Long-term Lung Function and Disease Progression in Children With Early Onset Primary Ciliary Dyskinesia Lung Disease

Trial ID or NCT#

NCT00722878

Status

not recruiting iconNOT RECRUITING

Purpose

Primary ciliary dyskinesia (PCD), also known as Kartagener syndrome, is a genetic disorder of the cilia, which are microscopic hair-like cells. Cilia work to keep the respiratory system clean by moving mucus that contains debris to the large airways, where it can be coughed out. People with PCD have cilia that do not move properly and therefore are not effective in cleaning the respiratory system. This study will determine when PCD starts and how it changes over time, specifically in terms of how well the lungs work, what germs grow in lung secretions, and how the lungs look on computed tomography (CT) scans.

Official Title

Early Onset and Progression of Primary Ciliary Dyskinesia Lung Disease Prior to 10 Years of Age

Eligibility Criteria

Ages Eligible for Study: Younger than 4 Years
Sexes Eligible for Study: ALL
Accepts Healthy Volunteers: No
Inclusion Criteria:
  1. * Younger than 5 years of age* Diagnosis of PCD or probable PCD based on criteria listed above* Parent or legal guardian willing to give informed consent
Exclusion Criteria:
  1. * Unable to attend follow-up appointments* History of lung transplant* Any co-existing severe diseases that may have significant impact on lung function, respiratory infections, or overall health status (i.e., severe congenital heart disease, severe scoliosis, AIDS, cancer, or end-stage kidney disease)

Investigator(s)

Carlos Milla
Mark M. Davis