UMIN-CTR Clinical Trial

Basic information
Official scientific title of the study	Exploratory Phase Trial of OCH-NCNP1 in Healthy Subjects and Patients with Multiple Sclerosis
Title of the study (Brief title)	Exploratory Phase Trial of OCH-NCNP1
Region	Japan

Condition
Condition	Multiple Sclerosis
Classification by specialty	Neurology
Classification by malignancy	Others
Genomic information	NO

Objectives
Narrative objectives1	The purpose of this study is to evaluate safety of OCH-NCNP1, to elucidate its pharmacokinetics and to explore biomarkers related to its efficacy.
Basic objectives2	Safety
Basic objectives -Others
Trial characteristics_1	Exploratory
Trial characteristics_2	Explanatory
Developmental phase	Phase I

Assessment
Primary outcomes	Adverse effects
Key secondary outcomes	(1) Exploration of biomarkers related to the efficacy 1) DNA microarray analysis 2) Frequency of NKT, NK and T cell subsets 3) Frequency of Th1/Th2/Th17 cells 4) Frequency of NK1/NK2 cells 5) Cytokine concentrations of the serum and cerebrospinal fluid (2) Pharmacokinetics (3) Expanded Disability Status Scale (EDSS) and Functional Scale (FS) (4) MRI scan 1) Number of new T2-hyperintense lesions 2) Number of new T1-hypointense lesions 3) Number of Gd-enhancing lesions in T1-weighted images 4) FLAIR images

Base
Study type	Interventional

Study design
Basic design	Single arm
Randomization	Non-randomized
Randomization unit
Blinding	Open -no one is blinded
Control	Uncontrolled
Stratification
Dynamic allocation
Institution consideration
Blocking
Concealment

Intervention
No. of arms	1
Purpose of intervention	Treatment
Type of intervention	Medicine
Interventions/Control_1	STEP1: healthy subjects OCH-NCNP1 is orally administered once. The dose is gradually increased from 0.3, 1, 3, 10, 30, 100, up to 300 mg. STEP2: patients with multiple sclerosis OCH-NCNP1 is orally administered once a week for 13 weeks to the 0.3mg dosage group and for 4 weeks to the 1mg or 3mg dosage group. The dose is gradually increased from 0.3, 1, up to 3 mg.
Interventions/Control_2
Interventions/Control_3
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	65 years-old >
Gender	Male and Female
Key inclusion criteria	Inclusion criteria for healthy subjects (1) Provision of written informed consent to participate in this study (2) 20=< Age < 50 (3) Women who promise to prevent conception for at least 90 days after the last administration (4) 18.5 =< BMI < 25 Inclusion criteria for patients with multiple sclerosis (1) Provision of written informed consent to participate in this study. (2) Patients who are diagnosed relapsing remitting multiple sclerosis based on modified McDonald criteria in 2006. (3) 20 =< Age < 65. (4) Expanded Disability Status Scale =< 7. (5) Women who promise to prevent conception for at least 90 days after the last administration (6) Patients who are stable neurologically Other protocol-defined inclusion criteria may apply.
Key exclusion criteria	Exclusion criteria for healthy subjects 1. Women who are pregnant or lactating 2. History of liver diseases 3. Positive for Beta-glucan or positive for QuantiFERON 4. History of HIV infection 5. History of HBV or HCV infection 6. Subjects who were administered with some medicine within two weeks 7. Compromised subjects 8. Inflammatory bowel disease 9. Subjects with prolongation of QT/QTc interval. 10. Smokers Exclusion criteria for patients with relapsing remitting multiple sclerosis 1. Patients who diagnosed as Neuromyelitis Optica 2. Women who are pregnant or lactating 3. Patients who are allergic to Gd-contrast medium 4. History of liver diseases or liver transplantation 5. Liver dysfunction in the screening test and baseline physical examination 6. Beta-glucan-positive or QuantiFERON-positive 7. Positive for Anti-Aquaporin 4 antibody 8. History of HIV infection 9. History of HBV or HCV infection 10. Lymphocyte number < 800 /mm3 in peripheral blood 11. Compromised Patients 12. Inflammatory Bowel disease 13. Subjects with prolongation of QT/QTc interval. 14. Smokers 15. Bronchial Asthma Other protocol-defined exclusion criteria may apply.
Target sample size	30

Research contact person
Name of lead principal investigator	Tomoko Okamoto
Organization	National Center of Neurology and Psychiatry
Division name	Department of Neurology, National Center Hospital
Address	4-1-1, Ogawahigashi, Kodaira, Tokyo 187-8502, Japan
TEL	042-341-2711
Email	okamotot@ncnp.go.jp

Public contact
Name of contact person	Sachiko Tsukamoto
Organization	National Center of Neurology and Psychiatry
Division name	Department of Clinical Research Promotion
Address	4-1-1, Ogawahigashi, Kodaira, Tokyo 187-8502, Japan
TEL	042-341-2711
Homepage URL
Email	chiken13@ncnp.go.jp

Sponsor
Institute	National Center of Neurology and Psychiatry
Institute
Department

Funding Source
Organization	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	2012年9月20日（初回届）

Institutions
Institutions	国立精神･神経医療研究センター（東京都）/National Center of Neurology and Psychiatry（Tokyo）

Other administrative information
Date of disclosure of the study information	2012 Year 11 Month 23 Day

Progress
Recruitment status	Completed
Date of protocol fixation	2012 Year 10 Month 26 Day
Anticipated trial start date	2012 Year 11 Month 27 Day
Last follow-up date	2017 Year 08 Month 14 Day
Date of closure to data entry	2017 Year 08 Month 22 Day
Date trial data considered complete
Date analysis concluded

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

Management information
Registered date	2012 Year 11 Month 22 Day
Last modified on	2017 Year 08 Month 22 Day

Link to view the page
URL(English)	https://upload.umin.ac.jp/cgi-open-bin/icdr_e/ctr_view.cgi?recptno=R000011008