A Longitudinal, Observational Study of Primary Ciliary Dyskinesia in Adults
Completed
Conditions studied: Primary Ciliary Dyskinesia
In brief
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 facts
- Study ID
- NCT05685186
- Run by
- ReCode Therapeutics
- People needed
- 31
- Starts
- 2023-03-08
- Expected to finish
- 2025-06-30
- Last updated by the study team
- 2026-05-04
Who can join
Age: 18 and older, up to 60. Sex: any. Healthy volunteers: accepted.
You may qualify if…
- 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
You may not qualify if…
- 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).
Where it is running
- University of North Carolina at Chapel Hill — Chapel Hill, North Carolina, United States
Full record on ClinicalTrials.gov
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