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)
Overview
- Phase
- Phase 3
- Intervention
- Eculizumab
- Conditions
- Guillain-Barre Syndrome
- Sponsor
- Alexion Pharmaceuticals, Inc.
- Enrollment
- 57
- Locations
- 1
- Primary Endpoint
- Time to First Reaching a Hughes Functional Grade (FG) Score <=1
- Status
- Completed
- Last Updated
- 7 months ago
Overview
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.
Investigators
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.
Arms & Interventions
Eculizumab
Participants will receive eculizumab.
Intervention: Eculizumab
Placebo
Participants will receive placebo.
Intervention: Placebo
Outcomes
Primary Outcomes
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
- Number of Participants With A Hughes Functional Grade (FG) Score <=1(Week 8, Week 24)
- Free Complement Component 5 in Serum(Week 24)
- Number of Participants Who Required Mechanical Ventilator Support(Up to Week 24)
- Number of Participants With A Hughes Functional Grade Score Improvement of >=3(Week 24)
- Hemolytic Complement Activity in Serum(Week 24)
- Length of Stay in the Hospital(Up to Week 24)
- Concentration of Eculizumab in Serum(Up to Week 24)
- Number of Participants With Treatment-emergent Adverse Events (TEAEs)(Day 1 up to Week 24)
- Number of Participants With Positive Antidrug Antibodies(Up to Week 12)