Efgartigimod for the Treatment of Guillain-Barré Syndrome

NCT06885762 | Not Yet Recruiting Phase 2 FDA Drug

• 1 other identifier: 2025-010-01
• Study Type: interventional
• Target: 20
• Locations: 0 countries
• Sites: N/A

Brief Summary

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

Conditions

Guillain-Barre Syndrome (GBS)

Outcome Measures

Primary Outcomes (1)

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

  The mean time to first achieve a GBS-DS score of ≤1 within 12 weeks.

  12 weeks.

Study Arms (1)

Treatment group

EXPERIMENTAL

Drug: efgartigimod

Interventions

efgartigimod DRUG

efgartigimod 20 mg/kg was administered via intravenous infusion on day 1 and day 5 of the treatment period only.

Treatment group

Eligibility Criteria

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

You may qualify if:

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

You may not qualify if:

• Patients with GBS symptoms for more than 2 weeks;
• Known hypersensitivity of the study subject to any component of the investigational drug or any other anti-neonatal Fc receptor (FcRn) agent;
• Any uncontrolled active infection or severe infection within 8 weeks prior to screening;
• 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;
• Vaccination within 4 weeks prior to screening or planned vaccination during the study period;
• Pregnant or breastfeeding, or planning to become pregnant during the study period, or women of childbearing potential not using effective contraception;
• Currently participating in another clinical trial of similar investigational agents (FcRn antagonists);
• Use of the investigational drug within 3 months prior to screening or within five half-lives of the drug (whichever is longer);
• 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;
• 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.

Sponsors & Collaborators

• Zhongming Qiu: lead

MeSH Terms

Conditions

Guillain-Barre Syndrome

Interventions

efgartigimod alfa

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

Central Study Contacts

Zhaoyou Meng

CONTACT

0086-023-68774449; mengzhaoyou@tmmu.edu.cn

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: March 13, 2025
• First Posted: March 20, 2025
• Study Start: April 13, 2025
• Primary Completion (Estimated): June 30, 2026
• Study Completion (Estimated): December 31, 2026
• Last Updated: March 20, 2025

Record last verified: 2025-02