Guillain-Barré SyndromePhase 2GREATNCT07806916
Efficacy and Safety of Efgartigimod in the Treatment of Guillain-Barré Syndrome(GREAT)
A Phase 2 study testing Intravenous immunoglobulin (IVIG) and Efgartigimod Alfa-Fcab in people with Guillain-Barré Syndrome, run by Beijing Tiantan Hospital.
Trial 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… Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Guillain-Barré Syndrome
- What is being tested?
- Intravenous immunoglobulin (IVIG) and Efgartigimod Alfa-Fcab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 110 participants
- Where
- No sites listed yet
- Study dates
- Sep 2026to Dec 2027
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
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- 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
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
About this study
Official title: Efficacy and Safety of Efgartigimod in the Treatment of Guillain-Barré Syndrome: A Prospective, Multicenter, Randomized Controlled Clinical Trial
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
- Being tested
- Intravenous immunoglobulin (IVIG), Efgartigimod Alfa-Fcab
- Keywords
- Efgartigimod, Guillain-Barré Syndrome
- Medical subject headings
- Guillain-Barre Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
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
Efgartigimod Alfa-FcabDrug
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.
| Study group | Role | What they receive |
|---|---|---|
| Intravenous Immunoglobulin (IVIg) | Active comparator | 0.4g/kg of IVIg daily for 5 days
Interventions:
Drug: Intravenous Immunoglobulin (IVIg) Receives: Drug: Intravenous immunoglobulin (IVIG) |
| Efgartigimod Alfa-Fcab | Experimental | 20mg/kg of Intravenous efgartigimod on days 1 and 5
Interventions:
Drug: Efgartigimod Alfa-Fcab Receives: Drug: Efgartigimod Alfa-Fcab |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: week 4
Number and seriousness of adverse events
safety monitoring
Measured: Through study completion, an average of 2 years
Secondary outcomes (17)
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.
Measured: week 1,2,3,8,12,24
Change from baseline in 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.
Measured: week 1,2,3,4,8,12,24
Number of days to GBS-DS ≤2 within 24 weeks
Measured: 24 weeks
Proportion of patients with GBS-DS ≤2
Measured: week 1, 2, 3, 4, 8, 12, and 24
Neuropathy Impairment Scale (NIS)
Peripheral neuropathy assessment. Scores range from 0 to 244, with higher scores indicating a worse outcome
Measured: week 1,4,8,12,24
Inflammatory Rasch-Built Overall Disability Scale (I-RODS)
Functional outcome assessment. Scores range from 0 to 48, with higher scores indicating a better outcome
Measured: week 1,4,8,12,24
Inflammatory Neuropathy Cause and Treatment (INCAT) Scale
Functional outcome assessment. Scores range from 0 to 5 for upper limbs or lower limbs, with higher scores indicating a worse outcome
Measured: week 1,4,8,12,24
Medical Research Council Sum Score(MRC-SS)
Quantifies strength.Scores range from 0 to 60, with a higher score indicating more strength.
Measured: week 1,4,8,12,24
Modified Rankin Scale (mRS)
Modified Rankin Scale (mRS) is a commonly used scale for measuring the degree of disability or dependence in the daily activities of people who have suffered a stroke or other causes of neurological disability. Scores range from 0 to 6,with higer scores indicating a worse outcome
Measured: week 1,4,8,12,24
Incidence of assisted mechanical ventilation
Measured: 24 weeks
Duration of mechanical ventilation (hours)
Measured: 24 weeks
Number of days in an intensive care unit
Measured: 24 weeks
Number of days to hospital discharge
Measured: 24 weeks
Hospitalization cost (inpatient cost)
CNY (Chinese Yuan) Total direct medical costs incurred during the index hospitalization for the study disease, including bed fees, medications, laboratory and imaging tests, nursing, and medical materials, as recorded in the hospital billing system or inpatient settlement records.Indirect costs (e.g., lost productivity, transportation, caregiver costs) are not included unless otherwise specified.
Measured: 24 weeks
Forced Vital Capacity (FVC)
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.
Measured: week 4,12,24
Maximal Inspiratory Pressure (MIP)
Pulmonary function test that evaluates the strength of inspiratory muscles, primarily the diaphragm. Units are cm H2O (centimeters of water)
Measured: week 4,12,24
Maximal Expiratory Pressure (MEP)
Pulmonary function test that evaluates the strength of expiratory muscles. Units are cm H2O (centimeters of water).
Measured: week 4,12,24
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Sep 2026
- Study startSep 2026 (estimated)
- Primary completionJun 2027 (estimated)
- Study completionDec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Guillain-Barré SyndromeStudies of the same condition, open and under way.
- More trials run by Beijing Tiantan HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind
- Who is masked
- Outcomes assessor
- Enrolment
- 110 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Beijing Tiantan Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Zaiqiang Zhang, Beijing Tiantan Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07806916
- Sponsor's protocol ID
- KY2026-050-02
- Acronym
- GREAT
- Status last verified
- Sep 2026
- First submitted
- 2 Sep 2026
- Last updated
- 18 Sep 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 18 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Efficacy and Safety of Efgartigimod in the Treatment of Guillain-Barré Syndrome: A Prospective, Multicenter, Randomized Controlled Clinical Trial
- ClinicalTrials.gov ID
- NCT07806916
- Acronym / sponsor ID
- GREAT / KY2026-050-02
- Conditions
- Guillain-Barré Syndrome
- Keywords
- Efgartigimod, Guillain-Barré Syndrome
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 110 participants (estimated)
- Lead sponsor
- Beijing Tiantan HospitalUniversity, hospital or other
- Study start
- Sep 2026 (estimated)
- Primary completion
- Jun 2027 (estimated)
- Study completion
- Dec 2027 (estimated)
- First posted
- 8 Sep 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind (outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Intravenous Immunoglobulin (IVIg) | Active comparator | Drug: Intravenous immunoglobulin (IVIG)0.4g/kg of IVIg daily for 5 days Interventions: Drug: Intravenous Immunoglobulin (IVIg) |
| Efgartigimod Alfa-Fcab | Experimental | Drug: Efgartigimod Alfa-Fcab20mg/kg of Intravenous efgartigimod on days 1 and 5 Interventions: Drug: Efgartigimod Alfa-Fcab |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- 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
Key exclusion criteria (14 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Intravenous immunoglobulin (IVIG)IVIg is the standard-of-care treatment for GBS. The same brand of intravenous immunoglobulin (IVIG) was administered across different study sites | Drug | Intravenous Immunoglobulin (IVIg) |
| Efgartigimod Alfa-FcabEfgartigimod 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. | Drug | Efgartigimod Alfa-Fcab |
Primary outcome
Guillain-Barre Syndrome Disability Scale (GBS-DS)
Time frame: 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
Time frame: Through study completion, an average of 2 years
safety monitoring
17 secondary outcomes
Guillain-Barre Syndrome Disability Scale (GBS-DS)
Time frame: week 1,2,3,8,12,24
Change from baseline in Guillain-Barre Syndrome Disability Scale (GBS-DS)
Time frame: week 1,2,3,4,8,12,24
Number of days to GBS-DS ≤2 within 24 weeks
Time frame: 24 weeks
Proportion of patients with GBS-DS ≤2
Time frame: week 1, 2, 3, 4, 8, 12, and 24
Neuropathy Impairment Scale (NIS)
Time frame: week 1,4,8,12,24
Inflammatory Rasch-Built Overall Disability Scale (I-RODS)
Time frame: week 1,4,8,12,24
Inflammatory Neuropathy Cause and Treatment (INCAT) Scale
Time frame: week 1,4,8,12,24
Medical Research Council Sum Score(MRC-SS)
Time frame: week 1,4,8,12,24
Modified Rankin Scale (mRS)
Time frame: week 1,4,8,12,24
Incidence of assisted mechanical ventilation
Time frame: 24 weeks
Duration of mechanical ventilation (hours)
Time frame: 24 weeks
Number of days in an intensive care unit
Time frame: 24 weeks
Number of days to hospital discharge
Time frame: 24 weeks
Hospitalization cost (inpatient cost)
Time frame: 24 weeks
Forced Vital Capacity (FVC)
Time frame: week 4,12,24
Maximal Inspiratory Pressure (MIP)
Time frame: week 4,12,24
Maximal Expiratory Pressure (MEP)
Time frame: week 4,12,24
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Sep 2026.
- Individual participant dataSharing planned: Yes
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Intravenous immunoglobulin (IVIG) and Efgartigimod Alfa-Fcab in people with Guillain-Barré Syndrome, run by Beijing Tiantan Hospital.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 18 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Zaiqiang Zhang Zhang, MD (86-10-59975570, ttyy0142011@126.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Guillain-Barre Syndrome Disability Scale (GBS-DS) (time frame: week 4). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Sep 2026.