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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register.
Register: ClinicalTrials.gov
Last refreshed on: 12 December 2020
Main ID:  NCT02835742
Date of registration: 13/07/2016
Prospective Registration: Yes
Primary sponsor: Niigata University Medical & Dental Hospital
Public title: Pulmonary Alveolar Proteinosis GM-CSF Inhalation Efficacy Trial in Japan
Scientific title: Pulmonary Alveolar Proteinosis GM-CSF Inhalation Efficacy Trial in Japan
Date of first enrolment: September 1, 2016
Target sample size: 78
Recruitment status: Completed
URL:  https://clinicaltrials.gov/show/NCT02835742
Study type:  Interventional
Study design:  Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).  
Phase:  Phase 2
Countries of recruitment
Japan
Contacts
Key inclusion & exclusion criteria

Inclusion Criteria:

1. Age over 16 years and below 80 years (as of the date of registration).

2. Can provide signed informed consent.

3. Willing and able to comply with scheduled visits, treatment plan, laboratory tests,
and other study procedures specified in the protocol (including short-term hospital
admission).

4. Autoimmune pulmonary alveolar proteinosis diagnosed by both HR-CT and biopsy and/or
BAL as well as GM-CSF antibodies in serum positive.

5. PaO2 < 70 mmHg after 5 minutes spine position at room air, or PaO2 < 75 mmHg after 5
minutes spine position at room air and with symptom(s) including cough, sputum and
exertional dyspnea

Exclusion Criteria:

1. Diagnosed as secondary or hereditary pulmonary alveolar proteinosis

2. WBC of 12,000/mm3 or more

3. Fever of 38 degree celsius or more

4. Severe edema

5. History of malignant disease within recent 5 years (not applied to the treated cases
of uterine carcinoma in situ and local basal cell carcinoma)

6. Complication of cardiovascular diseases including congestive heart failure, angina
pectoris, hemorrhagic tendency, etc with severe condition.

7. Complication of respiratory diseases such as pulmonary infectious disease(incl.
pulmonary tuberculosis), bronchial asthma, lung fibrosis ,interstitial pneumonitis, or
bronchiectasis, in which the evaluations of safety and efficacy of GM-CSF therapy are
considered as difficult.

8. History or complication of infectious diseases which require systemic administration
of antibiotics, antifungal or antiviral agents within recent 2 weeks.

9. Treatment with other cytokines

10. Pregnant or possibly pregnant women, lactating women, and women who desire to become
pregnant during the study period

11. Patients who have been treated with whole-lung lavage, repeated segmental-lung lavage,
or rituximab within 6 months before the start of the study (this criterion does not
apply to patients for whom 6 months or more have elapsed after their last lavage or
rituximab)

12. Severe liver dysfunction (AST > 100 IU/L and/or ALT > 100 IU/L and/or T-bil >3.0mg/dL)

13. Severe renal dysfunction (Ccr < 30 mL/min, calculated by Cockcroft-Gault (CG) formula)

14. Previous experience of severe and unexplained side-effects during aerosol delivery of
any kind of medicinal product

15. Treatment with oral or intravenous administration or inhalation of corticosteroids.

16. Treatment with other inhaled drugs.

17. Previously treated with GM-CSF before the start of the study.

18. Demonstrate hypersensitivity to GM-CSF agent.

19. Other patients judged to be inappropriate for the study by the attending physician
(e.g., patients who are unlikely to complete treatment or are uncooperative).



Age minimum: 16 Years
Age maximum: 80 Years
Gender: All
Health Condition(s) or Problem(s) studied
Pulmonary Alveolar Proteinosis, Autoimmune
Intervention(s)
Drug: Placebo
Drug: Sargramostim
Primary Outcome(s)
Change value of AaDO2 between baseline and 24 weeks [Time Frame: 24 weeks]
Secondary Outcome(s)
Secondary ID(s)
PAGE
Source(s) of Monetary Support
Please refer to primary and secondary sponsors
Secondary Sponsor(s)
Ethics review
Results
Results available:
Date Posted:
Date Completed:
URL:
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