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

Alexion Pharmaceuticals

Status and phase: Completed; Phase 3

Conditions: Guillain-Barre Syndrome

Treatments: Drug: Placebo; Biological: Eculizumab

Study type: Interventional

Funder types: Industry

Identifiers

NCT04752566

ECU-GBS-301

Details and patient eligibility

About

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.

Full 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.

Enrollment: 57 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Triple Blind

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57 participants in 2 patient groups, including a placebo group

Eculizumab

Experimental group

Description:

Participants will receive eculizumab.

Treatment:

Biological: Eculizumab

Placebo

Placebo Comparator group

Description:

Participants will receive placebo.

Treatment:

Drug: Placebo