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

Alexion Pharmaceuticals, Inc.57 enrolled

Overview

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.

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.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

TRIPLE

Enrollment

Conditions

Guillain-Barre Syndrome

Interventions

EculizumabBIOLOGICAL

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

PlaceboDRUG

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

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

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.

Locations (22)

Research Site

Bunkyō City, Japan

Research Site

Chiba, Japan

Outcomes

Primary Outcomes

Time to First Reaching a Hughes Functional Grade (FG) Score <=1

The mobility of the participants was evaluated on a 7 point disability functional grade scale and described as Hughes FG score of 0 (Healthy, no signs or symptoms of Guillain-Barré syndrome); 1 (Minor signs or symptoms and able to run); 2 (Able to walk 5 metre (m) across an open space without assistance); 3 (Able to walk 5 m across an open space with the help of one person and waist-level walking-frame, stick, or sticks); 4 (Chairbound/bedbound: unable to walk as in 3); 5 (Requiring assisted ventilation \[for at least part of day or night\]) and 6 (Dead), where higher numbers indicate more severe impairment. The Kaplan-Meier estimate of time to event of FG\<=1 is reported. Time (days) to first event=Date of first event-Date of first dose+1. Participants who discontinued early without achieving FG \<= 1 were censored at the date of discontinuation. Participants who completed the study without achieving FG\<=1 were censored at the date of study completion.

Time frame: Up to Week 24

Secondary Outcomes

Number of Participants With A Hughes Functional Grade (FG) Score <=1

The mobility of the participants was evaluated on a 7 point disability FG scale and described as Hughes FG score of 0 (Healthy, no signs or symptoms of Guillain-Barré syndrome); 1 (Minor signs or symptoms and able to run); 2 (Able to walk 5 m across an open space without assistance); 3 (Able to walk 5 m across an open space with the help of one person and waist-level walking-frame, stick, or sticks); 4 (Chairbound/bedbound: unable to walk as in 3); 5 (Requiring assisted ventilation \[for at least part of day or night\]) and 6 (Dead), where higher numbers indicate more severe impairment. If a participant had an FG score \<= 1 prior to or at Week 8 and Week 24, respectively, then the participant is considered a responder. Otherwise, participants discontinued prior to Week 8 and Week 24 or with an FG score \> 1 at Week 8 and Week 24 are nonresponders, respectively.

Time frame: Week 8, Week 24

Number of Participants With A Hughes Functional Grade Score Improvement of >=3

The mobility of the participants was evaluated on a 7 point disability FG scale and described as Hughes FG score of 0 (Healthy, no signs or symptoms of Guillain-Barré syndrome); 1 (Minor signs or symptoms and able to run); 2 (Able to walk 5 m across an open space without assistance); 3 (Able to walk 5 m across an open space with the help of one person and waist-level walking-frame, stick, or sticks); 4 (Chairbound/bedbound: unable to walk as in 3); 5 (Requiring assisted ventilation \[for at least part of day or night\]) and 6 (Dead), where higher numbers indicate more severe impairment. Participants with a change from baseline in FG score (value at Week 24 - baseline value) \<= -3 were considered a responder. Participants with change from baseline \> -3 and participants who discontinued prior to Week 24 were considered non-responders.

Data from ClinicalTrials.gov

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