Phase 3
N=57
A Study to Evaluate the Efficacy and Safety of Eculizumab in Guillain-Barré Syndrome
Guillain-Barre Syndrome
Bottom Line
View on ClinicalTrials.gov: NCT04752566 ↗Enrolled (actual)
57
Serious AEs
8.8%
Results posted
Feb 2024
Primary outcome: Primary: Time to First Reaching a Hughes Functional Grade (FG) Score <=1 — 112.0; 168.0 Days — p=0.8938
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Eculizumab (Biological); Placebo (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Alexion Pharmaceuticals, Inc.
- Primary completion
- Aug 2022
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Time to First Reaching a Hughes Functional Grade (FG) Score <=1 |
112.0; 168.0 | 0.8938 |
| SECONDARY Number of Participants With A Hughes Functional Grade (FG) Score <=1 |
12; 7; 20; 13 | 0.8987 |
| SECONDARY Number of Participants With A Hughes Functional Grade Score Improvement of >=3 |
16; 10 | 0.6739 |
| SECONDARY Number of Participants With Treatment-emergent Adverse Events (TEAEs) |
34; 19 | — |
| SECONDARY Free Complement Component 5 in Serum |
131.0438; 147.8833 | — |
| SECONDARY Hemolytic Complement Activity in Serum |
91.79; 91.83 | — |
| SECONDARY Length of Stay in the Hospital |
49.5; 39.8 | — |
| SECONDARY Number of Participants Who Required Mechanical Ventilator Support |
8; 1 | — |
| SECONDARY Concentration of Eculizumab in Serum |
4.690 | — |
| SECONDARY Number of Participants With Positive Antidrug Antibodies |
0; 0 | — |
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.
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.
Data sourced from ClinicalTrials.gov (NCT04752566). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.