Defining the Genetic Etiology of Suppurative Lung Disease in Children and Adults (NCT04702243) | Clinical Trial Compass
CompletedNot Applicable
Defining the Genetic Etiology of Suppurative Lung Disease in Children and Adults
United States436 participantsStarted 2020-12-01
Plain-language summary
The investigators will utilize a systematic approach for the diagnostic evaluation of patients to identify characteristics which may distinguish between Primary Immunodeficiency (PID) disorders versus Primary Ciliary Dyskinesia (PCD).
Who can participate
Age range5 Years – 45 Years
SexALL
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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Pediatric subjects (aged 5-17 years): Inclusion criteria include the major criterion (bronchiectasis in \> 1 lobe on current or chest CT in previous 24 months, if available for review), plus one minor criterion, or two minor criteria, if bronchiectasis is not present, (including at least 1 "lung" minor criteria).
Adult subjects (aged 18-45 years): Inclusion criteria include the major criteria (bronchiectasis in \> 1 lobe on current or chest CT in previous 36 months, if available for review), plus one minor criterion, or three minor criteria, if bronchiectasis is not present, (including at least 1 "lung" minor criteria).
Inclusion Criteria:
General Criteria
* Age 5-45 years
* Male and Female Subjects
* All races and ethnicities
Major Clinical Criteria
\- Bronchiectasis in \> 1 lobe
Minor Clinical Criteria, Lung
* Neonatal respiratory distress (in term neonates with O2 requirement)
* Chronic wet cough (year-round for at least 12 months)
* Recurrent episodes of bacterial bronchitis
* Recurrent pneumonia (confirmed on chest x-ray)
* Respiratory non-tuberculous mycobacteria (NTM) (documented respiratory NTM culture)
Minor Clinical Criteria, Other
* Chronic nasal congestion
* Recurrent/chronic paranasal sinusitis
* Ongoing middle-ear disease and/or tympanostomy tube placement at age ≥ 4 years
* Organ laterality defect
* Low nasal nitric oxide (\< 77 nL/min) (by plateau measurement)
* Confirmed family history of PID or PCD
Exclusion Criteria:
* Anyone who has a confirmed…
What they're measuring
1
Number of Participants with a Confirmed Diagnosis of PCD or PID
Timeframe: Up to approximately 4 years
2
Prevalence of Neonatal Respiratory Distress Seen in PCD and PID
Timeframe: During a single 6-hour visit
3
Prevalence of the Onset of Chronic Nasal Congestion Before Six Months of Age Seen in PCD and PID
Timeframe: During a single 6-hour visit
4
Prevalence of the Onset of Daily Wet Cough Before Six Months of Age Seen in PCD and PID
Timeframe: During a single 6-hour visit
5
Prevalence of Laterality Defects Seen in PCD and PID
Timeframe: During a single 6-hour visit
6
Prevalence of Chronic/Recurrent Sinus Disease Seen in PCD and PID
Timeframe: During a single 6-hour visit
7
Prevalence of Chronic/Recurrent Middle Ear Disease Seen in PCD and PID
Timeframe: During a single 6-hour visit
8
Prevalence of Recurrent Pneumonia/Sepsis Seen in PCD and PID