Evaluating Efgartigimod in Patients With Guillain-Barré Syndrome

NCT05701189 | Withdrawn Phase 2 DMC FDA Drug

• 1 other identifier: 852292
• Study Type: interventional
• Target: N/A
• Locations: 1 country
• Sites: 1

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? 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.

Conditions

Guillain-Barre 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 in adverse events in the studied population

  Safety monitoring

  Through study completion, an average of 3 years

Secondary Outcomes (9)

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

  12 and 24 weeks

• MRC Sum Score

  12 and 24 weeks

• Inflammatory Rasch-Built Overall Disability Scale (I-RODS)

  4, 8, 12, and 24 weeks

• Number of Days on Respirator

  4, 8, 12, and 24 weeks

• Number of days in an intensive care unit

  4, 8, 12, and 24 weeks

Other Outcomes (1)

• Study effect of Efgartigimod on IgG levels

  change from baseline to 4 weeks and 24 weeks

Study Arms (2)

Efgartigimod Alfa-Fcab

EXPERIMENTAL

20mg/kg of Intravenous efgartigimod on days 1 and 5, with normal saline administered as placebo on days 2-4

Drug: Efgartigimod Alfa-Fcab

Intravenous Immunoglobulin (IVIg)

ACTIVE COMPARATOR

0.4g/kg of IVIg daily for 5 days

Drug: Intravenous Immunoglobulin (IVIg)

Interventions

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.

Also known as: Vyvgart

Efgartigimod Alfa-Fcab

Intravenous Immunoglobulin (IVIg) DRUG

IVIg is the standard-of-care treatment for GBS. Brand of IVIG used may vary per institutional standards

Also known as: Standard of Care IVIG

Intravenous Immunoglobulin (IVIg)

Eligibility Criteria

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

You may qualify if:

• Provision of signed and dated informed consent form
• Stated willingness to comply with all study procedures and availability for the duration of the study
• Male or female, aged 18 years or older
• Have a diagnosis of GBS according to the National Institute of Neurological Disorders and Stroke Diagnostic Criteria for Guillain-Barré Syndrome
• Onset of GBS-related weakness ≤14 days prior to infusion
• GBS-DS score of 3, 4, or 5

You may not qualify if:

• Pregnant and lactating women, and those intending to become pregnant during the trial or within 90 days after the last dosing. Women of childbearing potential should have a negative serum pregnancy test at Screening and a negative urine pregnancy test at Baseline prior to administration of IMP. Note: Women of childbearing potential should use a highly effective method of contraception (i.e., pregnancy rate of less than 1% per year) during the trial and for 90 days after the last administration of the IMP. They must be on a stable regimen, for at least 1 month, of combined estrogen and progestogen hormonal contraception with inhibition of ovulation, progestogen-only hormonal contraception associated with inhibition of ovulation, intrauterine device (IUD), intrauterine hormone-releasing system, bilateral tubal occlusion, vasectomized partner, or agree upon continuous abstinence from heterosexual sexual contact.
• Male patients who are sexually active and do not intend to use effective methods of contraception (as mentioned above) during the trial or within 90 days after the last dosing or male patients who plan to donate sperm during the trial or within 90 days after the last dosing. Note: Sterilized male patients who have had vasectomy with documented aspermia post-procedure, or male patients who have a partner of non-childbearing potential, can be included.
• GBS DS of 2 or less.
• 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.
• Patients with more than 14 days after onset of symptoms.
• Patients with known IgG deficiency.
• Patients with recurrent GBS.
• Use of investigational drug within 3 months or 5 half-lives of the drug (whichever is longer) prior to Screening.
• 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.
• Patients with clinical evidence of other significant serious disease or patients who underwent a recent major surgery, which could confound the results of the trial or put the patient at undue risk. Patients with renal/hepatic function impairment can be included.

Sponsors & Collaborators

• Chafic Karam: lead
• argenx: collaborator

Study Sites (1)

Hospital of University of Pennsylvania

Philadelphia, Pennsylvania, 19104, United States

Location

MeSH Terms

Conditions

Guillain-Barre Syndrome

Interventions

efgartigimod alfa; 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

Study Officials

• Chafic Karam, MD

  Staff Physician and Associate Professor of Clinical Neurology

  PRINCIPAL INVESTIGATOR

• Colin Quinn, MD

  Staff Physician and Associate Professor of Clinical Neurology

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Staff Physician and Associate Professor of Clinical Neurology

Study Record Dates

• First Submitted: January 5, 2023
• First Posted: January 27, 2023
• Study Start: September 10, 2024
• Primary Completion: January 19, 2026
• Study Completion: February 20, 2026
• Last Updated: March 12, 2026

Record last verified: 2026-03

Locations

US (1)