Ignite Creation Date:
2024-05-05 @ 5:26 PM
Last Modification Date:
2024-10-26 @ 9:31 AM
Study NCT ID:
NCT00450918
Status:
COMPLETED
Last Update Posted:
2019-10-07
First Post:
2007-03-20
Brief Title:
Evaluating Progression of and Diagnostic Tools for Primary Ciliary Dyskinesia in Children and Adolescents
Sponsor:
University of North Carolina Chapel Hill
Organization:
University of North Carolina Chapel Hill
Study Overview
Official Title:
Longitudinal Study of Primary Ciliary Dyskinesia Participants 5-18 Years of Age
Status:
COMPLETED
Status Verified Date:
2019-10
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
Mucociliary clearance in which mucus secretions are cleared from the breathing airways is the primary defense mechanism for the lungs Inhaled particles including microbes that can cause infections are normally entrapped in mucus on the airway surfaces and then cleared out by the coordinated action of tiny hair-like structures called cilia Individuals with primary ciliary dyskinesia PCD have defective mucociliary clearance which in turn leads to lung infections and disease The purpose of this study is to determine how lung disease progresses over time in children and adolescents with PCD
Detailed Description:
PCD is a rare genetic disorder in which impaired mucus clearance commonly results in chronic cough and infections in the airways sinuses and middle ears Long lasting airway infection ultimately leads to structural damage to the airways known as bronchiectasis and in turn loss of lung function While PCD shares some similarities with the disease cystic fibrosis it is important to distinguish PCD from cystic fibrosis In particular the age of onset and progression of PCDs clinical lung disease including timing of specific microbial pathogen infections and bronchiectasis remain poorly defined The purpose of this study is to determine how lung disease progresses over time in children and adolescents with PCD Specific attention will be directed toward determining whether certain factors play a role in lung disease progression The study will also evaluate diagnostic tools and quality of life among individuals with PCD Filling these gaps of knowledge may help to improve the clinical management of PCD in the future
This longitudinal study will last 5 years There will be a total of 5 study visits and these visits will occur yearly Each study visit will last 3 to 4 hours All study visits will include a medical history review physical exam height weight and vital sign measurements sampling of respiratory fluids and mucus lung function tests and questionnaires The initial visit may also include using a probe to measure nasal nitric oxide levels and blood collection for genetic testing Study visits 1 3 and 5 will also include blood collection for pregnancy testing and a high resolution computed tomography HRCT scan of the chest to image the lungs At the end of each month participants will report any use of oral inhaled or intravenous antibiotics
This longitudinal study will last 5 years There will be a total of 5 study visits and these visits will occur yearly Each study visit will last 3 to 4 hours All study visits will include a medical history review physical exam height weight and vital sign measurements sampling of respiratory fluids and mucus lung function tests and questionnaires The initial visit may also include using a probe to measure nasal nitric oxide levels and blood collection for genetic testing Study visits 1 3 and 5 will also include blood collection for pregnancy testing and a high resolution computed tomography HRCT scan of the chest to image the lungs At the end of each month participants will report any use of oral inhaled or intravenous antibiotics
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| RDCRN 5901 | OTHER | UNC | httpsreporternihgovquickSearchU54HL096458 |
| U54HL096458 | NIH | None | None |