Guillain-Barré Syndrome
Conditions
Keywords
Efgartigimod, Guillain-Barré Syndrome
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
Interventions
IVIg is the standard-of-care treatment for GBS. The same brand of intravenous immunoglobulin (IVIG) was administered across different study sites
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.
Sponsors
Beijing Tiantan Hospital
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
SINGLE (Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to 70 Years
Healthy volunteers
No
Inclusion criteria
1. Aged 18-70 years 2. 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). 3. Onset of GBS within 14 days 4. GBS Disability Scale (GBS-DS) score of 3 to 5
Exclusion criteria
1. Patients with other diagnoses cannot be excluded 2. Patients with recurrent GBS 3. Patients with more than 14 days after onset of symptoms 4. GBS DS of 2 or less 5. Patients unable to cooperate with scale assessments due to psychiatric disorders or impaired consciousness 6. 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 7. 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 8. Total IgG level ≤ 6 g/L at screening 9. 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 10. Subjects with serious underlying diseases, such as cardiac dysfunction, coagulopathy dysfunction 11. Pregnant and lactating women, and those intending to become pregnant during the trial or within 90 days after the last dosing 12. 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 13. Use of investigational drug within 3 months or 5 half-lives of the drug (whichever is longer) prior to Screening 14. 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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Guillain-Barre Syndrome Disability Scale (GBS-DS) | week 4 | Scoring system that assesses the functional status of GBS subjects. Scores range from 0 to 6, with higher scores indicating a worse outcome. |
| Number and seriousness of adverse events | Through study completion, an average of 2 years | safety monitoring |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Guillain-Barre Syndrome Disability Scale (GBS-DS) | week 1，2，3，8，12，24 | Scoring system that assesses the functional status of GBS subjects. Scores range from 0 to 6, with higher scores indicating a worse outcome. |
| 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 | Peripheral neuropathy assessment. Scores range from 0 to 244, with higher scores indicating a worse outcome |
| Inflammatory Rasch-Built Overall Disability Scale (I-RODS) | week 1,4,8,12,24 | Functional outcome assessment. Scores range from 0 to 48, with higher scores indicating a better outcome |
| Inflammatory Neuropathy Cause and Treatment (INCAT) Scale | week 1,4,8,12,24 | — |
| MRC Sum Score | week 1,4,8,12,24 | Quantifies strength, with a higher score indicating more strength. |
| Modified Rankin Scale (mRS) | week 1,4,8,12,24 | — |
| Incidence of assisted mechanical ventilation | 24 weeks | — |
| Duration of mechanical ventilation (hours) | 24 weeks | — |
| Number of days in an intensive care unit | 24 weeks | — |
| Number of days to hospital discharge | 24 weeks | — |
| Total hospitalization cost (in RMB) | 24 weeks | — |
| Forced Vital Capacity (FVC) | week 4,12,24 | Pulmonary function test that measures the maximum amount of air you can forcibly exhale from your lungs after fully inhaling. Units are Liters and percentages, when comparing to a predicted value. |
| Maximal Inspiratory Pressure (MIP) | week 4,12,24 | Pulmonary function test that evaluates the strength of inspiratory muscles, primarily the diaphragm. Units are cm H2O (centimeters of water) |
| Maximal Expiratory Pressure (MEP) | week 4,12,24 | Pulmonary function test that evaluates the strength of expiratory muscles. Units are cm H2O (centimeters of water). |
Contacts
CONTACTZaiqiang Zhang Zhang, MD
Outcome results
None listed