- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT00179439
NPDT Evaluation in Children With CFTR and (PSC) (NPD)
Nasal Potential Difference Testing: Evaluation of Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Function in Children With Primary Sclerosing Cholangitis (PSC)
Study Overview
Status
Intervention / Treatment
Detailed Description
The purpose of this protocol is to perform Nasal Transepithelial Potential Difference (NTPD) testing to assess the function of the cystic fibrosis gene product, a chloride channel referred to as CFTR, in patients diagnosed with PSC and/or inflammatory bowel disease in childhood and currently 12 years of age and greater.
Dr. Freedman's laboratory has shown that there is an increased prevalence of CFTR abnormalities in adults with PSC as demonstrated by genotype and phenotype analysis. We hypothesize that abnormalities in CFTR based on exhaustive genotype and phenotype assessments are associated with the presence of PSC in children. We would like to enroll patients with inflammatory bowel disease and no PSC to use as a "control group".
Subjects with PSC and/or inflammatory bowel disease diagnosed in childhood, currently aged 12 years and above, will be enrolled in study protocols at Children's Hospital in Boston, which will have received their local IRB approval. The only role for BIDMC will be to perform NTPD testing on these subjects. No other assessment or testing will be performed at our site. We will not be involved in any other aspect of care for these subjects.
Study Type
Enrollment (Anticipated)
Contacts and Locations
Study Locations
-
-
Massachusetts
-
Boston, Massachusetts, United States, 02215
- Beth Israel Deaconess Medical Center
-
-
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
Inclusion Criteria:
- 12 years of age and older
- Must have diagnosis of primary sclerosing cholangitis and/or inflammatory bowel disease
- Absence of other liver disease, such as viral hepatitis, drug-induced liver disease, and metabolic/hereditary liver disease
- No exclusion based on sex, race, and ethnic background
Study Plan
How is the study designed?
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
---|
CFTR DNA analysis
|
Secondary Outcome Measures
Outcome Measure |
---|
Nasal potential difference testing
|
Sweat test
|
Collaborators and Investigators
Collaborators
Investigators
- Study Director: Harpreet Pall, MD, Boston Children's Hospital
- Principal Investigator: Steven D Freedman, MD, PhD, Beth Israel Deaconess Medical Center
Study record dates
Study Major Dates
Study Start (Actual)
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 (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- 2004P-000316
- 02820-4
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
product manufactured in and exported from the U.S.
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.
Clinical Trials on Primary Sclerosing Cholangitis
-
Medical University of WarsawNational Science Centre, PolandRecruitingPrimary Sclerosing Cholangitis (PSC)Poland
-
Mayo ClinicActive, not recruiting
-
Cascade Pharmaceuticals, IncCovanceCompletedPrimary Sclerosing Cholangitis (PSC)United States
-
HighTide Biopharma Pty LtdCompletedPrimary Sclerosing Cholangitis (PSC)United States, Canada
-
Intercept PharmaceuticalsCompletedPrimary Sclerosing Cholangitis (PSC)United States, Italy
-
Mirum Pharmaceuticals, Inc.CompletedPrimary Sclerosing Cholangitis (PSC)United States, United Kingdom, Canada
-
Mayo ClinicCompletedPrimary Sclerosing Cholangitis (PSC)United States
-
Gilead SciencesCompletedPrimary Sclerosing Cholangitis (PSC)United States, Belgium, Canada, Germany, United Kingdom, Italy, Spain, Denmark, Netherlands, Sweden
-
Brigham and Women's HospitalCompletedSclerosing CholangitisUnited States
-
Boston Scientific CorporationCompletedPrimary Sclerosing Cholangitis (PSC)United States, Canada, Netherlands, Norway
Clinical Trials on nasal potential difference testing
-
Children's Hospital of PhiladelphiaCompleted
-
Massachusetts General HospitalBill and Melinda Gates FoundationEnrolling by invitation
-
University Medicine GreifswaldFriedrich Loefler Institute for Animal Health; EUROIMMUN AGActive, not recruiting
-
Hamilton Health Sciences CorporationFirestone Institute for Respiratory HealthCompletedNasal Lavage Fluid | Eosinophils | Reproducibility of ResultsCanada
-
Marco J. BrunoBoston Scientific CorporationRecruitingCholangiopancreatography, Endoscopic Retrograde | Drug Resistance, Multiple, BacterialUnited States, India, Netherlands, Italy
-
University of PittsburghNational Institute on Deafness and Other Communication Disorders (NIDCD)CompletedMiddle-ear BarotraumaUnited States
-
University of PatrasNot yet recruiting
-
University of PittsburghNational Heart, Lung, and Blood Institute (NHLBI); National Institutes of Health...CompletedChronic Obstructive Pulmonary Disease | Emphysema | HIVUnited States
-
Dartmouth-Hitchcock Medical CenterRecruitingCystic FibrosisUnited States
-
Connecticut Children's Medical CenterUConn HealthRecruiting