A Clinical Study of ANX005 and IVIG in Subjects With Guillain Barré Syndrome (GBS)

Study Overview
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Brief Summary
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This study is a multi center, open-label, study of ANX005 in combination with IVIg in subjects diagnosed with GBS.

Detailed Description
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The study will enroll approximately 12 subjects into one cohort, with subjects receiving 75 mg/kg of ANX005, a dose shown to be safe and well-tolerated as monotherapy and that provides a meaningful level of exposure to be tested in combination with IVIg. Dosing of IVIg will be administered as is the current standard of care (0.4 g/kg/day x 5 days) and ANX005 will be administered concurrently, beginning on Day 1 or 2 of the treatment period. Subjects will be followed for 6 months after treatment for observation and evaluation.

Official Title
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A Phase 1b Study to Evaluate the Safety, Tolerability and Drug-Drug Interactions of ANX005 and Intravenous Immunoglobulin (IVIg) in Subjects With Guillain Barré Syndrome

Conditions
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Guillain-Barré Syndrome

Intervention / Treatment
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* Drug: ANX005
* Drug: Intravenous immunoglobulin


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 ≤14 days prior to infusion GBS-DS score of 3, 4, or 5 Exclusion Criteria: Clinically significant findings that may interfere with the conduct of the study or the interpretation of the data Be at risk of suicide or self-harm Received previous treatment with plasma exchange for GBS Any diagnosis of a variant of GBS Have a history of anaphylaxis or severe systemic response to immunoglobulin Documented, clinically significant, pre-existing polyneuropathy from another cause Clinically significant intercurrent illness, medical condition, or medical history History of chronic use of steroid or immunosuppressant medication Active alcohol, drug, or substance abuse Females who are pregnant, breast feeding, or unable or unwilling to use highly effective methods of contraception throughout the study

Ages Eligible for Study
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Minimum Age: 18 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
Intervention Model: Single Group Assignment
Interventional Model Description: Open label combination treatment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Open Label Treatment Arm<br>One (1) dose of ANX005, 75 mg/kg, will be administered IV. IVIg, 0.4 g/kg, will be administered for 5 consecutive Days. | Drug: ANX005<br>* investigational drug<br>* Other names: Anti C1q Antibody;Drug: Intravenous immunoglobulin<br>* investigational drug<br>* Other names: IVIg;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Safety and tolerability of ANX005 when administered in combination with IVIg: incidence of TEAEs, SAEs, AE's | As measured by incidence of TEAEs, SAEs, AE's related to ANX005, SAE's related to ANX005, Grade 3 or higher AEs, Grade 3 or higher AEs related to ANX005, AEs leading to study or treatment discontinuation. | 6 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Pharmacokinetics of ANX005 when administered in combination with IVIg | As measured by ANX005 serum concentrations | 3 months | 
| Pharmacodynamics of ANX005 when administered in combination with IVIg | As measured by CH50 and C1q serum concentrations | 4 months | 
| Values and change from baseline in Guillain-Barré Syndrome Disability Score (GBS-DS) by visit | The 6-point Guillain-Barré Syndrme Disability Score (GBS-DS) is a widely accepted and easily obtainable scoring system used to assess functional status of GBS subjects. The score is as follows: 0 = Healthy, 1 = Minor symptoms and capable of running, 2 = Able to walk independently 10 meters or more but unable to run, 3=Able to walk more than 10 meters across an open space with help, 4 = Bedridden or chair bound, 5 = Needing mechanical ventilation, 6 = death | 6 months |