A National Registry For Pulmonary Alveolar Proteinosis
ConditionPulmonary Alveolar Proteinosis
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
AgeNot listed
SponsorChildren's Hospital Medical Center, Cincinnati
About this trial
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 criteria
Qualifiers
Written informed consent and assent, if applicable
History of chest computed tomogram or chest radiograph findings compatible with PAP
Positive (Abnormal) serum GMAb test -OR-
Lung biopsy clearly documenting the presence of PAP of any type or degree -OR-
Disqualifiers
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.
Individuals that do not have a diagnosis of PAP
Individuals that do not have a diagnosis of autoimmune PAP
Trial design
Treatments tested in this trial
- Not listed
Trial groups
500 Participants
are grouped into 1 trial groupSponsors and collaborators
Children's Hospital Medical Center, Cincinnati
Lead sponsor
Rare Diseases Clinical Research Network
Collaborator
National Heart, Lung, and Blood Institute (NHLBI)
Collaborator
National Center for Advancing Translational Sciences (NCATS)
Collaborator
Source ClinicalTrials.gov