UMIN-CTR Clinical Trial

Basic information
Official scientific title of the study	SK-1401 (rhGM-CSF agent for inhalation) GM-CSF Inhalation Pharmacokinetic study (NTU Trial)
Title of the study (Brief title)	SK-1401 (rhGM-CSF agent for inhalation) GM-CSF Inhalation Pharmacokinetic study (NTU Trial)
Region	Japan

Condition
Condition	autoimmune pulmonary alveolar proteinosis
Classification by specialty	Pneumology
Classification by malignancy	Others
Genomic information	NO

Objectives
Narrative objectives1	Evaluate Pharmacokinetics and determine the safety of GM-CSF single dose inhalation.
Basic objectives2	Pharmacokinetics
Basic objectives -Others
Trial characteristics_1	Others
Trial characteristics_2
Developmental phase	Phase I

Assessment
Primary outcomes	Evaluate PK (0.5, 1, 2, 4, 8, 12 and 24 hours after inhalation).
Key secondary outcomes	Safety

Base
Study type	Interventional

Study design
Basic design	Parallel
Randomization	Non-randomized
Randomization unit
Blinding	Open -no one is blinded
Control	Dose comparison
Stratification	NO
Dynamic allocation	NO
Institution consideration	Institution is not considered as adjustment factor.
Blocking	NO
Concealment	No need to know

Intervention
No. of arms	3
Purpose of intervention	Treatment
Type of intervention	Medicine
Interventions/Control_1	125ug Single dose
Interventions/Control_2	250ug Single dose
Interventions/Control_3	500ug Single dose
Interventions/Control_4
Interventions/Control_5
Interventions/Control_6
Interventions/Control_7
Interventions/Control_8
Interventions/Control_9
Interventions/Control_10

Eligibility
Age-lower limit	20 years-old <=
Age-upper limit	80 years-old >
Gender	Male and Female
Key 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.
Key exclusion criteria	1. WBC of 12000/ul 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 (CTCAE Grade 1 or higher) 8. renal dysfunction (CTCAE Grade 1 or higher) 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 prescribed medicines.
Target sample size	14

Research contact person
Name of lead principal investigator	Koh Nakata
Organization	Niigata University Medical and Dental Hospital
Division name	Bioscience Medical Reseach Center
Address	1-754, Asahimachi dori, Chuo-ku, Niigata
TEL	025-227-0847
Email	radical@med.niigata-u.ac.jp

Public contact
Name of contact person	Takahiro Ueda
Organization	Niigata University Medical and Dental Hospital
Division name	Bioscience Medical Reseach Center
Address	1-754, Asahimachi dori, Chuo-ku, Niigata
TEL	025-227-2331
Homepage URL
Email	page-office@umin.ac.jp

Sponsor
Institute	Niigata University Medical and Dental Hospital Bioscience Medical Reseach Center
Institute
Department

Funding Source
Organization	Japan Agency for Medical Research and Development
Organization
Division
Category of Funding Organization	Japanese Governmental office
Nationality of Funding Organization	Japan

Other related organizations
Co-sponsor
Name of secondary funｄer(s)

Secondary IDs
Secondary IDs	NO
Study ID_1
Org. issuing International ID_1
Study ID_2
Org. issuing International ID_2
IND to MHLW

Institutions
Institutions	新潟大学医歯学総合病院（新潟県) 東京大学医学部附属病院（東京都）

Other administrative information
Date of disclosure of the study information	2016 Year 06 Month 01 Day

Progress
Recruitment status	Completed
Date of protocol fixation	2016 Year 03 Month 11 Day
Anticipated trial start date	2016 Year 06 Month 20 Day
Last follow-up date	2016 Year 09 Month 20 Day
Date of closure to data entry	2016 Year 10 Month 30 Day
Date trial data considered complete	2016 Year 11 Month 30 Day
Date analysis concluded	2017 Year 03 Month 30 Day

Related information
URL releasing protocol
Publication of results	Unpublished
URL releasing results
Results
Other related information

Management information
Registered date	2016 Year 04 Month 28 Day
Last modified on	2017 Year 10 Month 18 Day

Link to view the page
URL(English)	https://upload.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000025497