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

Completed
Conditions
Cystic Fibrosis
Registration Number
NCT01521338
Lead Sponsor
Steven M Rowe
Brief Summary

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.

Detailed Description

* 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

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
120
Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

Study Type
OBSERVATIONAL
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
Primary Endpoint for the Core StudyChange in FEV1% predicted between Visit 1 and Visit 5

Change in FEV1% predicted between Visit 1 and visit 5

Secondary Outcome Measures
NameTimeMethod
Change in sweat chloride between Visit 1 and Visit 5.VISIT 1 AND VISIT 5

Change in sweat chloride between Visit 1 and Visit 5.

Change in body weight between Visit 1 and Visit 5.VISIT 1 AND VISIT 5

Change in body weight between Visit 1 and Visit 5.

Trial Locations

Locations (36)

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

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University of Alabama at Birmingham
🇺🇸Birmingham, Alabama, United States

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