Detection of Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Activity in Rectal Tissues From Human Subjects

NCT01078740 | Completed DMC

• 1 other identifier: ICM001
• Study Type: observational
• Target: 57
• Locations: 1 country
• Sites: 3

Brief Summary

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.

Conditions

Cystic Fibrosis

Keywords

cystic fibrosis; cystic fibrosis transmembrane conductance regulator; biomarkers; Intestinal Current Measurement

Outcome Measures

Primary Outcomes (1)

• To develop and improve techniques to detect mature CFTR protein at the rectal cell membrane by performing rectal biopsies and ex-vivo Intestinal Current Measurement (ICM).

  24 hours

Secondary Outcomes (1)

• To estimate the range of detectable CFTR activity among human rectal tissue of subjects with and without cystic fibrosis.

  24 hours

Study Arms (2)

Non-CF subjects

Rectal tissue obtained from study subjects without cystic fibrosis (CF) as part of scheduled colonoscopy/biopsies performed for clinical care

CF subjects

Rectal tissue obtained from study subjects with cystic fibrosis (CF) in one of three ways: * Rectal biopsy as part of scheduled colonoscopy/biopsies performed for clinical care * Sigmoidoscopy/biopsy added onto a scheduled, unrelated procedure or surgery performed under general anesthesia * Sigmoidoscopy/biopsy performed for the sole purpose of obtaining rectal tissue for the current study

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Non-CF subjects: Adults undergoing endoscopic procedures (colonoscopies) who meet the eligibility criteria. CF subjects: Adults with a diagnosis of CF who meet the eligibility criteria. CF subjects can include both patients who will be undergoing endoscopic (colonoscopies) and nonendoscopic procedures requiring anesthesia or sedation as a part of clinical care (e.g., sinus surgery, central line placement), or CF subjects not undergoing planned surgical/anesthetic procedures.

You may qualify if:

• Male or female 18-75 years of age at enrollment
• Undergoing colonoscopy for clinical care
• Written informed consent obtained from subject or subject's legal representative and ability for subject to comply with the requirements of the study

You may not qualify if:

• Any medical condition or laboratory values that in the opinion of the onsite principal investigator and/or collaborating gastroenterologist may place the subject at significant risk by undergoing the additional research related biopsies
• Subjects with a history of radiation therapy to the rectum, prostate and/or pelvic area
• CF subjects undergoing a surgical procedure for clinical care
• Confirmed diagnosis of CF based on the following criteria: ∆F508 homozygous
• Male or female 18 years of age or greater at enrollment
• Patient undergoing planned colonoscopy or other surgical procedure and agrees to undergo sigmoidoscopy and biopsy
• Written informed consent obtained from subject or subject's legal representative and ability for subject to comply with the requirements of the study
• Any medical condition or laboratory values that in the opinion of the onsite principal investigator and/or collaborating gastroenterologist may place the subject at significant risk by undergoing the additional research related biopsies, including:
• Significantly diseased distal rectal/gastrointestinal (GI) tissue (as judged by the collaborating gastroenterologist including radiation injury or history of radiation therapy to the rectum, prostate and/or pelvic area
• Significant hemorrhoids or vascular abnormalities (as judged by the collaborating gastroenterologist)
• Significant colonic infection (as judged by the collaborating gastroenterologist)
• CF subjects undergoing sigmoidoscopy biopsy procedure for study purposes only
• Confirmed diagnosis of CF based on the following criteria: ∆F508 homozygous
• Male or female 18 years of age or greater at enrollment
• Written informed consent obtained from subject or subject's legal representative and ability for subject to comply with the requirements of the study

Sponsors & Collaborators

• CF Therapeutics Development Network Coordinating Center: lead
• Cystic Fibrosis Foundation: collaborator

Study Sites (3)

University of Alabama at Birmingham Hospital

Birmingham, Alabama, 35249, United States

Location

University of Iowa Hospitals and Clinics

Iowa City, Iowa, 52242, United States

Location

University of North Carolina Hospitals

Chapel Hill, North Carolina, 27514, United States

Location

Biospecimen

Retention: SAMPLES WITH DNA

Rectal biopsy

MeSH Terms

Conditions

Cystic Fibrosis

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases

Study Officials

• JP Clancy, MD

  University of Alabama at Birmingham

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: NETWORK
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 26, 2010
• First Posted: March 2, 2010
• Study Start: October 1, 2009
• Primary Completion: July 1, 2010
• Study Completion: August 1, 2010
• Last Updated: March 7, 2013

Record last verified: 2013-03

Locations

US (3)