A Study to Evaluate the Efficacy and Safety of Eculizumab in Guillain-Barré Syndrome

Study Overview
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Brief Summary
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This is a Phase 3, prospective, multicenter, placebo controlled, double blind, randomized study to investigate the efficacy and safety of eculizumab in participants with severe GBS, defined using the Hughes Functional Grade (FG) scale as progressively deteriorating FG3 or FG4/FG5 within 2 weeks from onset of weakness due to GBS. This study will be conducted only at sites in Japan.

Detailed Description
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Eligible participants will be randomized to receive intravenous (IV) infusion of eculizumab or placebo at a 2:1 ratio. All participants will be on concomitant IV immunoglobulin G (Ig) therapy as per standard of care.

Official Title
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A Phase 3, Prospective, Multicenter, Double Blind, Randomized, Placebo Controlled Study to Evaluate the Efficacy and Safety of Eculizumab in Patients With Guillain-Barré Syndrome (GBS)

Conditions
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Guillain-Barre Syndrome

Intervention / Treatment
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* Biological: Eculizumab
* Drug: Placebo


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Participants who meet the GBS criteria. Participants who were able to run prior to onset of GBS symptoms. Participants with onset of weakness due to GBS < 2 weeks before screening. Participants unable to walk unaided for ≥ 5 meters (progressively deteriorating FG3 or FG4 to FG5). Participants who are already on IVIg or deemed eligible for and who will start IVIg. Participants who can start their first dose of study drug before the end of the IVIg treatment period. Exclusion Criteria: Participants who have previously received or are currently receiving treatment with complement modulators. Participants who have been administered another investigational product within 30 days or 5 half-lives (whichever is longer) prior to providing consent or are currently participating in another interventional study. Participants who have received rituximab within 12 weeks prior to screening. Participants who are being considered for or are already on plasmapheresis. Participants who have received immunosuppressive treatment during the 4 weeks prior to providing consent.

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Eculizumab<br>Participants will receive eculizumab. | Biological: Eculizumab<br>* Eculizumab will be administered via IV infusion once a week for 4 weeks.<br>* Other names: Soliris;|
| Placebo Comparator: Placebo<br>Participants will receive placebo. | Drug: Placebo<br>* Placebo will be administered via IV infusion once a week for 4 weeks.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Time To First Reaching A Hughes FG Score ≤ 1 |  | Up to Week 24 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Proportion Of Participants With A Hughes FG Score ≤ 1 |  | Week 24 | 
| Proportion Of Participants With A Hughes FG Score Improvement Of ≥ 3 |  | Week 24 | 
| Proportion Of Participants With A Hughes FG Score ≤ 1 |  | Week 8 | 
| Incidence Of Treatment-emergent Adverse Events |  | Up to Week 24 | 
| Free Complement Component 5 In Serum |  | Week 24 | 
| Hemolytic Complement Activity In Serum |  | Week 24 | 
| Length Of Stay In The Hospital |  | Up to Week 24 | 
| Duration Of Ventilator Support |  | Up to Week 24 | 
| Concentration Of Eculizumab In Serum |  | Up to Week 24 | 
| Incidence Of Antidrug Antibodies |  | Up to Week 24 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Eculizumab, Soliris, Alexion, C5 Inhibition Therapy