Efficacy and Safety of Efgartigimod in the Treatment of Guillain-Barré Syndrome(GREAT)

NCT07806916 | Not Yet Recruiting Phase 2

• 1 other identifier: KY2026-050-02
• Study Type: interventional
• Target: 110
• Locations: 0 countries
• Sites: N/A

Brief 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? Participants will: Take drug Efgartigimod or Intravenous Immunoglobulin during hospitalization Undergo six blood draws in six follow-up study visits to evaluate the concentration of IgG Keep a diary of their symptoms and drugs using at the same time

Conditions

Guillain-Barré Syndrome

Keywords

Efgartigimod; Guillain-Barré Syndrome

Outcome Measures

Primary Outcomes (2)

• Guillain-Barre Syndrome Disability Scale (GBS-DS)

  Scoring system that assesses the functional status of GBS subjects. Scores range from 0 to 6, with higher scores indicating a worse outcome.

  week 4

• Number and seriousness of adverse events

  safety monitoring

  Through study completion, an average of 2 years

Secondary Outcomes (17)

• Guillain-Barre Syndrome Disability Scale (GBS-DS)

  week 1，2，3，8，12，24

• Change from baseline in Guillain-Barre Syndrome Disability Scale (GBS-DS)

  week 1,2,3,4,8,12,24

• Number of days to GBS-DS ≤2 within 24 weeks

  24 weeks

• Proportion of patients with GBS-DS ≤2

  week 1, 2, 3, 4, 8, 12, and 24

• Neuropathy Impairment Scale (NIS)

  week 1,4,8,12,24

Other Outcomes (1)

• Study effect of Efgartigimod on IgG levels

  week 1, 2, 3 ,4 ,8 ,12 ,24

Study Arms (2)

Intravenous Immunoglobulin (IVIg)

ACTIVE COMPARATOR

0.4g/kg of IVIg daily for 5 days Interventions: Drug: Intravenous Immunoglobulin (IVIg)

Drug: Intravenous immunoglobulin (IVIG)

Efgartigimod Alfa-Fcab

EXPERIMENTAL

20mg/kg of Intravenous efgartigimod on days 1 and 5 Interventions: Drug: Efgartigimod Alfa-Fcab

Drug: Efgartigimod Alfa-Fcab

Interventions

Intravenous immunoglobulin (IVIG) DRUG

IVIg is the standard-of-care treatment for GBS. The same brand of intravenous immunoglobulin (IVIG) was administered across different study sites

Intravenous Immunoglobulin (IVIg)

Efgartigimod Alfa-Fcab DRUG

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.

Efgartigimod Alfa-Fcab

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Aged 18-70 years
• Diagnosis of sensorimotor or pure motor Guillain-Barré syndrome (GBS) based on the 2023 EAN/PNS guidelines (corresponding to classical GBS in the Chinese Guidelines for the Diagnosis and Treatment of Guillain-Barré Syndrome 2024).
• Onset of GBS within 14 days
• GBS Disability Scale (GBS-DS) score of 3 to 5

You may not qualify if:

• Patients with other diagnoses cannot be excluded
• Patients with recurrent GBS
• Patients with more than 14 days after onset of symptoms
• GBS DS of 2 or less
• Patients unable to cooperate with scale assessments due to psychiatric disorders or impaired consciousness
• Positive serum test at screening for an active viral infection with any of the following conditions: Hepatitis B virus (HBV) that is indicative of an acute or chronic infection; Hepatitis C virus (HCV) based on HCV antibody assay; Positive HIV serology at screening
• Patients with any known severe bacterial, viral or fungal infection or any major episode of infection that required hospitalization or injectable antimicrobial therapy in the last 8 weeks prior to Screening
• Total IgG level ≤ 6 g/L at screening
• Concomitant diseases that can cause peripheral neuropathy, such as metabolic neuropathies (e.g., diabetes, alcoholism), connective tissue disease-associated neuropathies (e.g., rheumatoid arthritis, Sjögren's syndrome, systemic lupus erythematosus), or trauma, which may confound the trial results
• Subjects with serious underlying diseases, such as cardiac dysfunction, coagulopathy dysfunction
• Pregnant and lactating women, and those intending to become pregnant during the trial or within 90 days after the last dosing
• Patients who have a history of malignancy, including malignant thymoma, or myeloproliferative or lymphoproliferative disorders, unless deemed cured by adequate treatment with no evidence of recurrence for ≥ 3 years before Screening. Patients with completely excised non-melanoma skin cancer (such as basal cell carcinoma or squamous cell carcinoma) or cervical carcinoma in situ would be permitted at any time
• Use of investigational drug within 3 months or 5 half-lives of the drug (whichever is longer) prior to Screening
• Use of immunosuppressants, monoclonal antibodies, or other immunomodulatory drugs within 6 months prior to enrollment; use of corticosteroids at a dose exceeding 20 mg within 3 months prior to enrollment; or use of IVIG or plasma exchange (PE) within 1 month prior to enrollmen

Sponsors & Collaborators

• Beijing Tiantan Hospital: lead

MeSH Terms

Conditions

Guillain-Barre Syndrome

Interventions

Immunoglobulins, Intravenous

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

Intervention Hierarchy (Ancestors)

Immunoglobulin G; Immunoglobulin Isotypes; Antibodies; Immunoglobulins; Immunoproteins; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Serum Globulins; Globulins

Central Study Contacts

Zaiqiang Zhang Zhang, MD

CONTACT

86-10-59975570; ttyy0142011@126.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: SINGLE
• Who Masked: OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Chief Physician,Professor

Study Record Dates

• First Submitted: September 2, 2026
• First Posted: September 8, 2026
• Study Start: September 1, 2026
• Primary Completion (Estimated): June 1, 2027
• Study Completion (Estimated): December 1, 2027
• Last Updated: September 18, 2026

Record last verified: 2026-09

Data Sharing

• IPD Sharing: Will share