Clinical trial NCT02840708: SK-1401 (rhGM-CSF Agent for Inhalation) GM-CSF Inhalation Pharmacokinetic Study
Official title: SK-1401 (rhGM-CSF Agent for Inhalation) GM-CSF Inhalation Pharmacokinetic Study
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Pulmonary Alveolar Proteinosis, Autoimmune
Lead sponsor: Niigata University Medical & Dental Hospital
Sponsor class: OTHER
First posted: 2016-07-21
Enrollment: 14
Sex: ALL
Minimum age (years): 20
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: yes
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: Objective: Evaluate Pharmacokinetics and determine the safety of GM-CSF single dose inhalation.

Study Design: Pharmacokinetic open study


Eligibility criteria: Inclusion Criteria:

1. Subjects judged to be appropriate for the study by the attending physician
2. can provide signed informed consent.

   aPAP patient must meet the following
3. aPAP patient aged over 20 and below 80 years old (as of the date of registration)
4. aPAP severity is mild or moderate. (not severe)

   Healthy volunteer must meet the following
5. Japanese healthy male subject aged over 20 and below 45 years old (as of the date of registration).
6. BMI (Body mass index) is between 18 and 25.

Exclusion Criteria:

1. WBC of 12,000/mcl or more
2. Fever of 38 degree celsius or more
3. History of malignant disease within recent 5 years (not applied to the treated cases of local basal cell carcinoma)
4. Complication of severe cardiovascular diseases including congestive heart failure, angina pectoris, hemorrhagic tendency, etc.
5. 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.
6. History or complication of infectious diseases which require systemic administration of antibiotics, antifungal or antiviral agents within recent 2 weeks.
7. liver dysfunction
8. renal dysfunction
9. Previous experience of severe and unexplained adverse events during aerosol delivery of any kind of medicinal product
10. Positive for HIV-Ab, HBs-Ag, HCV-Ab, STS or TPHA
11. allergic to GM-CSF.
12. addicted to illegal drugs
13. Participation to other clinical trials within 12 weeks before registration.
14. smoking within 5 years
15. cannot follow the procedure defined in this protocol

    aPAP patient must exclude the following
16. Pregnant or possibly pregnant women, lactating women, and women who desire to become pregnant during the study period
17. 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.
18. taking other inhalation.

    Healthy volunteer must exclude the following
19. taking any medicines (incl. OTC).

Primary outcomes:
measure: Serum level of Sargramostim
timeFrame: 0.5,1,2,4,8,12 and 24 hours after inhalation
Secondary outcomes:
Other outcomes:
Locations:
city: Niigata
country: Japan
facility: Niigata University Med & Dental Hospital
Study officials:
References: