UMIN-CTR Clinical Trial

Basic information
Official scientific title of the study	A prospective, multicenter, Phase II study to evaluate the safety and efficacy of eculizumab in subjects with Guillain-Barre syndrome
Title of the study (Brief title)	Japanese Eculizumab Trial for GBS:JET-GBS
Region	Japan

Condition
Condition	Guillan-Barre syndrome
Classification by specialty	Neurology
Classification by malignancy	Others
Genomic information	NO

Objectives
Narrative objectives1	To characterize the overall efficacy, safety, and tolerability of eculizumab in GBS subjects
Basic objectives2	Safety,Efficacy
Basic objectives -Others
Trial characteristics_1
Trial characteristics_2
Developmental phase	Phase II

Assessment
Primary outcomes	[Safety] Expressed frequency and severity of incidence of AE/SAEs after treatment with eculizumab and IVIg [Efficacy] Proportion of subjects who reach a score of FG2 or lower on FG scale at week 4
Key secondary outcomes	1.Proportion of subjects with improvement of one or more scores on the FG scale at each visit 2.Proportion of subjects who are able to walk unaided (FG2 or lower) at each visit 3.Duration required for improvement by at least one grade on FG scale 4.Proportion of subjects who reach FG 1 or 0 at week 24 5.Change in the FG score between peak disability score and the scores at each visit 6.Proportion of subjects with a clinically relevant improvement in the R-ODS score. An increase in the R-ODS score (0-48) converted to the centile metric score (0-100) by at least six points at each visit 7.Proportion of subjects with a clinically relevant improvement in ONLS. (a decrease in the ONLS score from baseline by at least 1 point) at each visit 8.Proportion of subjects who require ventilatory support (FG 5) and frequency of the incidence. 9.Duration of ventilatory support 10.Occurrence of relapse from the start of the IP administration period until the end of the post IP period 11.Overall survival from the start of the IP administration period until the end of the post IP period (OS) 12.Change in grip strength at each visit 13.Change in results of the manual muscle test (MMT scale) at each visit 14.Change in the rate and results of below measures on the nerve conduction test parameter: distal latency, CMAP amplitude, motor nerve conduction velocity, minimal F wave latency, SNAP amplitude, sensory nerve conduction velocity 15.Change in breathing capacity at each visit 16.Proportion of patients who undergo re-administration of IVIg

Base
Study type	Interventional

Study design
Basic design	Parallel
Randomization	Randomized
Randomization unit	Individual
Blinding	Double blind -all involved are blinded
Control	Placebo
Stratification	NO
Dynamic allocation	YES
Institution consideration	Institution is not considered as adjustment factor.
Blocking	NO
Concealment	Central registration

Intervention
No. of arms	2
Purpose of intervention	Treatment
Type of intervention	Medicine
Interventions/Control_1	Intravenous administration of eculizumab 900 mg (3 vials) once a week for a total of 4 times.
Interventions/Control_2	Intravenous administration of placebo once a week for a total of 4 times.
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	18 years-old <=
Age-upper limit	Not applicable
Gender	Male and Female
Key inclusion criteria	1.Subjects >= 18 years of age at the time of obtaining informed consent 2.Patients with onset of muscular weakness due to GBS less than 2 weeks before the time of consent 3.Patients unable to walk unaided for >=5 meters (progressively deteriorating FG3 or FG 4,5) 4.Patients who are already on IVIg or deemed eligible for and who will start IVIg (Generally, administration of 400mg/kg over 5 days) 5.Patients who can start their first dose of eculizumab within 2 weeks from onset of weakness and before the end of the IVIg treatment period 6.Female subjects of child bearing potential with a negative result in their pregnancy test. All subjects must be able to practice an effective, reliable, medically approved method of contraception during the IP administration period and up to 5 months after IP administration is ended. 7.Patients who can be hospitalized during IP administration period. 8.Patients who have signed the informed consent form
Key exclusion criteria	1.Patients who are being considered for or are already on plasmapheresis. 2.Patients who are pregnant or lactating. 3.Patients showing clear clinical evidence of peripheral polyneuropathy other than GBS, e.g. diabetic (except for mild sensory disturbance) or severe vitamin B1 deficiency related. 4.Patients who have received immunosuppressive treatment (e.g. azathioprine, cyclosporine, tacrolimus, or >20 mg prednisolone daily) during the 4 weeks prior to providing consent. 5.Patients who are known to have severe concurrent disease (such as malignancy with uncontrolled primary tumors or metastatic lesions, severe cardiovascular disease, severe COPD, or TB). 6.Patients who are unable to comply with study procedures and the treatment regimen. 7.Patients who have received rituximab within 24 weeks prior to providing consent. 8.Patients with a history of or unresolved Neisseria meningitides. 9.Patients with active infectious diseases determined to be clinically severe by the principal investigator or sub-investigator that are not being appropriately treated with antibiotics. 10.Patients who that cannot be treated with antibiotic prophylaxis due to allergies. 11.Patients who are allergic to eculizumab. 12.Patients who are known to have or are suspected of having hereditary complement deficiencies. 13.Patients who have been administered another investigational product within 12 weeks prior to providing consent or are currently participating in another trial. 14.Patients with any condition that, in the opinion of the principal investigator or sub-investigator, could increase the patient's risk by participating in the study or confound the outcome of the study. 15.Patients who have a history of Eculizumab treatment for GBS.
Target sample size	33

Research contact person
Name of lead principal investigator	Satoshi Kuwabara
Organization	Chiba University Hospital
Division name	Department of Neurology
Address	1-8-1 Inohana, Chuo-ku, Chiba-city, Chiba-prefecture
TEL	043-222-7171
Email	kuwabara-s@faculty.chiba-u.jp

Public contact
Name of contact person	Sonoko Misawa
Organization	Chiba University Hospital
Division name	Department of Neurology
Address	1-8-1 Inohana, Chuo-ku, Chiba-city, Chiba-prefecture
TEL	043-222-7171
Homepage URL	http://www.chiba-crc.jp/jet-gbs_trial/index.html
Email	sonoko.m@mb.infoweb.ne.jp

Sponsor
Institute	Chiba University, Graduate School of Medicine Department of Neurology
Institute
Department

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

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	北海道大学病院(北海道)、獨協医科大学病院(栃木県)、防衛医科大学校病院(埼玉県)、千葉大学医学部附属病院(千葉県)、慶應義塾大学病院(東京都)、東京大学医学部附属病院(東京都)、東京医科歯科大学医学部附属病院(東京都)、北里大学病院(神奈川県)、名古屋大学医学部附属病院(愛知県)、近畿大学医学部附属病院(大阪府)、神戸市立医療センター中央市民病院(兵庫県)、徳島大学病院(徳島県)、九州大学病院(福岡県)

Other administrative information
Date of disclosure of the study information	2015 Year 07 Month 02 Day

Progress
Recruitment status	Completed
Date of protocol fixation	2015 Year 06 Month 08 Day
Anticipated trial start date	2015 Year 07 Month 13 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	2015 Year 07 Month 02 Day
Last modified on	2017 Year 05 Month 27 Day

Link to view the page
URL(English)	https://upload.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000020978