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Sponsored by: |
Office of Rare Diseases (ORD) |
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Information provided by: | Office of Rare Diseases (ORD) |
ClinicalTrials.gov Identifier: | NCT00450918 |
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.
Condition |
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Primary Ciliary Dyskinesia |
Study Type: | Observational |
Study Design: | Cohort, Prospective |
Official Title: | Longitudinal Study of Primary Ciliary Dyskinesia: Participants 5-18 Years of Age |
Blood samples
Estimated Enrollment: | 150 |
Study Start Date: | August 2006 |
Estimated Study Completion Date: | August 2014 |
Estimated Primary Completion Date: | August 2014 (Final data collection date for primary outcome measure) |
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 PCD's 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.
Ages Eligible for Study: | 5 Years to 18 Years |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
Sampling Method: | Non-Probability Sample |
Children and adolescents with primary ciliary dyskinesia
Inclusion Criteria:
Exclusion Criteria:
United States, California | |
Stanford University | Recruiting |
Palo Alto, California, United States, 94304 | |
Contact: Carlos Milla, MD cmilla@stanford.edu | |
Principal Investigator: Carlos Milla, MD | |
United States, Colorado | |
The Children's Hospital | Recruiting |
Denver, Colorado, United States, 80218 | |
Contact: Shelley Mann, RN, BSN 303-864-5416 mann.shelley@tchden.org | |
Principal Investigator: Scott Sagel, MD | |
United States, Missouri | |
Washington University | Recruiting |
St. Louis, Missouri, United States, 63130 | |
Contact: Jane Quante, RN 314-454-2353 quante_j@kids.wustl.edu | |
Principal Investigator: Thomas Ferkol, MD | |
United States, North Carolina | |
University of North Carolina at Chapel Hill | Recruiting |
Chapel Hill, North Carolina, United States, 27599 | |
Contact: Susan Minnix, RN, BSN 919-843-5308 sminnix@med.unc.edu | |
Principal Investigator: Margaret W. Leigh, MD | |
Sub-Investigator: Michael R. Knowles, MD | |
Sub-Investigator: Stephanie Davis, MD | |
United States, Washington | |
Children's Hospital and Regional Medical Center | Recruiting |
Seattle, Washington, United States, 98105 | |
Contact: Sharon McNamara, RN, MN 206-987-3921 ext 1 sharon.mcnamara@seattlechildrens.org | |
Canada, Ontario | |
The Hospital for Sick Children | Recruiting |
Toronto, Ontario, Canada | |
Contact: Donna Wilkes 416-813-4903 donna.wilkes@sickkids.ca | |
Principal Investigator: Margaret Rosenfeld, MD | |
Sub-Investigator: Rob Gibson, MD |
Responsible Party: | University of North Carolina, Chapel Hill ( Margaret W. Leigh, MD ) |
Study ID Numbers: | RDCRN 5901 |
Study First Received: | March 20, 2007 |
Last Updated: | December 15, 2008 |
ClinicalTrials.gov Identifier: | NCT00450918 |
Health Authority: | United States: Federal Government |
Kartagener Syndrome |
Otorhinolaryngologic Diseases Heart Diseases Primary ciliary dyskinesia Cardiovascular Abnormalities Bronchiectasis Disease Progression Central Nervous System Diseases Situs Inversus Dyskinesias Kartagener Syndrome Dextrocardia |
Kartagener syndrome Situs inversus viscerum Signs and Symptoms Genetic Diseases, Inborn Respiratory Tract Diseases Movement Disorders Ciliary Motility Disorders Neurologic Manifestations Congenital Abnormalities Heart Defects, Congenital |
Respiratory System Abnormalities Bronchial Diseases Nervous System Diseases Cardiovascular Diseases |