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A Longitudinal, Observational Study of Primary Ciliary Dyskinesia in Adults
Sponsor: ReCode Therapeutics
Summary
The goal of this observational study is to characterize clinical measures and biomarkers of airway disease in adults with primary ciliary dyskinesia (PCD) and in a group of healthy volunteers (HV) to establish normative values. Lung function, mucociliary clearance, radiological findings, and clinical findings will be assessed. Furthermore, quality of life will be assessed using QOL-PCD, a disease specific questionnaire.
Key Details
Gender
All
Age Range
18 Years - 60 Years
Study Type
OBSERVATIONAL
Enrollment
50
Start Date
2023-03-08
Completion Date
2025-06-30
Last Updated
2025-04-29
Healthy Volunteers
Yes
Conditions
Interventions
Spirometry
To assess lung function
Multiple Breath Washout (MBW)
To measure Lung Clearance Index (LCI)
Mucociliary Clearance (MCC)
To measure lung clearance after the inhalation of radiolabeled particles
CT of the chest
Low radiation to assess structural lung disease
MRI of the chest
To assess lung function and structural lung disease
Locations (1)
University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, United States