Efficacy and Safety of ANX005 in Subjects With Guillain-Barré Syndrome

Study Overview
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Brief Summary
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This study is intended to evaluate the efficacy and safety of ANX005 administered by intravenous (IV) infusion to participants recently diagnosed with Guillain-Barré Syndrome (GBS). The total duration of study participation is approximately 6 months.

Official Title
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A Randomized, Double-blind, Placebo-controlled Phase 3 Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of ANX005 in Subjects With Guillain-Barré Syndrome

Conditions
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Guillain-Barre Syndrome

Intervention / Treatment
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* Drug: ANX005
* Drug: Placebo


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Diagnosis of GBS according to the National Institute of Neurological Disorders and Stroke Diagnostic Criteria for Guillain-Barré Syndrome. Onset of GBS-related weakness ≤10 days prior to start of infusion. GBS-DS score of 3,4, or 5 at screening and at Day 1 prior to infusion. Exclusion Criteria: Clinically significant findings on the screening electrocardiogram (ECG), laboratory test results, or physical examination that are not specific to GBS that may interfere with the conduct of the study, the interpretation of the data, or increase the participant's risk. Body weight <30 kilograms (kg) or >150 kg at screening. Unresponsive (inexcitable) nerve conduction study results in all nerves tested during screening. Previous or intended treatment with either plasma exchange or intravenous immunoglobulin for GBS. Diagnosis of a variant of GBS, including Miller Fisher syndrome, Bickerstaff's encephalitis, and overlap syndromes. History of prior episode of GBS. GBS-related weakness that improved since symptom onset, including an improvement between screening and Day 1 (baseline).

Ages Eligible for Study
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Minimum Age: 16 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: ANX005 Treatment Group - Dose 1<br>Participants will receive a single IV infusion of ANX005 (Dose 1) on Day 1. | Drug: ANX005<br>* Solution for intravenous infusion<br>|
| Experimental: ANX005 Treatment Group - Dose 2<br>Participants will receive a single IV infusion of ANX005 (Dose 2) on Day 1. | Drug: ANX005<br>* Solution for intravenous infusion<br>|
| Placebo Comparator: Placebo Group<br>Participants will receive a single IV infusion of placebo on Day 1. | Drug: Placebo<br>* Solution for intravenous infusion<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| GBS Disability Score (GBS-DS) at Week 8 |  | Week 8 | 
| Number of Participants with Adverse Events | Number participants recently diagnosed with GBS who experience adverse events. | Through Month 6 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Medical Research Council (MRC) Sum Score at Week 8 |  | Week 8 | 
| MRC Sum Score at Day 8 |  | Day 8 | 
| Duration (Days) of Ventilation Support Over 26 Weeks |  | 26 weeks | 
| GBS Disability Score (GBS-DS) |  | Week 26 | 
| Number of Participants Requiring Intensive Care Unit Stay |  | 26 weeks | 
| Duration (Days) of Intensive Care Unit Stays |  | 26 weeks | 
| Patient Global Impression of Change Scores |  | Week 8 and Week 26 |