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

Cystic Fibrosis Clinical Trial

— GOAL- e2  

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01521338
• Other study ID #: F11120200
• Status: Completed
• Start date: January 2014
• Est. completion date: June 2021

Study information

• Verified date: June 2021
• Source: University of Alabama at Birmingham
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

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 information / eligibility

• Status: Completed
• Enrollment: 120
• Est. completion date: June 2021
• Est. primary completion date: June 2021
• Accepts healthy volunteers: No
• Gender: All
• Age group: 6 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Cystic Fibrosis
• Fibrosis

Locations

Country	Name	City	State
United States	University of Michigan	Ann Arbor	Michigan
United States	The Children's Hospital Colarado	Aurora	Colorado
United States	The Johns Hopkins University	Baltimore	Maryland
United States	University of Alabama at Birmingham	Birmingham	Alabama
United States	Children's Hospital Boston	Boston	Massachusetts
United States	Massachusetts General Hospital	Boston	Massachusetts
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	Medical University of South Carolina	Charleston	South Carolina
United States	University of Chicago	Chicago	Illinois
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	University of Texas Southwestern/Children's Medical Center of Dallas	Dallas	Texas
United States	National Jewish Health	Denver	Colorado
United States	Children's Hospital of Michigan	Detroit	Michigan
United States	Grand Rapids CF Center	Grand Rapids	Michigan
United States	Baylor College of Medicine/Texas Children's Hospital	Houston	Texas
United States	Indianapolis University	Indianapolis	Indiana
United States	Riley Hospital for Children Indiana University Medical Center	Indianapolis	Indiana
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	University of Kentucky	Lexington	Kentucky
United States	University of Arkansas for Medical Sciences	Little Rock	Arkansas
United States	Froedtert & Medical College of Wisconsin	Milwaukee	Wisconsin
United States	University of Minnesota	Minneapolis	Minnesota
United States	West Virginia University CF Medical Center	Morgantown	West Virginia
United States	Vanderbilt Children's Hospital Vanderbilt University Medical Center	Nashville	Tennessee
United States	Yale	New Haven	Connecticut
United States	Stanford University Medical Center	Palo Alto	California
United States	Children's Hospital of Philadelphia	Philadelphia	Pennsylvania
United States	Phoenix Childrens Hospital	Phoenix	Arizona
United States	Children's Hospital of Pittsburgh of UPMC- University of Pittsburgh	Pittsburgh	Pennsylvania
United States	Oregon Health Sciences University	Portland	Oregon
United States	Intermountain Cystic Fibrosis Center University of Utah Health Sciences Center	Salt Lake City	Utah
United States	Toledo Children's Hospital	Toledo	Ohio

Sponsors (1)

Lead Sponsor	Collaborator
Steven M Rowe

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	ASSOCIATION BETWEEN PRIMARY AND SECONDARY ENDPOINTS	The core and sub-studies will be linked so that associations between primary and secondary endpoints from each sub-study and clinical parameters (e.g., spirometry, weight, and sweat chloride) collected as part of the Core Study may be explored.	1 YEAR
Primary	Primary Endpoint for the Core Study	Change in FEV1% predicted between Visit 1 and visit 5	Change in FEV1% predicted between Visit 1 and Visit 5
Secondary	Change in sweat chloride between Visit 1 and Visit 5.	Change in sweat chloride between Visit 1 and Visit 5.	VISIT 1 AND VISIT 5
Secondary	Change in body weight between Visit 1 and Visit 5.	Change in body weight between Visit 1 and Visit 5.	VISIT 1 AND VISIT 5