NCT00030056

GM-CSF in Patients With Pulmonary Alveolar Proteinosis

Trial of GM-CSF for Alveolar Proteinosis

• Status: Terminated
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 48 (estimated)
• Sponsor: The Cleveland Clinic · Academic / Other
• Sex: All
• Age: 18 Years – 70 Years
• Healthy volunteers: Not accepted

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

Conditions

• Pulmonary Alveolar Proteinosis

Interventions

Type	Name	Description
DRUG	GM-CSF (granulocyte-macrophage colony-stimulating factor, sargramostim)

Timeline

• Start date: 2001-09-01
• Completion: 2005-12-01
• First posted: 2002-01-31
• Last updated: 2015-03-25

Locations

3 sites across 1 country: United States