Home › NCT02461615
A National Registry For Pulmonary Alveolar Proteinosis
Who can join
All ages · All sexes
Full 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
About the study
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
Sponsor: Children's Hospital Medical Center, Cincinnati · Participants: 500 · Started: 2015-04
Contact the study team
- Brenna C Carey, Ms, PhD · Phone: 513-636-8916
Official record on ClinicalTrials.gov — NCT02461615
Locations in the U.S.
| Ohio | Cincinnati Children's Hospital Medical Center, Cincinnati |
From ClinicalTrials.gov, data retrieved Sep 30, 2026. Each study sets its own eligibility; the study team decides who can join.