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

• ClinicalTrials.gov Identifier: NCT04035135
• Recruitment Status: Completed
• First Posted: July 29, 2019
• Last Update Posted: August 11, 2021
• Sponsor: Annexon, Inc.
• Collaborators: ResearchPoint Global; International Centre for Diarrhoeal Disease Research, Bangladesh
• Information provided by (Responsible Party): Annexon, Inc.

Study Description

Brief Summary:

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

Condition or disease	Intervention/treatment	Phase
Guillain-Barré Syndrome	Drug: ANX005; Drug: Intravenous immunoglobulin	Phase 1

Detailed Description:

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.

Study Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 14 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Intervention Model Description: Open label combination treatment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: 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
• Actual Study Start Date: January 29, 2020
• Actual Primary Completion Date: May 19, 2021
• Actual Study Completion Date: May 19, 2021

Arms and Interventions

Arm	Intervention/treatment
Experimental: Open Label Treatment Arm; 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; investigational drug; Other Name: Anti C1q Antibody; Drug: Intravenous immunoglobulin; investigational drug; Other Name: IVIg

Outcome Measures

Primary Outcome Measures :

1. Safety and tolerability of ANX005 when administered in combination with IVIg: incidence of TEAEs, SAEs, AE's [ Time Frame: 6 months ]
   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.

Secondary Outcome Measures :

1. Pharmacokinetics of ANX005 when administered in combination with IVIg [ Time Frame: 3 months ]
   As measured by ANX005 serum concentrations
2. Pharmacodynamics of ANX005 when administered in combination with IVIg [ Time Frame: 4 months ]
   As measured by CH50 and C1q serum concentrations
3. Values and change from baseline in Guillain-Barré Syndrome Disability Score (GBS-DS) by visit [ Time Frame: 6 months ]
   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

Eligibility Criteria

• Ages Eligible for Study: 18 Years and older (Adult, Older Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Criteria

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

Contacts and Locations

Locations

Bangladesh

National Institute of Neurosciences and Hospital

Dhaka, Bangladesh

Denmark

Aarhus University Hospital

Aarhus, Denmark

Sponsors and Collaborators

Annexon, Inc.

ResearchPoint Global

International Centre for Diarrhoeal Disease Research, Bangladesh

Investigators

• Study Director: Henk-André Kroon, MD, MBA; Annexon, Inc.

More Information

• Responsible Party: Annexon, Inc.
• ClinicalTrials.gov Identifier: NCT04035135
• Other Study ID Numbers: ANX005-GBS-03
• First Posted: July 29, 2019
• Last Update Posted: August 11, 2021
• Last Verified: August 2021

Individual Participant Data (IPD) Sharing Statement:

• Plan to Share IPD: No

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:

Guillain-Barre Syndrome; Syndrome; Disease; Pathologic Processes; Polyradiculoneuropathy; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Demyelinating Diseases; Polyneuropathies; Peripheral Nervous System Diseases; Neuromuscular Diseases; Autoimmune Diseases; Immune System Diseases; Post-Infectious Disorders; Chronic Disease; Disease Attributes; Immunoglobulins; Immunoglobulins, Intravenous; Antibodies; gamma-Globulins; Rho(D) Immune Globulin; Immunologic Factors; Physiological Effects of Drugs