A Longitudinal, Observational Study of Primary Ciliary Dyskinesia in Adults (NCT05685186) | Clinical Trial Compass
CompletedNot Applicable
A Longitudinal, Observational Study of Primary Ciliary Dyskinesia in Adults
United States31 participantsStarted 2023-03-08
Plain-language 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.
Who can participate
Age range18 Years – 60 Years
SexALL
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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).