewski, MD
Phone: 412-692-7060
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Portland, Oregon 97227
Principal Investigator: Michael Powers, MD
Phone: 503-494-7807
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Salt Lake City, Utah 84132
Principal Investigator: Theodore Liou, MD
Phone: 801-585-0401
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Toledo, Ohio 43606
Principal Investigator: Bruce Barnett, MD
Phone: 419-291-4630
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