Pulmonary Alveolar Proteinosis GM-CSF Inhalation Efficacy Trial in Japan

NCT02835742•Niigata University Medical & Dental Hospital

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.

Eligibility

Age

16 - 80 years

Sex

ALL

Healthy Volunteers

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
+9 more criteria

Locations (1)

Niigata University Med & Dental Hospital

Niigata, Japan

Key Dates

Start Date

September 1, 2016

Primary Completion Date

August 1, 2017

Completion Date

June 1, 2018

Last Updated

February 7, 2019

Enrollment

ACTUAL participants

Conditions

Pulmonary Alveolar Proteinosis, Autoimmune

Interventions

Sargramostim

DRUG

Placebo

DRUG