Longitudinal Characterization of Respiratory Tract Exacerbations and Treatment Responses in Primary Ciliary Dyskinesia

Completed

Conditions studied: Primary Ciliary Dyskinesia, Kartagener Syndrome

In brief

The overall objective of this longitudinal, observational study is to provide information needed to inform the design of future interventional trials of respiratory exacerbation prevention and treatment in children and adults with primary ciliary dyskinesia (PCD).

Key facts

Study ID

NCT05161858

Run by

University of North Carolina, Chapel Hill

People needed

105

Starts

2022-03-29

Expected to finish

2025-09-30

Last updated by the study team

2025-12-10

Who can join

Age: 6 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Diagnosis of PCD
• Clinical features consistent with PCD PLUS
• At least 1 diagnostic test consistent with PCD:
• i) Biallelic pathogenic variants in PCD-associated genes identified by genetic panel testing including deletion/duplication analysis; ii) Ciliary ultrastructural defect by transmission electron microscopy known to be disease-causing, including outer dynein arm defects, outer dynein arm plus inner dynein arm (IDA) defects, IDA defects with microtubular disorganization and absent central pair
• Age ≥ 6 years
• At least one course of antibiotics (oral or IV) in the prior year prescribed to treat new or increased respiratory symptoms
• Smart phone and/or internet access available in home
• Informed consent provided by participant or parent/guardian, with assent provided as applicable

You may not qualify if…

• Acute course of antibiotics for respiratory symptoms completed <14 days prior to enrollment or Visit 1 (evaluated at enrollment and Visit 1; visit may be rescheduled >14 days after completion of antibiotics)
• Developmental or cognitive disability that would impair ability to complete PRO instruments or perform spirometry
• Congenital heart disease OTHER THAN repaired or resolved atrial septal defect (ASD) or ventricular septal defect (VSD)
• Asplenia or functional asplenia
• Co-existing non-pulmonary disease that, in the opinion of the investigator, could have significant impact on lung function or health-related quality of life (e.g., severe scoliosis) or overall health status (e.g., cancer, severe renal disease)
• Listed for or post-lung transplantation

Where it is running

• Stanford University — Palo Alto, California, United States
• Children's Hospital Colorado — Aurora, Colorado, United States
• Washington University in St. Louis — St Louis, Missouri, United States
• University of North Carolina at Chapel Hill — Chapel Hill, North Carolina, United States
• Seattle Children's Hospital — Seattle, Washington, United States
• The Hospital for Sick Children — Toronto, Ontario, Canada
• McGill University — Montreal, Quebec, Canada

Full record on ClinicalTrials.gov

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