A Study to Evaluate the Efficacy and Safety of Eculizumab in Guillain-Barré Syndrome
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)
1 other identifier
interventional
57
1 country
22
Brief 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 due to GBS. This study will be conducted only at sites in Japan.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_3
Started Mar 2021
22 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
February 9, 2021
CompletedFirst Posted
Study publicly available on registry
February 12, 2021
CompletedStudy Start
First participant enrolled
March 8, 2021
CompletedPrimary Completion
Last participant's last visit for primary outcome
August 3, 2022
CompletedStudy Completion
Last participant's last visit for all outcomes
August 3, 2022
CompletedResults Posted
Study results publicly available
February 9, 2024
CompletedSeptember 8, 2025
September 1, 2025
1.4 years
February 9, 2021
June 6, 2023
September 4, 2025
Conditions
Keywords
Outcome Measures
Primary Outcomes (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.
Up to Week 24
Secondary Outcomes (9)
Number of Participants With A Hughes Functional Grade (FG) Score <=1
Week 8, Week 24
Number of Participants With A Hughes Functional Grade Score Improvement of >=3
Week 24
Number of Participants With Treatment-emergent Adverse Events (TEAEs)
Day 1 up to Week 24
Free Complement Component 5 in Serum
Week 24
Hemolytic Complement Activity in Serum
Week 24
- +4 more secondary outcomes
Study Arms (2)
Eculizumab
EXPERIMENTALParticipants will receive eculizumab.
Placebo
PLACEBO COMPARATORParticipants will receive placebo.
Interventions
Eculizumab will be administered via IV infusion once a week for 4 weeks.
Eligibility Criteria
You may qualify if:
- 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.
You may not qualify if:
- 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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (22)
Research Site
Bunkyō City, 113-8519, Japan
Research Site
Chiba, 260-0877, Japan
Research Site
Fukuoka, 814-0180, Japan
Research Site
Hiroshima, 730-8518, Japan
Research Site
Kagoshima, 890-8520, Japan
Research Site
Kawagoe-shi, 350-8550, Japan
Research Site
Kawasaki-shi, 216-8511, Japan
Research Site
Kitakyushu-shi, 807-8555, Japan
Research Site
Kobe, 650-0047, Japan
Research Site
Kumamoto, 860-8556, Japan
Research Site
Kurashiki-shi, 710-8602, Japan
Research Site
Matsumoto-shi, 390-8621, Japan
Research Site
Mitaka-shi, 181-8611, Japan
Research Site
Nagoya, 466-8560, Japan
Research Site
Niigata, 951-8520, Japan
Research Site
Nishinomiya-shi, 663-8501, Japan
Research Site
Sapporo, 060-8648, Japan
Research Site
Sayama, 589-8511, Japan
Research Site
Sendai, 983-8520, Japan
Research Site
Shimotsuga-gun, 321-0293, Japan
Research Site
Ube-shi, 755-8505, Japan
Research Site
Yokohama, 236-0004, Japan
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Results Point of Contact
- Title
- Alexion Pharmaceuticals Inc.
- Organization
- Alexion Pharmaceuticals Inc.
Publication Agreements
- PI is Sponsor Employee
- No
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- RANDOMIZED
- Masking
- TRIPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
February 9, 2021
First Posted
February 12, 2021
Study Start
March 8, 2021
Primary Completion
August 3, 2022
Study Completion
August 3, 2022
Last Updated
September 8, 2025
Results First Posted
February 9, 2024
Record last verified: 2025-09
Data Sharing
- IPD Sharing
- Will not share