Cystic FibrosisNCT01078740
Detection of Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Activity in Rectal Tissues From Human Subjects
An observational study of Cystic Fibrosis, run by CF Therapeutics Development Network Coordinating Center at 3 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 75 yearsHealthy volunteers can take part
- Conditions the study is for
- Cystic Fibrosis
- How many people
- 57 participants
- Where
- 3 sitesUnited States
- Study dates
- Oct 2009to Aug 2010
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
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.
Who may be able to join
- 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
- 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
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- Written informed consent obtained from subject or subject's legal representative and ability for subject to comply with the requirements of the study
- 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
Who cannot join
- 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
- 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)
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- Significant colonic infection (as judged by the collaborating gastroenterologist)
- CF subjects undergoing sigmoidoscopy biopsy procedure for study purposes only
- 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)
Trial locations and contacts
3 sites in 1 country.
United States3 sites
University of Alabama at Birmingham Hospital
Birmingham, Alabama, 35249
University of North Carolina Hospitals
Chapel Hill, North Carolina, 27514
University of Iowa Hospitals and Clinics
Iowa City, Iowa, 52242
About this study
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.
Read the 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.
- Conditions
- Cystic Fibrosis
- Keywords
- cystic fibrosis, cystic fibrosis transmembrane conductance regulator, biomarkers, Intestinal Current Measurement
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| 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 |
What is being measured
The primary outcome is the main question the study is designed to answer.
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).
Measured: 24 hours
Secondary outcomes (1)
To estimate the range of detectable CFTR activity among human rectal tissue of subjects with and without cystic fibrosis.
Measured: 24 hours
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Mar 2010 (estimated)
- Study startOct 2009
- Primary completionJul 2010
- Study completionAug 2010
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 57 participants (actual)
Sponsor and investigators
- Lead sponsor
- CF Therapeutics Development Network Coordinating Center
- Sponsor type
- Research network
- Collaborators
- Cystic Fibrosis Foundation
- Responsible party
- Sponsor
- Investigators
- JP Clancy, MD, Principal investigator, University of Alabama at Birmingham
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01078740
- Sponsor's protocol ID
- ICM001
- Status last verified
- Mar 2013
- First submitted
- 26 Feb 2010
- Last updated
- 7 Mar 2013
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 7 Mar 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Detection of Cystic Fibrosis Transmembrane Conductance Regulator CFTR) Activity in Rectal Tissues From Human Subjects
- ClinicalTrials.gov ID
- NCT01078740
- Acronym / sponsor ID
- ICM001
- Conditions
- Cystic Fibrosis
- Keywords
- cystic fibrosis, cystic fibrosis transmembrane conductance regulator, biomarkers, Intestinal Current Measurement
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 57 participants (actual)
- Lead sponsor
- CF Therapeutics Development Network Coordinating CenterResearch network
- Collaborators
- Cystic Fibrosis Foundation
- Study start
- Oct 2009
- Primary completion
- Jul 2010 (actual)
- Study completion
- Aug 2010 (actual)
- First posted
- 2 Mar 2010 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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 design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| 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 |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
- 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.
Key inclusion criteria (12 listed)
- 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
Key exclusion criteria (17 listed)
- CF subjects undergoing a surgical procedure for clinical care
- 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:
- CF subjects undergoing sigmoidoscopy biopsy procedure for study purposes only
- 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)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
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
1 secondary outcome
To estimate the range of detectable CFTR activity among human rectal tissue of subjects with and without cystic fibrosis.
Time frame: 24 hours
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Mar 2013.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
JP Clancy, MD principal investigator
University of Alabama at Birmingham
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Cystic Fibrosis, run by CF Therapeutics Development Network Coordinating Center at 3 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 12 inclusion and 17 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 7 Mar 2013. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 7 Mar 2013.