UMIN-CTR Clinical Trial

Basic information
Official scientific title of the study	The safety and efficacy of tocilizumab in patients with neuromyelitis optica: Multicenter Study
Title of the study (Brief title)	Tocilizumab in patients with neuromyelitis optica: Multicenter Study
Region	Japan

Condition
Condition	neuromyelitis optica
Classification by specialty	Neurology
Classification by malignancy	Others
Genomic information	NO

Objectives
Narrative objectives1	The purpose of this study is to evaluate safety and efficacy of anti-IL-6R antibody tocilizumab in intractable neuromyelitis optica and to explore its mechanism of action.
Basic objectives2	Safety,Efficacy
Basic objectives -Others
Trial characteristics_1	Exploratory
Trial characteristics_2	Pragmatic
Developmental phase	Not applicable

Assessment
Primary outcomes	-Adverse events up to 2 years -MRI lesion parameters, relapsing rate, and EDSS up to 2 years
Key secondary outcomes

In outcomes field, the entry of just a few words such as "safety" or "efficiency" will not be accepted. Specify the name of outcome measures, including the time when you plan to measure. Usually, only one primary outcome is accepted. Write the other outcomes in "secondary outcomes" field.

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	Tocilizumab is to be infused every 4 weeks at 8 mg/kg of body weight for 6 years.
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

In interventions field, include the details of interventions, such as duration, amount, and frequency. If the intervention includes prescription or use of medical devices, duration is required.

Eligibility
Age-lower limit	20 years-old <=
Age-upper limit	65 years-old >=
Gender	Male and Female
Key inclusion criteria	Diagnosis of neuromyelitis optica (NMO), as defined by 2006 criteria published in NEUROLOGY vol. 66 or NMO seropositive spectrum disorder (recurrent optic neuritis or longitudinally extensive transverse myelitis). All patients must be anti-aquaporin 4 antibody seropositive. Patients with intractable NMO who are resistant to the standard therapy or experiencing sever adverse reactions of the current therapy. History of at least one relapse during the 1 year prior to initiation of this study. Provision of written informed consent to participate in this study.
Key exclusion criteria	1) Patients with severe infection such as tuberculosis, pneumocystis carinii pneumonia, nontuberculous mycobacterial infection, hepatitis B, hepatitis C, or chronic active EB virus infection. 2) Patients with a history of tuberculosis infection or patients with pleural thickness or old tuberculin in chest X-ray. 3) Patients with a history of hypersensitivity to this drug. 4) Patients with a history of intestinal diverticulum. 5) Patients with interstitial pneumonitis. 6) Patients with leukopenia (white blood cell number < 4000/mm3), lymphopenia (lymphocyte number < 1000/mm3) or seropositivity for beta-D-glucan in peripheral blood. 7) Pregnant women or patients with possible pregnancy. 8) Patients who can not provide consent to participate in this study by themselves. 9) Patients whose EDSS are more than 7.
Target sample size	15

Research contact person
Name of lead principal investigator	Takashi Yamamura
Organization	National Institute of Neuroscience, NCNP
Division name	Department of Immunology
Address	4-1-1, Ogawahigashi, Kodaira, Tokyo 187-8502, Japan
TEL	042-341-2711
Email	yamamura@ncnp.go.jp

Public contact
Name of contact person	Takashi Yamamura
Organization	National Institute of Neuroscience, NCNP
Division name	Department of Immunology
Address	4-1-1, Ogawahigashi, Kodaira, Tokyo 187-8502, Japan
TEL	042-341-2711
Homepage URL
Email	yamamura@ncnp.go.jp

Sponsor
Institute	Department of Immunology, National Institute of Neuroscience, NCNP
Institute
Department

Sponsor means an organization that is responsible for plan, deployment and report of the research including funding management. It doesn't mean funding agency". Therefore, all clinical trial should have the one.

Funding Source
Organization	Ministry of Health, Labour and Welfare
Organization
Division
Category of Funding Organization	Japanese Governmental office
Nationality of Funding Organization	Japan

Other related organizations
Co-sponsor	Department of Neurology, Kinki University Faculty of Mediceine
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

Other administrative information
Date of disclosure of the study information	2012 Year 05 Month 01 Day

Progress
Recruitment status	No longer recruiting
Date of protocol fixation	2011 Year 09 Month 30 Day
Anticipated trial start date	2012 Year 05 Month 01 Day
Last follow-up date
Date of closure to data entry
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 04 Month 30 Day
Last modified on	2016 Year 06 Month 30 Day

Link to view the page
URL(English)	https://upload.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000009239