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Guillain-Barre SyndromePhase 3NCT04752566

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

A Phase 3 study testing Eculizumab in people with Guillain-Barre Syndrome, run by Alexion Pharmaceuticals, Inc. at 22 sites in Japan.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

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… Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Guillain-Barre Syndrome
What is being tested?
Eculizumab
Type of treatment
Injection or infusion
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
57 participants
Where
22 sitesJapan
Study dates
8 Mar 2021to 3 Aug 2022
Phase
Phase 3
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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.

Who cannot join

  • 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.

Trial locations and contacts

22 sites in 1 country.

22 sites in 1 country

Japan22 sites

  • Research Site

    Bunkyō City, 113-8519

  • Research Site

    Chiba, 260-0877

  • Research Site

    Fukuoka, 814-0180

  • Research Site

    Hiroshima, 730-8518

  • Research Site

    Kagoshima, 890-8520

  • Research Site

    Kawagoe-shi, 350-8550

  • Research Site

    Kawasaki-shi, 216-8511

  • Research Site

    Kitakyushu-shi, 807-8555

  • Research Site

    Kobe, 650-0047

  • Research Site

    Kumamoto, 860-8556

  • Research Site

    Kurashiki-shi, 710-8602

  • Research Site

    Matsumoto-shi, 390-8621

  • Research Site

    Mitaka-shi, 181-8611

  • Research Site

    Nagoya, 466-8560

  • Research Site

    Niigata, 951-8520

  • Research Site

    Nishinomiya-shi, 663-8501

  • Research Site

    Sapporo, 060-8648

  • Research Site

    Sayama, 589-8511

  • Research Site

    Sendai, 983-8520

  • Research Site

    Shimotsuga-gun, 321-0293

  • Research Site

    Ube-shi, 755-8505

  • Research Site

    Yokohama, 236-0004

Other trials in Japan

About this study

Official 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)

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.

Read the detailed description

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.

Being tested
Eculizumab, Placebo
Keywords
Eculizumab, Soliris, Alexion, C5 Inhibition Therapy
Medical subject headings
Guillain-Barre Syndrome

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • EculizumabBiological

    Also called Soliris

    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.

Study groups in NCT04752566
Study groupRoleWhat they receive
EculizumabExperimentalParticipants will receive eculizumab.
Receives: Biological: Eculizumab
PlaceboPlacebo comparatorParticipants will receive placebo.
Receives: Drug: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. 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.

    Measured: Up to Week 24

Secondary outcomes (9)

  1. 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.

    Measured: Week 8, Week 24

  2. 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.

    Measured: Week 24

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

    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.

    Measured: Day 1 up to Week 24

  4. Free Complement Component 5 in Serum

    Measured: Week 24

  5. Hemolytic Complement Activity in Serum

    Measured: Week 24

  6. Length of Stay in the Hospital

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

    Measured: Up to Week 24

  7. Number of Participants Who Required Mechanical Ventilator Support

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

    Measured: Up to Week 24

  8. Concentration of Eculizumab in Serum

    Measured: Up to Week 24

  9. Number of Participants With Positive Antidrug Antibodies

    Measured: Up to Week 12

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted12 Feb 2021
  2. Study start8 Mar 2021
  3. Primary completion3 Aug 2022
  4. Study completion3 Aug 2022

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Triple blind
Who is masked
Participant, Care provider, Investigator
Enrolment
57 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04752566
Sponsor's protocol ID
ECU-GBS-301
Status last verified
Sep 2025
First submitted
9 Feb 2021
Last updated
8 Sep 2025
Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Publications

  • Kuwabara S, Kusunoki S, Kuwahara M, Yamano Y, Nishida Y, Ishida H, Kasuya T, Kupperman E, Lin Q, Frick G, Misawa S. Efficacy and safety of eculizumab in Guillain-Barre syndrome: A phase 3, multicenter, double-blind, randomized, placebo-controlled clinical trial. J Peripher Nerv Syst. 2024 Sep;29(3):339-349. doi: 10.1111/jns.12646. Epub 2024 Jul 10.

    PubMed 38987228

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Eculizumab in people with Guillain-Barre Syndrome, run by Alexion Pharmaceuticals, Inc. at 22 sites in Japan.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 6 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 8 Sep 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 22 sites in Japan. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 9 Feb 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Time to First Reaching a Hughes Functional Grade (FG) Score <=1 (time frame: Up to Week 24). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04752566, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Sep 2025.

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