Back to resultsA Study to Evaluate the Efficacy and Safety of Eculizumab in Guillain-Barré Syndrome
Primary Purpose
Guillain-Barre Syndrome
Status
Completed
Phase
Phase 3
Locations
Japan
Study Type
Interventional
Intervention
Eculizumab
Placebo
Sponsored by
About this trial
This is an interventional treatment trial for Guillain-Barre Syndrome focused on measuring Eculizumab, Soliris, Alexion, C5 Inhibition Therapy
Eligibility Criteria
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.
Sites / Locations
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Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Placebo Comparator
Arm Label
Eculizumab
Placebo
Arm Description
Participants will receive eculizumab.
Participants will receive placebo.
Outcomes
Primary Outcome Measures
Time To First Reaching A Hughes FG Score ≤ 1
Secondary Outcome Measures
Proportion Of Participants With A Hughes FG Score ≤ 1
Proportion Of Participants With A Hughes FG Score Improvement Of ≥ 3
Proportion Of Participants With A Hughes FG Score ≤ 1
Incidence Of Treatment-emergent Adverse Events
Free Complement Component 5 In Serum
Hemolytic Complement Activity In Serum
Length Of Stay In The Hospital
Duration Of Ventilator Support
Concentration Of Eculizumab In Serum
Incidence Of Antidrug Antibodies
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT04752566
Brief Title
A Study to Evaluate the Efficacy and Safety of Eculizumab in Guillain-Barré Syndrome
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)
Study Type
Interventional
2. Study Status
Record Verification Date
August 2022
Overall Recruitment Status
Completed
Study Start Date
March 8, 2021 (Actual)
Primary Completion Date
August 3, 2022 (Actual)
Study Completion Date
August 3, 2022 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Alexion
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
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.
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Guillain-Barre Syndrome
Keywords
Eculizumab, Soliris, Alexion, C5 Inhibition Therapy
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigator
Allocation
Randomized
Enrollment
57 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Eculizumab
Arm Type
Experimental
Arm Description
Participants will receive eculizumab.
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
Participants will receive placebo.
Intervention Type
Biological
Intervention Name(s)
Eculizumab
Other Intervention Name(s)
Soliris
Intervention Description
Eculizumab will be administered via IV infusion once a week for 4 weeks.
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Placebo will be administered via IV infusion once a week for 4 weeks.
Primary Outcome Measure Information:
Title
Time To First Reaching A Hughes FG Score ≤ 1
Time Frame
Up to Week 24
Secondary Outcome Measure Information:
Title
Proportion Of Participants With A Hughes FG Score ≤ 1
Time Frame
Week 24
Title
Proportion Of Participants With A Hughes FG Score Improvement Of ≥ 3
Time Frame
Week 24
Title
Proportion Of Participants With A Hughes FG Score ≤ 1
Time Frame
Week 8
Title
Incidence Of Treatment-emergent Adverse Events
Time Frame
Up to Week 24
Title
Free Complement Component 5 In Serum
Time Frame
Week 24
Title
Hemolytic Complement Activity In Serum
Time Frame
Week 24
Title
Length Of Stay In The Hospital
Time Frame
Up to Week 24
Title
Duration Of Ventilator Support
Time Frame
Up to Week 24
Title
Concentration Of Eculizumab In Serum
Time Frame
Up to Week 24
Title
Incidence Of Antidrug Antibodies
Time Frame
Up to Week 24
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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.
Facility Information:
Facility Name
Clinical Trial Site
City
Bunkyo-ku
ZIP/Postal Code
113-8519
Country
Japan
Facility Name
Clinical Trial Site
City
Chiba
ZIP/Postal Code
260-8677
Country
Japan
Facility Name
Clinical Trial Site
City
Fuchu
ZIP/Postal Code
183-0042
Country
Japan
Facility Name
Clinical Trial Site
City
Fukuoka
ZIP/Postal Code
814-0180
Country
Japan
Facility Name
Clinical Trial Site
City
Gifu
ZIP/Postal Code
501-1194
Country
Japan
Facility Name
Clinical Trial Site
City
Hiroshima
ZIP/Postal Code
730-8518
Country
Japan
Facility Name
Clinical Trial Site
City
Kagoshima
ZIP/Postal Code
890-8520
Country
Japan
Facility Name
Clinical Trial Site
City
Kawagoe
ZIP/Postal Code
350-8550
Country
Japan
Facility Name
Clinical Trial Site
City
Kawasaki
ZIP/Postal Code
216-8511
Country
Japan
Facility Name
Clinical Trial Site
City
Kitakyushu
ZIP/Postal Code
807-8556
Country
Japan
Facility Name
Clinical Trial Site
City
Kobe
ZIP/Postal Code
650-0047
Country
Japan
Facility Name
Clinical Trial Site
City
Kumamoto
ZIP/Postal Code
860-8556
Country
Japan
Facility Name
Clinical Trial Site
City
Kurashiki
ZIP/Postal Code
710-8602
Country
Japan
Facility Name
Clinical Trial Site
City
Kyoto
ZIP/Postal Code
602-8566
Country
Japan
Facility Name
Clinical Trial Site
City
Matsumoto
ZIP/Postal Code
390-8621
Country
Japan
Facility Name
Clinical Trial Site
City
Mibu
ZIP/Postal Code
321-0293
Country
Japan
Facility Name
Clinical Trial Site
City
Mitaka
ZIP/Postal Code
181-8611
Country
Japan
Facility Name
Clinical Trial Site
City
Nagoya
ZIP/Postal Code
466-8560
Country
Japan
Facility Name
Clinical Trial Site
City
Niigata
ZIP/Postal Code
951-8520
Country
Japan
Facility Name
Clinical Trial Site
City
Nishinomiya
ZIP/Postal Code
663-8501
Country
Japan
Facility Name
Clinical Trial Site
City
Osakasayama
ZIP/Postal Code
589-8511
Country
Japan
Facility Name
Clinical Trial Site
City
Sagamihara
ZIP/Postal Code
252-0375
Country
Japan
Facility Name
Clinical Trial Site
City
Sapporo
ZIP/Postal Code
060-8648
Country
Japan
Facility Name
Clinical Trial Site
City
Sendai
ZIP/Postal Code
983-8520
Country
Japan
Facility Name
Clinical Trial Site
City
Ube
ZIP/Postal Code
755-8505
Country
Japan
Facility Name
Clinical Trial Site
City
Yokohama
ZIP/Postal Code
236-0004
Country
Japan
12. IPD Sharing Statement
Plan to Share IPD
No
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A Study to Evaluate the Efficacy and Safety of Eculizumab in Guillain-Barré Syndrome
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