Clinical Trial: Study of Subcutaneous Injection of Low-dose rhGM-CSF +/- WLL in PAP.

Study Status: Recruiting
Recruit Status: Unknown status
Study Type: Interventional

Official Title: Study of Subcutaneous Injection of Low-dose Recombinant Granulocyte Macrophage-Colony Stimulating Factor (rhGM-CSF) +/- Whole Lung Lavage(WLL) in Pulmonary Alveolar Proteinosis

Brief Summary: The purpose of this study is to establish an efficient and economic treatment scheme by evaluation of the safety and efficacy of subcutaneous injection of low-dose rhGM-CSF, or of similar injection after whole lung lavage , in patients with PAP.

Detailed Summary: The purpose of this study is to establish an efficient and economic treatment scheme by evaluation of the safety and efficacy of subcutaneous injection of low-dose rhGM-CSF, or of similar injection after whole lung lavage , in patients with PAP. During the observation, study visits will occur at the end of each month. During the 1-year follow-up period which is lasting 6 months after the treatment, all participants will be required to check the various efficacy indicators.
Sponsor: Shanghai Pulmonary Hospital, Shanghai, China

Current Primary Outcome: Improvements in double pulmonary diffuse lesions （Chest CT score ） [ Time Frame: 6 months ]

Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Clinical symptoms observation: shod of breath, cough (according to each score standard） [ Time Frame: 6 months ]
  • Granulocyte Macrophage Colony Stimulating Factor(GM-CSF) Antibody titer change [ Time Frame: 6 months ]
  • Improvements in pulmonary function [ Time Frame: 6 months ]
    Pulmonary function tests include residual volume/total lung capacity(RV/TLC), forced vital capacity(FVC), forced expiratory volume in one second/forced vital capacity(FEV1/FVC), diffusing capacity of carbon monoxide(DLCO).
  • Improvements in arterial blood gas, including alveolar-arterial oxygen difference(A-aDO2), partial pressure of oxygen(PaO2), partial pressure of carbon dioxide in artery(PaCO2), arterial oxygen saturation(SaO2). [ Time Frame: 6 months ]


Original Secondary Outcome:

  • Clinical symptoms observation: shod of breath, cough (according to each score standard） [ Time Frame: 6 months ]
  • GM-CSF Antibody titer change [ Time Frame: 6 months ]
  • Improvements in pulmonary function（RV/TLC, FVC, FEV1/FVC, DLCO） [ Time Frame: 6 months ]
  • Improvements in arterial blood gas （A-aDO2、PaO2、PaCO2、SaO2） [ Time Frame: 6 months ]


Information By: Shanghai Pulmonary Hospital, Shanghai, China

Dates:
Date Received: November 7, 2013
Date Started: January 2012
Date Completion: October 2014
Last Updated: November 14, 2013
Last Verified: November 2013