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A Longitudinal, Observational Study of Primary Ciliary Dyskinesia in Adults
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.
Conditions:
🦠 Primary Ciliary Dyskinesia
🗓️ Study Start (Actual) 8 March 2023
🗓️ Primary Completion (Estimated) 20 December 2026
✅ Study Completion (Estimated) 20 May 2027
👥 Enrollment (Estimated) 50
🔬 Study Type OBSERVATIONAL
📊 Phase N/A
Locations:
📍 Chapel Hill, North Carolina, United States

📋 Eligibility Criteria

Description

    Inclusion Criteria:

    • * PCD diagnosis with confirmation of 2 identified pathogenic genetic variants within 1 of the following ultrastructure variants:
    • * DNAI1 ODA defect
    • * Other ODA defect
    • * IDA - MTD defect
    • * RS defect
    • * Informed consent

    Exclusion Criteria:

    • * Are a current smoker (e-cigarette, tobacco, or marijuana)
    • * Are a former smoker who discontinued smoking \<1 year prior to enrollment or has a cumulative 1+ pack-year smoking history
    • * Have a recent stable forced expiratory volume in one second (FEV1) \<35% predicted
    • * Have contraindications for MRI studies (implanted devices/materials; inability to tolerate; claustrophobia or severe anxiety that would preclude MRI/imaging)
    • * Have had a significant clinical radiation exposure (as determined by the investigator) within the past 6 months. Potential participants who have had a chest CT within the past 6 months may be eligible to be enrolled and their clinical CT will be utilized as the baseline for this study
    • * Are pregnant or breastfeeding
    • * Have any comorbidities likely to impact lung function (e.g., complex congenital heart disease, severe scoliosis, diseases involving immune dysregulation, lung transplantation, lung lobectomy, end-stage renal disease, or poor overall health status).
Ages Eligible for Study: 18 Years to 60 Years (ADULT)
Sexes Eligible for Study: ALL
Accepts Healthy Volunteers: Yes

🗓️ Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

  • First Submitted 19 December 2022
  • First Submitted that Met QC Criteria 5 January 2023
  • First Posted 13 January 2023

Study Record Updates

  • Last Update Submitted that Met QC Criteria 27 April 2023
  • Last Update Posted 1 May 2023
  • Last Verified April 2023
I'm interested ...!!