CompletedPhase 2

Pulmonary Alveolar Proteinosis GM-CSF Inhalation Efficacy Trial in Japan

Japan78 participantsStarted 2016-09-01

Plain-language 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.

Who can participate

Age range16 Years – 80 Years

SexALL

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Inclusion criteria

✓. Age over 16 years and below 80 years (as of the date of registration).
✓. Can provide signed informed consent.
✓. 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).
✓. Autoimmune pulmonary alveolar proteinosis diagnosed by both HR-CT and biopsy and/or BAL as well as GM-CSF antibodies in serum positive.
✓. 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

✕. Diagnosed as secondary or hereditary pulmonary alveolar proteinosis
✕. WBC of 12,000/mm3 or more
✕. Fever of 38 degree celsius or more
✕. Severe edema
✕. History of malignant disease within recent 5 years (not applied to the treated cases of uterine carcinoma in situ and local basal cell carcinoma)
✕. Complication of cardiovascular diseases including congestive heart failure, angina pectoris, hemorrhagic tendency, etc with severe condition.
✕. 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.
✕. History or complication of infectious diseases which require systemic administration of antibiotics, antifungal or antiviral agents within recent 2 weeks.

What they're measuring

Change value of AaDO2 between baseline and 24 weeks

Timeframe: 24 weeks

Trial details

NCT IDNCT02835742

SponsorNiigata University Medical & Dental Hospital

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2017-08

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