Pulmonary Alveolar Proteinosis GM-CSF Inhalation Efficacy Trial in Japan (NCT02835742) | Clinical Trial Compass
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 range
16 Years – 80 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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)
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Change value of AaDO2 between baseline and 24 weeks
Timeframe: 24 weeks
Trial details
NCT IDNCT02835742
SponsorNiigata University Medical & Dental Hospital
. 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.