UMIN-CTR Clinical Trial

Public title

A prospective, multicenter, Phase II study to evaluate the safety and efficacy of eculizumab in subjects with Guillain-Barre syndrome

Acronym

Japanese Eculizumab Trial for GBS:JET-GBS

Scientific Title

A prospective, multicenter, Phase II study to evaluate the safety and efficacy of eculizumab in subjects with Guillain-Barre syndrome

Scientific Title:Acronym

Japanese Eculizumab Trial for GBS:JET-GBS

Region

Japan

Condition

Guillan-Barre syndrome

Classification by specialty

Neurology

Classification by malignancy

Others

Genomic information

NO

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

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

Study type

Interventional

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

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

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

Name of lead principal investigator

1st name
Middle name
Last name	Satoshi Kuwabara

Organization

Chiba University Hospital

Division name

Department of Neurology

Zip code

Address

1-8-1 Inohana, Chuo-ku, Chiba-city, Chiba-prefecture

TEL

043-222-7171

Email

kuwabara-s@faculty.chiba-u.jp

Name of contact person

1st name
Middle name
Last name	Sonoko Misawa

Organization

Chiba University Hospital

Division name

Department of Neurology

Zip code

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

Institute

Chiba University, Graduate School of Medicine Department of Neurology

Institute

Department

Personal name

Organization

Japan Agency for Medical Research and Development

Organization

Division

Category of Funding Organization

Nationality of Funding Organization

Co-sponsor

Name of secondary funder(s)

Organization

Address

Tel

Email

Secondary IDs

NO

Study ID_1

Org. issuing International ID_1

Study ID_2

Org. issuing International ID_2

IND to MHLW

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)

Date of disclosure of the study information

2015

Year

07

Month

02

Day

URL releasing protocol

Publication of results

Unpublished

URL related to results and publications

Number of participants that the trial has enrolled

Results

Results date posted

Results Delayed

Results Delay Reason

Date of the first journal publication of results

Baseline Characteristics

Participant flow

Adverse events

Outcome measures

Plan to share IPD

IPD sharing Plan description

Recruitment status

Completed

Date of protocol fixation

2015

Year

06

Month

08

Day

Date of IRB

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

Other related information

Registered date

2015

Year

07

Month

02

Day

Last modified on

2017

Year

05

Month

27

Day

Link to view the page

Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000020978