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
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|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 ]
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|Ages Eligible for Study:||18 Years and older (Adult, Older Adult)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
Adult patients with mild-to-moderate autoimmune pulmonary alveolar and without spontaneous remission will be enrolled at Peking Union Medical College Hospital and Nanjing Drum Tower Hospital.
- Adult autoimmune PAP subjects will be included: 1) a positive PAS stain from BALF or lung biopsy; 2) high level of serum anti-GM-CSF antibody (>2.39ug/ml, the cut-off point in our hospital); 3) age above 18 years old; 4) exclude hereditary and secondary PAP.
- Able to give written informed consent and comply with the requirements of the study.
- Patients are not eligible for the whole lung lavage (WLL), decided by clinicians.
- Eligibility for GM-CSF inhalation: 1) Disease severity score (DSS) is 1-3; 2) No treatment with GM-CSF therapy or WLLin the 3 months prior to enrollment. Definition of DSS2: 1, no symptom and PaO2>=70mmHg；2, PaO2>=70mmHg with symptoms；3, PaO2>=60 and <70mmHg; 4, PaO2>=50 and <60mmHg; 5, PaO2<50mmHg.
- Patients will be observed for 3 months and all APAP patients who resolved spontaneously will be excluded from our study.
- PAP resulting from another condition (e.g. occupational exposure to silica, underlying HIV, respiratory infections, myeloproliferative disorder or leukaemia);
- A normal or low-titre serum anti-GM-CSF antibody (≤2.39ug/ml);
- History of severe allergic or anaphylactic reactions to humanized or murine monoclonal antibodies；
- Chronic lung disease associated with already existing respiratory failure (such as pulmonary emphysema or fibrosis);
- Other serious medical conditions which, in the opinion of the investigator, would make the patient unsuitable for the study.
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 email@example.com|
|Contact: Xin-Lun Tian, M.D.||10-69155039 ext firstname.lastname@example.org|
|Peking Union Medical College Hospital||Recruiting|
|Beijing, Beijing, China, 100730|
|Contact: Kai-Feng Xu, M.D. 10-69155039 ext 86 email@example.com|
|Contact: Xin-Lun Tian, M.D. 10-69155039 ext 86 firstname.lastname@example.org|
|Principal Investigator: Xin-Lun Tian, M.D.|
|Principal Investigator:||Kai-Feng Xu, M.D.||Peking Union Medical College Hosptial|
|Responsible Party:||Kaifeng Xu, MD, Peking Union Medical College Hospital|
|Other Study ID Numbers:||
|First Posted:||September 17, 2014 Key Record Dates|
|Last Update Posted:||September 17, 2014|
|Last Verified:||September 2014|
granulocyte-macrophage colony stimulating factor(GM-CSF)
Autoimmune pulmonary alveolar proteinosis (PAP)
Pulmonary Alveolar Proteinosis
Respiratory Tract Diseases
Immune System Diseases