NCT05701189

Evaluating Efgartigimod in Patients With Guillain-Barré Syndrome

Phase 2, Randomized, Patient- and Rater-blinded Single-site Trial Evaluating Safety and Efficacy of Efgartigimod in Patients With Guillain-Barré Syndrome.

• Status: Withdrawn
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 0 (actual)
• Sponsor: Chafic Karam · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

The goal of this clinical trial is to evaluate the safety and effectiveness of Efgartigimod in patients with Guillain-Barre syndrome (GBS). The main questions it aims to answer are: * Is Efgartigimod a safe treatment option for GBS patients? * Does treatment with Efgartigimod improve patient outcomes? In addition to standard-of-care procedures and assessments, participants will: * Undergo seven blood draws during hospitalization and in four follow-up study visits to evaluate the concentration of neurofilament light chain, a protein that is elevated in patients with Guillain-Barré syndrome. The presence of neurofilament light chain is believed to be indicative of damage to the nervous system, with higher levels resulting from greater damage. * Complete the Columbia Suicide Severity Rating Scale (C-SSRS) to monitor any suicidal ideation or behaviors during the course of the study.

Detailed description

The following procedures/assessments are standard-of-care for Guillain-Barré syndrome. They will not be performed solely for research purposes in this study, but the study team will use select results from them to draw conclusions related to this research: * The study doctor or study staff will ask you about your medical history and any changes to your medications * You will have a physical examination * Your vital signs, including blood pressure, pulse rate, breathing rate, body temperature, and weight will be measured * You will have an electrocardiogram (ECG) * Pulmonary function testing, including forced vital capacity (FVC), maximal inspiratory pressure (MIP), and maximal expiratory pressure (MEP) will be performed * Blood samples will be drawn to evaluate disease progression and safety/efficacy of treatment * You will undergo a lumbar puncture, commonly referred to as a spinal tap, to collect cerebrospinal fluid (CSF) for analysis * Nerve conduction studies (NCS) will be performed * Outcome assessments including the GBS Disability Scale (GBS-DS), MRC Sum Score, and Inflammatory Rasch-built Overall Disability Scale (I-RODS) will be performed

Conditions

• Guillain-Barre Syndrome

Interventions

Type	Name	Description
DRUG	Efgartigimod Alfa-Fcab	Efgartigimod is an anti-neonatal Fc receptor (FcRn) immunoglobulin G1 Fc fragment. The FcRn plans a critical role in extending the half-life of IgGs by rescuing them from lysosomal degradation. Antibodies that bind and subsequently block the FcRn with high affinity result in IgGs being degraded more rapidly instead of salvaged. This approach has been shown to be beneficial in the antibody-mediated disorder myasthenia gravis.
DRUG	Intravenous Immunoglobulin (IVIg)	IVIg is the standard-of-care treatment for GBS. Brand of IVIG used may vary per institutional standards

Timeline

• Start date: 2024-09-10
• Primary completion: 2026-01-19
• Completion: 2026-02-20
• First posted: 2023-01-27
• Last updated: 2026-03-12

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study