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Completed

NCT Number: NCT04752566

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

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.

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Key information

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Eculizumab

Biological

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

Other names: Soliris

Placebo

Drug

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

Primary outcomes

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

    Time frame: Up to Week 24

    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.

Secondary outcomes

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

    Time frame: Week 8, Week 24

    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.

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

    Time frame: Week 24

    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.

  3. Number of Participants With Treatment-emergent Adverse Events (TEAEs)

    Time frame: Day 1 up to Week 24

    TEAEs were defined as an adverse event (AE) with onset on or after the first dose of the study drug. An AE is any untoward medical occurrence in a participant, temporally associated with the use of study drug, whether or not considered related to the study drug. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section.

  4. Free Complement Component 5 in Serum

    Time frame: Week 24

  5. Hemolytic Complement Activity in Serum

    Time frame: Week 24

  6. Length of Stay in the Hospital

    Time frame: Up to Week 24

    For participants with multiple hospitalizations, the total duration of all hospitalizations was summarized.

  7. Number of Participants Who Required Mechanical Ventilator Support

    Time frame: Up to Week 24

    For participants with more than 1 episode of the same support, the total duration across all episodes was summarized.

  8. Concentration of Eculizumab in Serum

    Time frame: Up to Week 24

  9. Number of Participants With Positive Antidrug Antibodies

    Time frame: Up to Week 12

Sponsors and collaborators

Lead sponsor

Alexion Pharmaceuticals, Inc.

Industry

Registry information

Official study title

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)

Important dates

Study start
2021
Primary completion
2022
Study completion
2022
First posted
Feb 12, 2021
Registry last updated
Sep 8, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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