Status:
RECRUITING
A National Registry For Pulmonary Alveolar Proteinosis
Lead Sponsor:
Children's Hospital Medical Center, Cincinnati
Collaborating Sponsors:
Rare Diseases Clinical Research Network
National Heart, Lung, and Blood Institute (NHLBI)
Conditions:
Pulmonary Alveolar Proteinosis
Eligibility:
All Genders
Brief 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 kno...
Detailed Description
PAP is a rare syndrome of surfactant accumulation and resulting hypoxemic respiratory failure that occurs in a number of diseases classified pathogenically into three groups: primary PAP (caused by di...
Eligibility Criteria
Inclusion Criteria for Part A and Part B:
- Written informed consent and assent, if applicable
Inclusion Criteria for Part A (Cross Sectional Study of PAP Syndrome)
-
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
Inclusion Criteria For Part B (Longitudinal & PRO Survey Study of autoimmune PAP Patients)
-
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 Criteria or Part A and Part B:
- 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
Key Trial Info
Start Date :
April 1 2015
Trial Type :
OBSERVATIONAL
Allocation :
ESTIMATED
End Date :
December 1 2030
Estimated Enrollment :
500 Patients enrolled
Trial Details
Trial ID
NCT02461615
Start Date
April 1 2015
End Date
December 1 2030
Last Update
March 17 2026
Active Locations (1)
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1
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229