Status:
COMPLETED
G551D Observational Study- Expanded to Additional Genotypes and Extended for Long Therm Follow up (GOAL-e2)
Lead Sponsor:
Steven M Rowe
Conditions:
Cystic Fibrosis
Eligibility:
All Genders
6+ years
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 us...
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 ...
Eligibility Criteria
Inclusion
- Inclusion Criteria for Core Study:
- Male or female ≥ 6 years of age at Visit 1. :
- Must have a clinical diagnosis of cystic fibrosis and the following CFTR mutations:
- For Cohort 1 (Closed to enrollment June 30, 2012):
- G551D on at least 1 allele Any known or unknown mutations allowed on second allele.
- For Cohort 2:
- R117H on at least 1 allele Any known or unknown mutation on the second allele except G551D
- 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
- 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.)
- Clinically stable with no significant changes in health status within the 14 days prior to Visit 1.
- Written informed consent (and assent when applicable) obtained from subject or subject's legal representative.
- Exclusion Criteria for Core Study
- 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.
- Any upper or lower respiratory symptoms requiring treatment with oral, inhaled or IV antibiotics within the 2 weeks prior to Visit 1.
- History of solid organ transplantation.
- 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.
Exclusion
Key Trial Info
Start Date :
January 1 2014
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
June 1 2021
Estimated Enrollment :
120 Patients enrolled
Trial Details
Trial ID
NCT01521338
Start Date
January 1 2014
End Date
June 1 2021
Last Update
June 30 2021
Active Locations (36)
Enter a location and click search to find clinical trials sorted by distance.
1
University of Alabama at Birmingham
Birmingham, Alabama, United States, 35233
2
Phoenix Childrens Hospital
Phoenix, Arizona, United States
3
University of Arkansas for Medical Sciences
Little Rock, Arkansas, United States
4
Stanford University Medical Center
Palo Alto, California, United States, 94394