PAGEiiTrial

JRCT ID: jRCTs031220127

Registered date:10/06/2022

Basic Information

• Recruitment status: Recruiting
• Health condition(s) or Problem(s) studied: Autoimmune pulmonary alveolar proteinosis
• Date of first enrollment: 17/08/2022
• Target sample size: 60
• Study type: Interventional

Intervention(s)

Dissolve the vial in 4 mL of saline, 2 mL of which is set in the designated nebulizer an d inhaled. Inhalation shall be continued until the s et inhalation liquid is exhausted. Inhale twice a da y (morning and evening), inhale for 7 days, and th en take a 7 day rest period for 2 weeks as one cou rse, repeat 12 courses, and continue for a total of 24 weeks.
For additional inhalation administration, dissolve 500 mcg (2 vial) once a day in 4 ml of physiologic al saline, put it in a designated nebulizer, inhale f or 7 days before going to bed, and then take 2 we eks of drug holiday for 7 days as one course, 12 c ourses repeated, continuing for a total of 24 weeks.

Outcome(s)

Primary Outcome

Difference in alveolar-arterial oxygen pressure
difference change between 0-24 and 24-48
weeks in subjects who completed 48 weeks
of inhalation therapy.

Secondary Outcome

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

Key inclusion & exclusion criteria

• Age minimum: >= 12age old
• Age maximum: Not applicable
• Gender: Both

Include 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

Exclude 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.)

Related Information

• Primary Sponsor: Nakata Koh
• Source(s) of Monetary Support: Nobelphanma Co.,Ltd.