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NCT06546098
Autoimmune pulmonary alveolar proteinosis (aPAP) is a rare disorder in which a material called surfactant builds up in the lungs and makes it hard to breathe. In addition to shortness of breath, people with aPAP can experience persistent cough, overwhelming fatigue, unintentional changes in weight, chest or back pain, suddenly feeling out of shape, and general discomfort. Currently, there are no approved medications for aPAP in the United States, but the symptoms of aPAP can be treated with whole lung lavage (WLL). WLL is an invasive procedure that temporarily removes surfactant, and it can result in serious consequences like trauma to the lung, a collapsed lung, and prolonged requirement for artificial ventilation. Savara is studying an investigational drug called molgramostim nebulizer solution to see if it activates the cells that help clear surfactant from the lungs, which improves oxygen transfer from the lungs to the bloodstream. Molgramostim nebulizer solution is administered by inhalation using a hand-held nebulizer. In clinical trials, molgramostim nebulizer solution has shown improvements in gas exchange and patient reported outcomes. This expanded access program will make molgramostim nebulizer solution available to adult patients with diagnosed aPAP. Access must be obtained through the treating physician. Patients will dose molgramostim nebulizer solution 300 micrograms (mcg) once daily and be followed by their physician every 3 months to assess their clinical status and report any adverse events.
NCT00901511
This is a prospective, randomized, open-label, long-term, phase 2 study of inhaled granulocyte/macrophage-colony stimulating factor following whole lung lavage therapy in patients with autoimmune pulmonary alveolar proteinosis.
NCT03482752
SAV006-03 is an open-label extension study for participants who had completed the IMPALA study. At the baseline visit, eligible participants may continue or re-start treatment with 300 µg inhaled molgramostim (recombinant human Granulocyte-Macrophage Colony Stimulating Factor; GM-CSF) administered intermittently in cycles of seven days molgramostim, administered once daily, and seven days off treatment. Participants will be treated with inhaled molgramostim for up to 36 months. During the trial, whole lung lavage will be applied as rescue therapy.
NCT03531996
The major goal of this study is to conduct a prospective, longitudinal study of autoimmune PAP to examine outcome measures for disease severity of potential use in clinical practice and/or clinical research studies. These results will impact the field by: 1) improving an understanding of the clinical course of autoimmune PAP, 2) providing information on various clinical outcome and quality of life outcome measures to guide patients and physicians in making treatment choices, and 3) facilitate the development of pharmaco-therapeutics for autoimmune PAP and 4) better informing PAP researchers.
NCT03231033
Pulmonary alveolar proteinosis (PAP) is a syndrome of surfactant accumulation, respiratory failure, and innate immune deficiency for which therapy remains limited to whole lung lavage (WLL), an invasive physical procedure to remove surfactant unavailable at most medical centers. While PAP occurs in multiple diseases affecting men, women, and children of all ages and ethnic origins, in 85% of patients, it occurs as an idiopathic disease associated with neutralizing GM-CSF autoantibodies. Basic science and translational research has shown that idiopathic PAP is an autoimmune disease in which disruption of GM-CSF signaling impairs the ability of alveolar macrophages to clear surfactant and perform host defense functions. Recently, it has been shown that cholesterol toxicity drives pathogenesis in alveolar macrophages from GM-CSF deficient (Csf2-/-) mice and patients with autoimmune PAP. Loss of GM-CSF signaling reduces PU.1/CEBP-mediated expression of PPARγ and its downstream target ABCG1 (a cholesterol exporter important in macrophages). The cell responds by esterifying and storing cholesterol in vesicles to reduce toxicity. Eventually, vesicles fill the cell, impair intracellular transport and reduce uptake and clearance of surfactant from the lung surface resulting in disease manifestations. Recent data indicates that pioglitazone, a PPARγ agonist currently approved by the FDA for human use, increases cholesterol/surfactant clearance by alveolar macrophages from autoimmune PAP patients and Csf2-/- mice. Importantly, pioglitazone significantly reduced the severity of PAP lung disease in Csf2-/- mice after several months of therapy. Together, these observations suggest pioglitazone could be 'repurposed' as pharmacologic therapy for PAP.