- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT01078740
Detection of Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Activity in Rectal Tissues From Human Subjects
Detection of Cystic Fibrosis Transmembrane Conductance Regulator CFTR) Activity in Rectal Tissues From Human Subjects
Study Overview
Status
Conditions
Detailed Description
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.
Study Type
Enrollment (Actual)
Contacts and Locations
Study Locations
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Alabama
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Birmingham, Alabama, United States, 35249
- University of Alabama at Birmingham Hospital
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Iowa
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Iowa City, Iowa, United States, 52242
- University of Iowa Hospitals and Clinics
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North Carolina
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Chapel Hill, North Carolina, United States, 27514
- University of North Carolina Hospitals
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Sampling Method
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.
Description
Non-CF subjects
Inclusion Criteria:
- 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
Exclusion Criteria:
- 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
Inclusion Criteria:
- 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
Exclusion Criteria:
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
Inclusion Criteria:
- 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
- CBC, PT/PTT within acceptable range (see exclusion criteria for values) within 14 days of the procedure
- Negative pregnancy test (if applicable) completed within two (2) days of the procedure
Exclusion Criteria:
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:
- Bleeding diathesis (platelets <50,000, INR >1.5)
- Anemia (hemoglobin <10 gm/dL, or hematocrit <30%
- White blood count >20,000
- Neutropenia (ANC <1,500) or lymphopenia (absolute lymphocyte count <1,500)
- Positive pregnancy test (if applicable)
- Breastfeeding
- Significantly diseased distal rectal/GI tissue that could place the study subject at risk by participating in the study (as judged by the collaborating gastroenterologist, such as significant hemorrhoids, vascular abnormalities, colonic infection, radiation injury or history of radiation therapy to the rectum, prostate and/or pelvic area)
- Use of drugs with significant risks of compromising immunity (oral steroid use > 20 mg/day)
Study Plan
How is the study designed?
Design Details
Cohorts and Interventions
Group / Cohort |
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Non-CF subjects
Rectal tissue obtained from study subjects without cystic fibrosis (CF) as part of scheduled colonoscopy/biopsies performed for clinical care
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CF subjects
Rectal tissue obtained from study subjects with cystic fibrosis (CF) in one of three ways:
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
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).
Time Frame: 24 hours
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24 hours
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Secondary Outcome Measures
Outcome Measure |
Time Frame |
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To estimate the range of detectable CFTR activity among human rectal tissue of subjects with and without cystic fibrosis.
Time Frame: 24 hours
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24 hours
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Collaborators and Investigators
Collaborators
Investigators
- Principal Investigator: JP Clancy, MD, University of Alabama at Birmingham
Study record dates
Study Major Dates
Study Start
Primary Completion (Actual)
Study Completion (Actual)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Estimate)
Study Record Updates
Last Update Posted (Estimate)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
Other Study ID Numbers
- ICM001
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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