GM-CSF in Patients With Pulmonary 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).
Pulmonary Alveolar Proteinosis
Drug: GM-CSF (granulocyte-macrophage colony-stimulating factor, sargramostim)
|Study Design:||Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Primary Purpose: Treatment
|Official Title:||Trial of GM-CSF for Alveolar Proteinosis|
|Study Start Date:||September 2001|
|Estimated Study Completion Date:||December 2005|
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
Please refer to this study by its ClinicalTrials.gov identifier: NCT00030056
|United States, Colorado|
|National Jewish Medical Center|
|Denver, Colorado, United States|
|United States, Ohio|
|Cleveland Clinic Foundation|
|Cleveland, Ohio, United States, 44195|
|United States, Pennsylvania|
|The University of Pennsylvania Medical Center|
|Philadelphia, Pennsylvania, United States|