Clinical Trial: GM-CSF in Patients With Pulmonary Alveolar Proteinosis

Study Status: Terminated
Recruit Status: Terminated
Study Type: Interventional

Official Title: Trial of GM-CSF for Alveolar Proteinosis

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 Summary:

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

Sponsor: The Cleveland Clinic

Current Primary Outcome:

Original Primary Outcome:

Current Secondary Outcome:

Original Secondary Outcome:

Information By: FDA Office of Orphan Products Development

Dates:
Date Received: January 30, 2002
Date Started: September 2001
Date Completion: December 2005
Last Updated: March 24, 2015
Last Verified: January 2002