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

NCT04701164 | Completed Phase 3 DMC FDA Drug

• 1 other identifier: ANX005-GBS-02
• Study Type: interventional
• Target: 242
• Locations: 2 countries
• Sites: 11

Brief Summary

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.

Conditions

Guillain-Barre Syndrome

Outcome Measures

Primary Outcomes (2)

• 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 Outcomes (7)

• 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

Study Arms (3)

ANX005 Treatment Group - Dose 1

EXPERIMENTAL

Participants will receive a single IV infusion of ANX005 (Dose 1) on Day 1.

Drug: ANX005

ANX005 Treatment Group - Dose 2

EXPERIMENTAL

Participants will receive a single IV infusion of ANX005 (Dose 2) on Day 1.

Drug: ANX005

Placebo Group

PLACEBO COMPARATOR

Participants will receive a single IV infusion of placebo on Day 1.

Drug: Placebo

Interventions

ANX005 DRUG

Solution for intravenous infusion

ANX005 Treatment Group - Dose 1; ANX005 Treatment Group - Dose 2

Placebo DRUG

Solution for intravenous infusion

Placebo Group

Eligibility Criteria

• Age: 16 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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).

Sponsors & Collaborators

• Annexon, Inc.: lead

Study Sites (11)

Dhaka Medical Center

Dhaka, Bangladesh

Location

National Institute of Neurosciences and Hospital (NINS)

Dhaka, Bangladesh

Location

Baguio General Hospital Medical Center

Baguio City, Philippines

Location

Batangas Medical Center

Batangas, Philippines

Location

Mary Mediatrix Medical Center

Batangas, Philippines

Location

Perpetual Succour Hospital

Cebu City, Philippines

Location

Cotabato Regional Medical Center

Cotabato City, Philippines

Location

Southern Philippines Medical Center

Davao City, Philippines

Location

Jose R. Reyes Memorial Medical Center

Manila, Philippines

Location

West Visayas State University Medical Center

Manila, Philippines

Location

Zamboanga City Medical Center

Zamboanga City, Philippines

Location

MeSH Terms

Conditions

Guillain-Barre Syndrome

Condition Hierarchy (Ancestors)

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; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Officials

• Clinical Trials

  Annexon, Inc.

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 6, 2021
• First Posted: January 8, 2021
• Study Start: December 17, 2020
• Primary Completion: April 20, 2024
• Study Completion: April 20, 2024
• Last Updated: May 29, 2025

Record last verified: 2025-05

Data Sharing

• IPD Sharing: Will not share

Locations

BA (2); PH (9)