Status:
UNKNOWN
Inhalation of Granulocyte-macrophage Colony-stimulating Factor (GM-CSF) for Autoimmune Pulmonary Alveolar Proteinosis (PAP)
Lead Sponsor:
Peking Union Medical College Hospital
Collaborating Sponsors:
The Affiliated Nanjing Drum Tower Hospital of Nanjing University Medical School
Conditions:
Autoimmune Pulmonary Alveolar Proteinosis
Eligibility:
All Genders
18+ years
Phase:
PHASE2
Brief Summary
The purpose of the study is to evaluate if inhaled granulocyte-macrophage colony stimulating factor delay the increase in alveolar-arterial oxygen difference, compared to no treatment, for adult patie...
Detailed Description
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 gra...
Eligibility Criteria
Inclusion
- Accessible population:
- 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.
Exclusion
- 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.
- Pregnancy.
Key Trial Info
Start Date :
August 1 2014
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
August 1 2017
Estimated Enrollment :
42 Patients enrolled
Trial Details
Trial ID
NCT02243228
Start Date
August 1 2014
End Date
August 1 2017
Last Update
September 17 2014
Active Locations (1)
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1
Peking Union Medical College Hospital
Beijing, Beijing Municipality, China, 100730