G551D Observational Study- Expanded to Additional Genotypes and Extended for Long Therm Follow up (GOAL-e2)
NCT ID: NCT01521338
Last Updated: 2021-06-30
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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COMPLETED
120 participants
OBSERVATIONAL
2014-01-31
2021-06-30
Brief Summary
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Detailed Description
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* 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
Conditions
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Study Design
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COHORT
PROSPECTIVE
Eligibility Criteria
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Inclusion Criteria
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
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.
6 Years
ALL
No
Sponsors
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Steven M Rowe
OTHER
Responsible Party
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Steven M Rowe
Principal Investigator
Principal Investigators
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Steven M Rowe, MD
Role: PRINCIPAL_INVESTIGATOR
University of Alabama at Birmingham
Locations
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University of Alabama at Birmingham
Birmingham, Alabama, United States
Phoenix Childrens Hospital
Phoenix, Arizona, United States
University of Arkansas for Medical Sciences
Little Rock, Arkansas, United States
Stanford University Medical Center
Palo Alto, California, United States
The Children's Hospital Colarado
Aurora, Colorado, United States
National Jewish Health
Denver, Colorado, United States
Yale
New Haven, Connecticut, United States
University of Chicago
Chicago, Illinois, United States
Riley Hospital for Children Indiana University Medical Center
Indianapolis, Indiana, United States
Indianapolis University
Indianapolis, Indiana, United States
University of Kentucky
Lexington, Kentucky, United States
The Johns Hopkins University
Baltimore, Maryland, United States
Massachusetts General Hospital
Boston, Massachusetts, United States
Children's Hospital Boston
Boston, Massachusetts, United States
University of Michigan
Ann Arbor, Michigan, United States
Children's Hospital of Michigan
Detroit, Michigan, United States
Grand Rapids CF Center
Grand Rapids, Michigan, United States
University of Minnesota
Minneapolis, Minnesota, United States
The Children's Mercy Hospital--University of Missouri at Kansas City
Kansas City, Missouri, United States
Dartmouth Hitchcock Clinic-Lebanon
Lebanon, New Hampshire, United States
Women and Children's Hospital of Buffalo
Buffalo, New York, United States
University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States
University Hospitals Case Medical Center/Rainbow Babies and Children's Hospital
Cleveland, Ohio, United States
Nation Wide Childrens Hospital
Columbus, Ohio, United States
Toledo Children's Hospital
Toledo, Ohio, United States
Oregon Health Sciences University
Portland, Oregon, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
Children's Hospital of Pittsburgh of UPMC- University of Pittsburgh
Pittsburgh, Pennsylvania, United States
Medical University of South Carolina
Charleston, South Carolina, United States
Vanderbilt Children's Hospital Vanderbilt University Medical Center
Nashville, Tennessee, United States
University of Texas Southwestern/Children's Medical Center of Dallas
Dallas, Texas, United States
Baylor College of Medicine/Texas Children's Hospital
Houston, Texas, United States
Intermountain Cystic Fibrosis Center University of Utah Health Sciences Center
Salt Lake City, Utah, United States
West Virginia University CF Medical Center
Morgantown, West Virginia, United States
Froedtert & Medical College of Wisconsin
Milwaukee, Wisconsin, United States
Countries
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References
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Donaldson SH, Laube BL, Corcoran TE, Bhambhvani P, Zeman K, Ceppe A, Zeitlin PL, Mogayzel PJ Jr, Boyle M, Locke LW, Myerburg MM, Pilewski JM, Flanagan B, Rowe SM, Bennett WD. Effect of ivacaftor on mucociliary clearance and clinical outcomes in cystic fibrosis patients with G551D-CFTR. JCI Insight. 2018 Dec 20;3(24):e122695. doi: 10.1172/jci.insight.122695.
van de Peppel IP, Doktorova M, Berkers G, de Jonge HR, Houwen RHJ, Verkade HJ, Jonker JW, Bodewes FAJA. IVACAFTOR restores FGF19 regulated bile acid homeostasis in cystic fibrosis patients with an S1251N or a G551D gating mutation. J Cyst Fibros. 2019 Mar;18(2):286-293. doi: 10.1016/j.jcf.2018.09.001. Epub 2018 Sep 29.
Other Identifiers
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F11120200
Identifier Type: -
Identifier Source: org_study_id
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