PAGEiiTrial

Phase 4

Recruiting

• Conditions: Autoimmune pulmonary alveolar proteinosis; pulmonary alveolar proteinosis, GM-CSF, autoimmune disease, anti-GM-CSF autoantibody; C5670459

• Registration Number: JPRN-jRCTs031220127
• Lead Sponsor: akata Koh

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2022-06-10
• Last Update Posted: 17 October 2023

Recruitment & Eligibility

• Status: Recruiting
• Sex: All
• Target Recruitment: 60

Inclusion Criteria

1) Persons aged older than 12 years at the time
of obtaining consent.
2) The name of the disease and the condition of th e disease have been fully explained
by the attendi ng physician in advance.
3) A person who has received sufficient explanatio n in participating in this research,
and who has obtained sufficient understanding and consent to the document at his / her own free will.
4) Baseline at 12 or 24 or 36 or 48 weeks afte r the start of administration
of the investigational drug
5) A case diagnosed with autoimmune alveolar proteinosis, that is, HRCT shows shadows matchin g this disease in both lungs, satisfying the following A or B, and serum anti-GM-CSF autoanti body concentration of 1.7 U / ml or more.
A: Typical pathological findings (intraalveolar rete ntion of PAS-positive protein-like substances) by tr ansbronchial lung biopsy or surgical lung biopsy (t horacic lung biopsy, etc.)
B: Typical findings in bronchoalveolar lavage fluid (white turbidity, increased protein-like substances, increased foamy macrophages)
6) A case with PaO2 under 70mmHg at spine position after breathlng roomair at least 5minute

Exclusion Criteria

1) a. cases diagnosed with secondary alveolar prot
einosis or hereditary alveolar proteinosis.
2). cases with leukocyte count of 12000/mcl or mo re at the time of enrollment.
3). cases with fever or more at the time of enrollme nt
4) cases with marked edema of the extremities
5) Patients with a history of malignant tumor within the past 5 years (excluding patients with stable disease and during the follow-up period)
6) cases with severe symptoms due to cardiovascu lar diseases such as congestive heart failure and a ngina
7) cases with respiratory complications such as bronchial asthma, pulmonary infection (including pulmonary tuberculosis), pulmonary fibrosis, interstitial pneumonia, and bronchiectasis, and its efficacy and safety are expected to be difficult to evaluate.However, patients with non-tuberculous mycobacterial disease, pulmonary aspergillosis, or pulmonary nocardiosis who are being treated or have not been treated and have stable symptoms may be registered at the discretion of the attending physician.
8) cases with a history of infectious diseases or complications that require systemic administration of antibacterial agents, antifungal agents, antiviral agents, etc. for the past 2 weeks.However, systemic administration of antibiotics for nontuberculous mycobacterial disease, pulmonary aspergillosis, and pulmonary nocardiosis is not limited to this.
9) cases receiving other cytokine therapy.
10) pregnant and potentially pregnant women, lact ating women, or women who wish to become preg nant during the exam
11) cases treated with whole lung lavage, repeated segmental lavage, and rituximab within one month of enrollment.
12)Patients with severe liver dysfunction are defined as having at least one of AST (GOT) and ALT (GPT) of 100 U/l or more as a guideline, and the attending physician will make a judgment based on the symptoms and changes in laboratory values.
13) cases with severe renal dysfunction (cases wit h a Ccr of less than 30 according to the Cockcroft & Gault equation).
14)Patients who received oral or intravenous steroids within 2 months of enrollment.
However, oral administration to prevent rejection after transplantation is not limited to this criteria.
15) cases receiving GM-CSF inhalation therapy wit hin one months from the time of enrollment
16) cases with hypersensitivity to Leukine compon
ent
17) other cases that the doctor in charge deems in
appropriate (cases that are considered difficult to
complete treatment, cases that interfere with inhal
er, vial operation, non-cooperative case etc.)

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Difference in alveolar-arterial oxygen pressure <br>difference change between 0-24 and 24-48 <br>weeks in subjects who completed 48 weeks <br>of inhalation therapy.

Secondary Outcome Measures

Name	Time	Method
1.Comparison of changes in alveolar-arterial <br>oxygen pressure difference from baseline to <br>24 weeks after the start of study in <br>groups A and B.<br>2.Comparison of the amount of change from baseline in A-aDO2 at 36 weeks after the start of inhalation.<br>3.lmprovement in clinical symptoms (cough,sputum,dyspnea) during baseline to 24 weeks and 24 to 48 weeks.<br>4.Improvement in arterial oxygen pressure during<br>baseline to 24 weeks and 24 to 48 weeks.<br>5.Improvement in arterial oxygen pessure at <br>spine position under room air at rest during <br>baseline to 24 weeks and 24 to 48 weeks. <br>6.Changes in the Medical Research Council <br>dyspnea scale during baseline to 24 and 24 <br>to 48 weeks.<br>7.Changes in A-aDO2 at each time point from the baseline.