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

NCT ID: NCT04752566

Last Updated: 2025-09-08

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

57 participants

Study Classification

INTERVENTIONAL

Study Start Date

2021-03-08

Study Completion Date

2022-08-03

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.

Conditions

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Guillain-Barre Syndrome

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

TRIPLE

Participants Caregivers Investigators

Study Groups

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Eculizumab

Participants will receive eculizumab.

Group Type EXPERIMENTAL

Eculizumab

Intervention Type BIOLOGICAL

Eculizumab will be administered via IV infusion once a week for 4 weeks.

Placebo

Participants will receive placebo.

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Placebo will be administered via IV infusion once a week for 4 weeks.

Interventions

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Eculizumab

Eculizumab will be administered via IV infusion once a week for 4 weeks.

Intervention Type BIOLOGICAL

Placebo

Placebo will be administered via IV infusion once a week for 4 weeks.

Intervention Type DRUG

Other Intervention Names

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Soliris

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.
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Alexion Pharmaceuticals, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Research Site

Bunkyō City, , Japan

Site Status

Research Site

Chiba, , Japan

Site Status

Research Site

Fukuoka, , Japan

Site Status

Research Site

Hiroshima, , Japan

Site Status

Research Site

Kagoshima, , Japan

Site Status

Research Site

Kawagoe-shi, , Japan

Site Status

Research Site

Kawasaki-shi, , Japan

Site Status

Research Site

Kitakyushu-shi, , Japan

Site Status

Research Site

Kobe, , Japan

Site Status

Research Site

Kumamoto, , Japan

Site Status

Research Site

Kurashiki-shi, , Japan

Site Status

Research Site

Matsumoto-shi, , Japan

Site Status

Research Site

Mitaka-shi, , Japan

Site Status

Research Site

Nagoya, , Japan

Site Status

Research Site

Niigata, , Japan

Site Status

Research Site

Nishinomiya-shi, , Japan

Site Status

Research Site

Sapporo, , Japan

Site Status

Research Site

Sayama, , Japan

Site Status

Research Site

Sendai, , Japan

Site Status

Research Site

Shimotsuga-gun, , Japan

Site Status

Research Site

Ube-shi, , Japan

Site Status

Research Site

Yokohama, , Japan

Site Status

Countries

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Japan

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

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ECU-GBS-301

Identifier Type: -

Identifier Source: org_study_id

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