Guillain-Barre SyndromePhase 2NCT05701189
Evaluating Efgartigimod in Patients With Guillain-Barré Syndrome
A Phase 2 study testing Efgartigimod Alfa-Fcab and Intravenous Immunoglobulin (IVIg) in people with Guillain-Barre Syndrome, run by Chafic Karam at 1 site in United States.
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? In addition to… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Guillain-Barre Syndrome
- What is being tested?
- Efgartigimod Alfa-Fcab and Intravenous Immunoglobulin (IVIg)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- 1 siteUnited States
- Study dates
- 10 Sep 2024to 20 Feb 2026
- Phase
- Phase 2
- Are there results?
- None posted in the registry
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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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.
Show all 10Show fewer
- 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.
Trial locations and contacts
1 site in 1 country.
United States1 site
Hospital of University of Pennsylvania
Philadelphia, Pennsylvania, 19104
About this study
Official title: Phase 2, Randomized, Patient- and Rater-blinded Single-site Trial Evaluating Safety and Efficacy of Efgartigimod in Patients With Guillain-Barré Syndrome.
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?
Read more
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.
Read the detailed description
The following procedures/assessments are standard-of-care for Guillain-Barré syndrome. They will not be performed solely for research purposes in this study, but the study team will use select results from them to draw conclusions related to this research:
- The study doctor or study staff will ask you about your medical history and any changes to your medications
- You will have a physical examination
- Your vital signs, including blood pressure, pulse rate, breathing rate, body temperature, and weight will be measured
- You will have an electrocardiogram (ECG)
- Pulmonary function testing, including forced vital capacity (FVC), maximal inspiratory pressure (MIP), and maximal expiratory pressure (MEP) will be performed
- Blood samples will be drawn to evaluate disease progression and safety/efficacy of treatment
- You will undergo a lumbar puncture, commonly referred to as a spinal tap, to collect cerebrospinal fluid (CSF) for analysis
- Nerve conduction studies (NCS) will be performed
- Outcome assessments including the GBS Disability Scale (GBS-DS), MRC Sum Score, and Inflammatory Rasch-built Overall Disability Scale (I-RODS) will be performed
- Conditions
- Guillain-Barre Syndrome
- Being tested
- Efgartigimod Alfa-Fcab, Intravenous Immunoglobulin (IVIg)
- 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.
Efgartigimod Alfa-FcabDrug
Also called Vyvgart
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.
Intravenous Immunoglobulin (IVIg)Drug
Also called Standard of Care IVIG
IVIg is the standard-of-care treatment for GBS. Brand of IVIG used may vary per institutional standards
| Study group | Role | What they receive |
|---|---|---|
| Efgartigimod Alfa-Fcab | Experimental | 20mg/kg of Intravenous efgartigimod on days 1 and 5, with normal saline administered as placebo on days 2-4 Receives: Drug: Efgartigimod Alfa-Fcab |
| Intravenous Immunoglobulin (IVIg) | Active comparator | 0.4g/kg of IVIg daily for 5 days Receives: Drug: Intravenous Immunoglobulin (IVIg) |
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 in adverse events in the studied population
Safety monitoring
Measured: Through study completion, an average of 3 years
Secondary outcomes (9)
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: 12 and 24 weeks
MRC Sum Score
Quantifies strength, with a higher score indicating more strength.
Measured: 12 and 24 weeks
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: 4, 8, 12, and 24 weeks
Number of Days on Respirator
as noted in the title
Measured: 4, 8, 12, and 24 weeks
Number of days in an intensive care unit
as noted in the title
Measured: 4, 8, 12, and 24 weeks
Number of days to hospital discharge
as noted in the title
Measured: 4, 8, 12, and 24 weeks
Mortality
Change in vital status
Measured: 4, 8, 12, and 24 weeks
Percentage of patients with secondary deterioration due to treatment-related fluctuations (TRF)
as noted in the title
Measured: 4, 8, 12, and 24 weeks
Adverse Events
occurrence of adverse events
Measured: 4, 8, 12, and 24 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Jan 2023
- Study start10 Sep 2024
- Primary completion19 Jan 2026
- Study completion20 Feb 2026 (estimated)
Why it stopped: Study will not be proceeding due to continued difficulties with participant enrollment at a single site.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Guillain-Barre SyndromeStudies of the same condition, open and under way.
- 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
- Double blind
- Who is masked
- Participant, Outcomes assessor
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Chafic Karam
- Sponsor type
- University, hospital or other
- Collaborators
- argenx
- Responsible party
- Sponsor-investigator, Chafic Karam, University of Pennsylvania
- Investigators
- Chafic Karam, MD, Principal investigator, Staff Physician and Associate Professor of Clinical NeurologyColin Quinn, MD, Study director, Staff Physician and Associate Professor of Clinical Neurology
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05701189
- Sponsor's protocol ID
- 852292
- Status last verified
- Mar 2026
- First submitted
- 5 Jan 2023
- Last updated
- 12 Mar 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 12 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase 2, Randomized, Patient- and Rater-blinded Single-site Trial Evaluating Safety and Efficacy of Efgartigimod in Patients With Guillain-Barré Syndrome.
- ClinicalTrials.gov ID
- NCT05701189
- Acronym / sponsor ID
- 852292
- Conditions
- Guillain-Barre Syndrome
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Study will not be proceeding due to continued difficulties with participant enrollment at a single site.
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Chafic KaramUniversity, hospital or other
- Collaborators
- argenx
- Study start
- 10 Sep 2024 (actual)
- Primary completion
- 19 Jan 2026 (actual)
- Study completion
- 20 Feb 2026 (estimated)
- First posted
- 27 Jan 2023 (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?
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.
Detailed description
The following procedures/assessments are standard-of-care for Guillain-Barré syndrome. They will not be performed solely for research purposes in this study, but the study team will use select results from them to draw conclusions related to this research:
- The study doctor or study staff will ask you about your medical history and any changes to your medications
- You will have a physical examination
- Your vital signs, including blood pressure, pulse rate, breathing rate, body temperature, and weight will be measured
- You will have an electrocardiogram (ECG)
- Pulmonary function testing, including forced vital capacity (FVC), maximal inspiratory pressure (MIP), and maximal expiratory pressure (MEP) will be performed
- Blood samples will be drawn to evaluate disease progression and safety/efficacy of treatment
- You will undergo a lumbar puncture, commonly referred to as a spinal tap, to collect cerebrospinal fluid (CSF) for analysis
- Nerve conduction studies (NCS) will be performed
- Outcome assessments including the GBS Disability Scale (GBS-DS), MRC Sum Score, and Inflammatory Rasch-built Overall Disability Scale (I-RODS) will be performed
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Efgartigimod Alfa-Fcab | Experimental | Drug: Efgartigimod Alfa-Fcab20mg/kg of Intravenous efgartigimod on days 1 and 5, with normal saline administered as placebo on days 2-4 |
| Intravenous Immunoglobulin (IVIg) | Active comparator | Drug: Intravenous Immunoglobulin (IVIg)0.4g/kg of IVIg daily for 5 days |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- 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
Key exclusion criteria (10 listed)
- 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.
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 |
|---|---|---|
| Efgartigimod Alfa-FcabAlso: VyvgartEfgartigimod 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 |
| Intravenous Immunoglobulin (IVIg)Also: Standard of Care IVIGIVIg is the standard-of-care treatment for GBS. Brand of IVIG used may vary per institutional standards | Drug | Intravenous Immunoglobulin (IVIg) |
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 in adverse events in the studied population
Time frame: Through study completion, an average of 3 years
Safety monitoring
9 secondary outcomes
Guillain-Barre Syndrome Disability Scale (GBS-DS)
Time frame: 12 and 24 weeks
MRC Sum Score
Time frame: 12 and 24 weeks
Inflammatory Rasch-Built Overall Disability Scale (I-RODS)
Time frame: 4, 8, 12, and 24 weeks
Number of Days on Respirator
Time frame: 4, 8, 12, and 24 weeks
Number of days in an intensive care unit
Time frame: 4, 8, 12, and 24 weeks
Number of days to hospital discharge
Time frame: 4, 8, 12, and 24 weeks
Mortality
Time frame: 4, 8, 12, and 24 weeks
Percentage of patients with secondary deterioration due to treatment-related fluctuations (TRF)
Time frame: 4, 8, 12, and 24 weeks
Adverse Events
Time frame: 4, 8, 12, and 24 weeks
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Mar 2026.
Related trials for Guillain-Barre Syndrome
Recruiting
High-Tech Rehabilitation Pathway for Acute Adult Neuromuscular Diseases - Fit4MedRob-Acute MND Project
Critical Illness Myopathy, Guillain Barré Syndrome
Not yet recruitingPhase 2
A Study of Empasiprubart as Add-On Therapy to IVIg in Adult Participants With Guillain-Barré Syndrome
Guillain-Barré Syndrome (GBS)
RecruitingPhase 3
An Open Label Clinical Study to Evaluate Tanruprubart (Also Commonly Known as ANX005) in Participants With Guillain-Barré Syndrome (FORWARD Study)
Guillain-Barre Syndrome
Not yet recruitingObservational study
Peripheral Nerve Ultrasound in Children With Guillain-Barré Syndrome
Guillain-Barre Syndrome
RecruitingObservational study
Early vs. Late Tracheostomy in Patients With Guillain -Barre Syndrome
Guillain Barre Syndrome, Mechanical Ventilation
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. This study is not recruiting at the moment.
Chafic Karam, MD principal investigator
Staff Physician and Associate Professor of Clinical Neurology
Colin Quinn, MD study director
Staff Physician and Associate Professor of Clinical Neurology
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Efgartigimod Alfa-Fcab and Intravenous Immunoglobulin (IVIg) in people with Guillain-Barre Syndrome, run by Chafic Karam at 1 site in United States.
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 6 inclusion and 10 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: withdrawn, as of the sponsor's last update on 12 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
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?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. 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 12 Mar 2026.