View clinical trials related to Pulmonary Alveolar Proteinosis.
Filter by:The major goal of this study is to evaluate a new type of cell transplantation therapy for individuals with hereditary PAP, study a new treatment that may be useful for treatment of other diseases, and research mechanisms that drive the development and function of lung macrophages.
Mutations in the MARS gene encoding methionyl-tRNA synthetase are responsible for a genetic form of alveolar proteinosis (PAP), but the pathophysiological mechanisms of the respiratory phenotype are not known. The main hypothesis is that the PAP phenotype in these patients is secondary to a defective clearance of the surfactant by the alveolar macrophages. The main objective of the study is to study the clearance capacity of lipoproteinaceous material by macrophages of patients with MARS related PAP. This will be investigate in cultured macrophages derived from peripheral blood monocytes of patients (patients with MARS related PAP) and controls (patients without MARS related PAP).
The purpose of the study is to evaluate the effectiveness of the sequential therapy with whole lung Lavage (WLL)/inhaling granulocyte-macrophage colony stimulating factor, compared to WLL only, for adult patients with severe autoimmune pulmonary alveolar proteinosis in China over a two-year period.
Generation of a common European database and biobank Continous assessment and implementation of guidelines and treatment protocols Establishment of a large observational cohort of chILD patients Determination the value of outcomes used in child Assess treatment variations used, deliver data from defined protocols and linked outcomes
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
The purpose of the study is to evaluate if inhaled granulocyte-macrophage colony stimulating factor delay the increase in alveolar-arterial oxygen difference, compared to no treatment, for adult patients with mild-to-moderate autoimmune pulmonary alveolar proteinosis in China over a two-year period.
The purpose of this study is to establish an efficient and economic treatment scheme by evaluation of the safety and efficacy of subcutaneous injection of low-dose rhGM-CSF, or of similar injection after whole lung lavage , in patients with PAP.