Cystic Fibrosis Clinical Trial
Official title:
Detection of Cystic Fibrosis Transmembrane Conductance Regulator CFTR) Activity in Rectal Tissues From Human Subjects
This is a pilot study. The purpose of the study is to facilitate the development of a new biomarker of cystic fibrosis transmembrane conductance regulator (CFTR) function using rectal tissue.
CF research has advanced to the point where small molecule agents have been developed to
overcome the underlying genetic defects caused by CFTR mutations.
There is a critical need to develop new sensitive biomarkers of CFTR function and
biochemistry that can be used in early phase clinical trials to demonstrate biologic effects
of investigative agents in vivo. Intestinal Current Measurement (ICM) from rectal biopsy
samples is an assay that has been proven to be sensitive and specific for CFTR function.
This method and site of investigation is particularly attractive, since CFTR is expressed at
high levels in the rectum, it is not altered by disease manifestations, and the tissue can
be studied ex vivo, providing more flexibility in the nature of the techniques to detect and
quantify CFTR activity.
This study will aid in the development of new biomarkers in human rectal tissue for use in
CF clinical trials. Testing compounds that are designed to restore function to
disease-causing CFTR genes and proteins will provide an opportunity to improve and
standardize techniques in the acquisition and measurement of CFTR activity in rectal biopsy
specimens.
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Observational Model: Cohort, Time Perspective: Prospective
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