Inhalation of Granulocyte-macrophage Colony-stimulating Factor (GM-CSF) for Autoimmune Pulmonary Alveolar Proteinosis (PAP)

• Register: ClinicalTrials.gov
• Last refreshed on: 19 February 2015
• Main ID: NCT02243228
• Date of registration: 14/09/2014
• Prospective Registration: No
• Primary sponsor: Peking Union Medical College Hospital
• Public title: Inhalation of Granulocyte-macrophage Colony-stimulating Factor (GM-CSF) for Autoimmune Pulmonary Alveolar Proteinosis (PAP)
• Scientific 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
• Date of first enrolment: August 2014
• Target sample size: 42
• Recruitment status: Recruiting
• URL: http://clinicaltrials.gov/show/NCT02243228
• Study type: Interventional
• Study design: Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
• Phase: Phase 2

Countries of recruitment

China

Contacts

• Name: Kai-Feng Xu, M.D.
• Affiliation: Peking Union Medical College Hosptial

• Name: Kai-Feng Xu, M.D.

• Name: Kai-Feng Xu, M.D.

Key inclusion & exclusion criteria

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.

Inclusion Criteria:

- 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 Criteria:

- 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.



Age minimum: 18 Years
Age maximum: N/A
Gender: Both

Health Condition(s) or Problem(s) studied

Autoimmune Pulmonary Alveolar Proteinosis

Intervention(s)

Drug: GM-CSF

Primary Outcome(s)

A-aDO2 difference [Time Frame: 2 years]

Secondary Outcome(s)

Severe adverse event [Time Frame: 2 years]

FEV1 difference [Time Frame: 2 years]

6 minutes walking distance difference [Time Frame: 2 years]

Time to relapse [Time Frame: up to 2 years]

Secondary ID(s)

XT 01

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

The Affiliated Nanjing Drum Tower Hospital of Nanjing University Medical School