Inhalation of Granulocyte-macrophage Colony-stimulating Factor (GM-CSF) for Autoimmune Pulmonary Alveolar Proteinosis (PAP)
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|ClinicalTrials.gov Identifier: NCT02243228|
Recruitment Status : Unknown
Verified September 2014 by Kaifeng Xu, Peking Union Medical College Hospital.
Recruitment status was: Recruiting
First Posted : September 17, 2014
Last Update Posted : September 17, 2014
|Condition or disease||Intervention/treatment||Phase|
|Autoimmune Pulmonary Alveolar Proteinosis||Drug: GM-CSF||Phase 2|
Autoimmune pulmonary alveolar proteinosis (PAP, previously known as idiopathic PAP) is a rare interstitial lung disease elicited by the formation of autoantibodies which neutralize the activity of granulocyte-macrophage colony-stimulating factor (GM-CSF) which decreases macrophage clearance of surfactant.
Currently, the standard treatment strategy for PAP is whole lung lavage (WLL)，which is invasive and has limitations. Inhaled GM-CSF therapy became a new option for PAP patients not only because of its effectiveness and safety, but it is convenient way for patients who are reluctant to do operation as well. We are planning to prospectively evaluate if inhaled granulocyte-macrophage colony stimulating factor would delay the increase in alveolar-arterial oxygen difference (A-aDO2, which is the most sensitive factor in evaluating APAP5), compared to placebo, for patients with mild-to-moderate autoimmune pulmonary alveolar over a two-year period.
A total of 42 subjects with APAP who meet the inclusion criteria will be enrolled at Peking Union Medical College Hospital and Nanjing Drum Tower Hospital. After observe APAP patients for 3 months to rule out patients who resolved spontaneously, the participants will undergo randomization (by random number table)and stratified into two different groups by their DSS. Then they will be treated by GM-CSF (using nebulizer, 150ug bid) every other week or no treatment for 6 months, and will be followed on an outpatient basis at 2 weeks, and 1, 3, 6, 9, 12, 15, 18, 21 and 24 months after initiation of therapy.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||42 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Prospective Study of Inhaling Granulocyte-macrophage Colony Stimulating Factor in Adult Patients With Mild-to-moderate Autoimmune Pulmonary Alveolar Proteinosis in China: a Randomized Open-label Study|
|Study Start Date :||August 2014|
|Estimated Primary Completion Date :||August 2017|
|Estimated Study Completion Date :||August 2017|
Experimental: GM-CSF, nebulizer
After the patients were randomly divided into two groups, they will be treated by GM-CSF (using nebulizer, 150ug bid) every other week for 6 months.
Other Name: HUABEI JIMUXIN
- A-aDO2 difference [ Time Frame: 2 years ]
- Time to relapse [ Time Frame: up to 2 years ]The definition of relapse were as follow: 1) newly requirement for whole lung lavage (WLL); or 2) PAP associated death; or 3) a reduction in PaO2 of more than 10mmHg, or an increase in DA-aO2 of more than 10mmHg; or 4) a worsen chest HRCT.
- FEV1 difference [ Time Frame: 2 years ]
- 6 minutes walking distance difference [ Time Frame: 2 years ]
- Severe adverse event [ Time Frame: 2 years ]
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02243228
|Contact: Kai-Feng Xu, M.D.||10-69155039 ext firstname.lastname@example.org|
|Contact: Xin-Lun Tian, M.D.||10-69155039 ext email@example.com|
|Peking Union Medical College Hospital||Recruiting|
|Beijing, Beijing, China, 100730|
|Contact: Kai-Feng Xu, M.D. 10-69155039 ext 86 firstname.lastname@example.org|
|Contact: Xin-Lun Tian, M.D. 10-69155039 ext 86 email@example.com|
|Principal Investigator: Xin-Lun Tian, M.D.|
|Principal Investigator:||Kai-Feng Xu, M.D.||Peking Union Medical College Hosptial|