Guillan-Barre syndrome
Conditions
Interventions
Intravenous administration of eculizumab 900 mg (3 vials) once a week for a total of 4 times.
Intravenous administration of placebo once a week for a total of 4 times.
Sponsors
Chiba University, Graduate School of Medicine Department of Neurology
Eligibility
Sex/Gender
All
Inclusion criteria
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
Exclusion criteria
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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| [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 | — |
Secondary
| Measure | Time frame |
|---|---|
| 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 | — |
Countries
Japan
Contacts
Public ContactSonoko Misawa
Chiba University Hospital Department of Neurology
Outcome results
None listed