SK-1401 (rhGM-CSF Agent for Inhalation) GM-CSF Inhalation Pharmacokinetic Study

Study Overview
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Brief Summary
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Objective: Evaluate Pharmacokinetics and determine the safety of GM-CSF single dose inhalation. Study Design: Pharmacokinetic open study

Official Title
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SK-1401 (rhGM-CSF Agent for Inhalation) GM-CSF Inhalation Pharmacokinetic Study

Conditions
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Pulmonary Alveolar Proteinosis, Autoimmune

Intervention / Treatment
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* Drug: Sargramostim


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Subjects judged to be appropriate for the study by the attending physician can provide signed informed consent. aPAP patient must meet the following aPAP patient aged over 20 and below 80 years old (as of the date of registration) aPAP severity is mild or moderate. (not severe) Healthy volunteer must meet the following Japanese healthy male subject aged over 20 and below 45 years old (as of the date of registration). BMI (Body mass index) is between 18 and 25. Exclusion Criteria: WBC of 12,000/mcl or more Fever of 38 degree celsius or more History of malignant disease within recent 5 years (not applied to the treated cases of local basal cell carcinoma) Complication of severe cardiovascular diseases including congestive heart failure, angina pectoris, hemorrhagic tendency, etc. Complication of respiratory diseases such as pulmonary infectious disease(incl. pulmonary tuberculosis), interstitial pneumonitis, lung fibrosis or bronchiectasis, in which the evaluations of safety of GM-CSF therapy are considered to be difficult. History or complication of infectious diseases which require systemic administration of antibiotics, antifungal or antiviral agents within recent 2 weeks. liver dysfunction renal dysfunction Previous experience of severe and unexplained adverse events during aerosol delivery of any kind of medicinal product Positive for HIV-Ab, HBs-Ag, HCV-Ab, STS or TPHA allergic to GM-CSF. addicted to illegal drugs Participation to other clinical trials within 12 weeks before registration. smoking within 5 years cannot follow the procedure defined in this protocol aPAP patient must exclude the following Pregnant or possibly pregnant women, lactating women, and women who desire to become pregnant during the study period Patients who have been treated with whole-lung lavage, repeated segmental-lung lavage, GM-CSF or rituximab within 6 months before the start of the study. taking other inhalation. Healthy volunteer must exclude the following taking any medicines (incl. OTC).

Ages Eligible for Study
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Minimum Age: 20 Years
Maximum Age: 80 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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Accepts Healthy Volunteers

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Active Comparator: 125mcg<br>SK-1401 125mcg single inhalation | Drug: Sargramostim<br> <br> |
| Active Comparator: 250mcg<br>SK-1401 250mcg single inhalation | Drug: Sargramostim<br> <br> |
| Active Comparator: 500mcg<br>SK-1401 500mcg single inhalation | Drug: Sargramostim<br> <br> |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Serum level of Sargramostim |  | 0.5,1,2,4,8,12 and 24 hours after inhalation |