Pulmonary Alveolar Proteinosis Clinical Trial
Official title:
Trial of GM-CSF for Alveolar Proteinosis
This is a study to determine the efficacy and safety of granulocyte-macrophage colony-stimulating factor (GM-CSF, sargramostim) administered subcutaneously to patients with pulmonary alveolar proteinosis (PAP).
PAP is a rare lung disease characterized by accumulation of surfactant phospholipids and
proteins within the lungs. There is no specific pharmacologic therapy for PAP and the
current practice of lung lavage under general anesthesia is invasive and has limitations.
Although it is unknown if the anti GM-CSF antibody is related to the disease pathogenesis,
observations suggest a role for GM-CSF in lung homeostasis as well as in the pathogenesis of
PAP.
Patients will receive subcutaneous GM-CSF or placebo once a day and will be followed on an
outpatient basis at 2 weeks, and 1, 2, 3, 4, 5 and 6 months after initiation of therapy.
Clinical response will determine dosing schedule and will be evaluated by symptom scores,
gas exchange data, and chest radiographs.
Completion date provided represents the completion date of the grant per OOPD records
;
Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Primary Purpose: Treatment
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