An observational study in Cystic Fibrosis, sponsored by Steven M Rowe. Completed at 36 sites in United States. Open to participants aged 6 Years and older. Per ClinicalTrials.gov, last updated 2021-06-30.
Sponsored by Steven M Rowe · Observational
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.
Optional:
1,581 studies on the registry are indexed under Cystic Fibrosis; 190 are open to participants now.
This study's enrollment of 120 is above the median of 85 across 482 observational studies indexed under Cystic Fibrosis.
Browse Cystic Fibrosis studies →This is the only study on the registry with Steven M Rowe as lead sponsor.
Counted across the registry records on this site, refreshed daily.
Patients with Cystic Fibrosis and the G551D, R117H and non-G551D gating Mutations ages 6 years old and older
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
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
Exclusion Criteria for Core Study
Primary Endpoint for the Core Study
Change in FEV1% predicted between Visit 1 and visit 5
Time frame: Change in FEV1% predicted between Visit 1 and Visit 5
Change in sweat chloride between Visit 1 and Visit 5.
Change in sweat chloride between Visit 1 and Visit 5.
Time frame: VISIT 1 AND VISIT 5
Change in body weight between Visit 1 and Visit 5.
Change in body weight between Visit 1 and Visit 5.
Time frame: VISIT 1 AND VISIT 5
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.
Time frame: 1 YEAR
This study is completed, as verified in Jun 2021. You cannot join it, but the record below documents what was studied.
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