GM-CSF in Patients With Pulmonary Alveolar Proteinosis
- Conditions
- Pulmonary Alveolar Proteinosis
- Registration Number
- NCT00030056
- Lead Sponsor
- The Cleveland Clinic
- Brief Summary
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).
- Detailed Description
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
Recruitment & Eligibility
- Status
- TERMINATED
- Sex
- All
- Target Recruitment
- 48
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (3)
Cleveland Clinic Foundation
🇺🇸Cleveland, Ohio, United States
The University of Pennsylvania Medical Center
🇺🇸Philadelphia, Pennsylvania, United States
National Jewish Medical Center
🇺🇸Denver, Colorado, United States