A National Registry For Pulmonary Alveolar Proteinosis
Children's Hospital Medical Center, Cincinnati
500 participants
Apr 1, 2015
OBSERVATIONAL
Conditions
Summary
The major goal of Part A of this study is to establish a National PAP Registry to help make reliable new research tests available to doctors to improve the diagnosis of PAP, increase awareness and knowledge of PAP, and give patients a 'seat at the table' in planning and conducting PAP research including the clinical testing of several new potential therapies. The major goal of Part B of this study is to define the natural history of autoimmune PAP (aPAP), develop a disease severity score that reflects how aPAP patients feel and function, and to develop and test novel tools to measure the severity of aPAP lung disease. Funding Source - FDA OOPD
Eligibility
Inclusion Criteria12
- Written informed consent and assent, if applicable
- History of chest computed tomogram or chest radiograph findings compatible with PAP
- History of diagnosis of PAP made by at least one of the following methods:
- Positive (Abnormal) serum GMAb test -OR-
- Lung biopsy clearly documenting the presence of PAP of any type or degree -OR-
- Bronchoalveolar lavage cytology compatible with PAP -OR-
- Recessive or compound mutations in genes known to cause PAP, i.e. GM-CSF receptor α or β chain, GM-CSF, surfactant protein B or C or ABCA3, ABCG1, ABCA1, TTF1
- Diagnosis of autoimmune PAP as indicated by:
- Positive (Abnormal) Serum GMAb Test -AND-
- History of chest CT or x-rays findings compatible with PAP -OR-
- Lung biopsy clearly documenting the presence of PAP of any type or degree -OR-
- Bronchoalveolar lavage cytology compatible with PAP
Exclusion Criteria5
- Individuals who have a serious medical illness that, in the opinion of the investigator, is likely to interfere with completion of the study will be excluded.
- For Part A (Cross-sectional Study of PAP Syndrome)
- Individuals that do not have a diagnosis of PAP
- For Part B (Longitudinal \& PRO Survey Study of autoimmune PAP Patients)
- Individuals that do not have a diagnosis of autoimmune PAP
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Locations(1)
View Full Details on ClinicalTrials.gov
For the most up-to-date information, visit the official listing.
NCT02461615