Efgartigimod for the Treatment of Guillain-Barré Syndrome

Safety and Efficacy of Efgartigimod in Guillain-Barré Syndrome

The goal of this clinical trial is to evaluating the efficacy and safety of efgartigimod in the treatment of Guillain-Barré Syndrome and exploring the immunological mechanisms of efgartigimod therapy in Guillain-Barré Syndrome. The main questions it aims to answer are:

Will efgartigimod improve the symptoms of participants? What medical problems do participants have when using efgartigimod?

Participants will:

On day 1 and day 5 of the treatment period, drug A was administered intravenously.

Keep a diary of their symptoms

Study Overview

• Status: Not yet recruiting
• Conditions: Guillain-Barre Syndrome (GBS)
• Intervention / Treatment: Drug: efgartigimod

• Study Type: Interventional
• Enrollment (Estimated): 20
• Phase: Phase 2; Phase 3

Contacts and Locations

• Study Contact: Name: Zhaoyou Meng; Phone Number: 0086-023-68774449; Email: mengzhaoyou@tmmu.edu.cn

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Aged 18 years or older;
2. Meet the diagnostic criteria as specified in the *European Guidelines for the Diagnosis and Treatment of Guillain-Barré Syndrome 2023 Edition*;
3. Onset of GBS symptoms within 2 weeks prior to enrollment;
4. GBS-DS score of 3-5;
5. Written informed consent obtained.

Exclusion Criteria:

1. Patients with GBS symptoms for more than 2 weeks;
2. Known hypersensitivity of the study subject to any component of the investigational drug or any other anti-neonatal Fc receptor (FcRn) agent;
3. Any uncontrolled active infection or severe infection within 8 weeks prior to screening;
4. Patients with other autoimmune diseases, such as Sjögren's syndrome, systemic lupus erythematosus, neuromyelitis optica, myasthenia gravis, multiple sclerosis, etc., requiring treatment with immunosuppressive agents;
5. Vaccination within 4 weeks prior to screening or planned vaccination during the study period;
6. Pregnant or breastfeeding, or planning to become pregnant during the study period, or women of childbearing potential not using effective contraception;
7. Currently participating in another clinical trial of similar investigational agents (FcRn antagonists);
8. Use of the investigational drug within 3 months prior to screening or within five half-lives of the drug (whichever is longer);
9. Patients with a history of malignancy, including malignant thymoma, myeloproliferative or lymphoproliferative disorders, unless considered cured by adequate treatment and without evidence of recurrence for ≥3 years prior to screening. Patients with completely resected non-melanoma skin cancer (e.g., basal cell carcinoma or squamous cell carcinoma) or carcinoma in situ of the cervix are eligible at any time;
10. Patients with clinical evidence of other significant severe diseases or who have recently undergone major surgery, which may confound study results or place the patient at undue risk. Patients with severe renal/hepatic impairment may be included.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Treatment group	Drug: efgartigimod; efgartigimod 20 mg/kg was administered via intravenous infusion on day 1 and day 5 of the treatment period only.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Guillain-Barré Syndrome Disability Scale (GBS-DS)	The mean time to first achieve a GBS-DS score of ≤1 within 12 weeks.	12 weeks.

Collaborators and Investigators

• Sponsor: Zhongming Qiu

Study record dates

Study Major Dates

• Study Start (Estimated): April 13, 2025
• Primary Completion (Estimated): June 30, 2026
• Study Completion (Estimated): December 31, 2026

Study Registration Dates

• First Submitted: March 13, 2025
• First Submitted That Met QC Criteria: March 13, 2025
• First Posted (Actual): March 25, 2025

Study Record Updates

• Last Update Posted (Actual): March 25, 2025
• Last Update Submitted That Met QC Criteria: March 13, 2025
• Last Verified: February 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Post-Infectious Disorders; Nervous System Diseases; Pathologic Processes; Neuromuscular Diseases; Chronic Disease; Disease Attributes; Autoimmune Diseases; Immune System Diseases; Peripheral Nervous System Diseases; Disease; Autoimmune Diseases of the Nervous System; Demyelinating Diseases; Polyneuropathies; Polyradiculoneuropathy; Syndrome; Guillain-Barre Syndrome
• Other Study ID Numbers: 2025-010-01

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No