Clinical Trial: Inhaled Granulocyte-Macrophage Colony Stimulating Factor (GM-CSF) in Hereditary Pulmonary Alveolar Proteinosis (PAP)
Study Status: Completed
Recruit Status: Completed
Study Type: Interventional
Official Title: Inhaled Granulocyte-Macrophage Colony Stimulating Factor (GM-CSF) in Hereditary Pulmonary Alveolar Proteinosis (PAP)
Brief Summary: The purpose of this study is to evaluate the therapeutic efficacy of inhaled recombinant human GM-CSF in individuals with hereditary Pulmonary Alveolar Proteinosis (PAP) due to partial dysfunction of the GM-CSF receptor.
Detailed Summary:
Sponsor: Children's Hospital Medical Center, Cincinnati
Current Primary Outcome: Time for the oxygen saturation to fall below 88% [ Time Frame: 6 months ]
Original Primary Outcome: Same as current
Current Secondary Outcome:
- Improvement in pulmonary function [ Time Frame: 6 months ]
- Improvement in exercise tolerance [ Time Frame: 6 months ]
- Improvement in CT scan [ Time Frame: 6 months ]
- Improvement in Quality of Life [ Time Frame: 6 months ]
- Improvement in dyspnea and fatigue [ Time Frame: 6 months ]
- Biomarkers will be assayed to detect changes from baseline values: surfactant protein-D (SP-D), KL-6, CEA), GM-CSF, GM-CSF autoantibodies, macrophage colony stimulating factor (M-CSF), and macrophage inflammatory protein -1 alpha MIP-1alpha. [ Time Frame: 6 months ]
Original Secondary Outcome: Same as current
Information By: Children's Hospital Medical Center, Cincinnati
Dates:
Date Received: December 12, 2011
Date Started: August 2012
Date Completion:
Last Updated: June 3, 2014
Last Verified: January 2013
