Pulmonary Alveolar Proteinosis GM-CSF Inhalation Efficacy Trial in Japan

Pulmonary Alveolar Proteinosis, Autoimmune Clinical Trial

Official title:

Pulmonary Alveolar Proteinosis GM-CSF Inhalation Efficacy Trial in Japan

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02835742
• Other study ID #: PAGE
• Status: Completed
• Phase: Phase 2
• Start date: September 1, 2016
• Est. completion date: June 2018

Study information

• Verified date: February 2019
• Source: Niigata University Medical & Dental Hospital
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

Objective: Determine the safety and efficacy of GM-CSF inhalation in patients with aPAP.

Study Design: multi-center, randomized, double-blind, placebo- controlled, safety/efficacy study.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 78
• Est. completion date: June 2018
• Est. primary completion date: August 2017
• Accepts healthy volunteers: No
• Gender: All
• Age group: 16 Years to 80 Years

Eligibility

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).

Related Conditions & MeSH terms

• Autoimmune Diseases
• Pulmonary Alveolar Proteinosis
• Pulmonary Alveolar Proteinosis, Autoimmune

Intervention

Drug:
• Sargramostim

• Placebo

Locations

Country	Name	City	State
Japan	Niigata University Med & Dental Hospital	Niigata

Sponsors (1)

Lead Sponsor	Collaborator
Niigata University Medical & Dental Hospital

Country where clinical trial is conducted

Japan, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change value of AaDO2 between baseline and 24 weeks		24 weeks