Encephalitis AutoimmunePhase 2ANSWERNCT07822776
Efgartigimod in Anti-NMDA Receptor Encephalitis.
A Phase 2 study testing Efgartigimod Alfa in people with Encephalitis Autoimmune, run by Beijing Tongren Hospital at 1 site in China.
Trial summary
This study is a multicenter, open-label, single-arm exploratory trial designed to enroll 20 eligible patients with anti-NMDAR encephalitis. It aims to evaluate the efficacy and safety of efgartigimod in the acute phase of anti-NMDAR encephalitis. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Encephalitis Autoimmune
- What is being tested?
- Efgartigimod Alfa
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 1 siteChina
- Study dates
- 25 Jun 2026to 30 Mar 2028
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Age ≥18 years old, male or female;
- Diagnosed as anti-NMDAR encephalitis, diagnostic criteria as follows:
- At least one of the following six major symptoms:
- Abnormal (mental) behavior or cognitive dysfunction
- Speech dysfunction (verbal urgency, hypospeech, mutism)
- Seizures
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- Movement disorders, dyskinesias, or postural rigidity/abnormalities
- Decreased level of consciousness
- Autonomic dysfunction or central hypoventilation in the presence of one or more of the six major symptoms;
- Positive anti-NMDAR IgG antibody: Diagnosis should be based on CSF antibody positivity using CBA. If only serum samples are available for testing, a positive CBA result must be supplemented with TBA on cultured neurons for definitive confirmation. Serum positivity at low titers (1:10) is not diagnostically significant.
- c．Reasonable exclusion of other etiologies.
- Patients with newly diagnosed or relapsed anti-NMDAR encephalitis who scored ≥2 on the mRS (5 patients each with mRS scores of 2-5);
- Newly diagnosed patients must not have received any prior immunosuppressive therapy.
- For relapsed patients:
- For subjects receiving rituximab, treatment must have commenced at least 2 months prior to screening, with the final dose administered no less than 4 weeks before randomization, and no improvement in mRS score within the 4 weeks preceding randomization.
- For subjects receiving other immunosuppressive agents (i.e., mycophenolate mofetil, cyclophosphamide, or azathioprine), treatment must have been ongoing for at least 2 months prior to screening, the dose must have been stable for at least 4 weeks prior to screening, and there must have been no improvement in the mRS score within 4 weeks prior to randomization.
- For subjects receiving oral corticosteroids, those receiving a stable daily dose of ≥20 mg of prednisolone (or its equivalent) with no increase in steroid dosage within 4 weeks prior to screening, and no improvement in mRS score within 4 weeks prior to randomization.
- For subjects receiving repeated courses (pulse therapy) of acute first-line therapy, treatment must be completed >2 weeks prior to randomization (baseline visit).
- Study subjects had received at least 3 days of glucocorticoid therapy at a dose of 500-1000 mg MP daily within 2 weeks prior to enrollment (baseline visit). They had transitioned to oral corticosteroid therapy and stable doses of non-steroidal immunosuppressive agents (NISIT) (limited to relapsed patients), and had not received IVIG or plasma exchange.
- For women of childbearing potential should use effective contraception during treatment and for at least 3 months after the last dose of Efgartigimod.
- Ability to sign an informed consent form, which includes agreeing to comply with the requirements and restrictions outlined in the informed consent form and this protocol.
Who cannot join
- Subjects should be excluded from the study if they meet any of the following criteria:
- Presence of any untreated teratoma or thymoma at baseline visit. Detection of teratoma or thymoma prior to or during the screening period is permitted if the disease is considered cured following treatment (typically surgical resection) within 1 week before baseline.
- Known allergy to any component of the study drug or any other anti-FcRn drug.
- Received IVIG or PE therapy within 2 weeks prior to screening.
- Research participants with clinically significant active infections (including unresolved or inadequately treated infections) as assessed by the investigator.
- Malignancies requiring chemotherapy.
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- Total IgG level ≤6 g/L in study subjects during screening visits.
- Pregnancy
- Patients with severe underlying conditions such as cardiac insufficiency, arrhythmia, or coagulation disorders.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
Beijing Tongren Hospital,Capital Medical University, Beijing, Beijing 100730
Beijing, 102600
Jiawei Wang, 18811612263, wangjwcq@163.com
About this study
Official title: Efficacy and Safety of Efgartigimod in Anti-N-methyl-D-aspartate Receptor Encephalitis: a Phase II Multicenter Single-arm Prospective Pilot Study
This study is a multicenter, open-label, single-arm exploratory trial designed to enroll 20 eligible patients with anti-NMDAR encephalitis. It aims to evaluate the efficacy and safety of efgartigimod in the acute phase of anti-NMDAR encephalitis. The study drug is efgartigimod, α injection administered intravenously at a dose of 10 mg/kg (maximum dose 1200 mg). Each infusion lasts approximately 1 hour, administered weekly for a total of 4 doses. The primary endpoint is the change in modified Rankin Scale (mRS) score at week 4 (day 28) post-treatment compared to baseline.
- Conditions
- Encephalitis Autoimmune
- Being tested
- Efgartigimod Alfa
- Keywords
- Anti-NMDAR encephalitis, Treatment, Antibody clearance, FcRn, Efgartigimod
- Medical subject headings
- Autoimmune Diseases of the Nervous System, Anti-N-Methyl-D-Aspartate Receptor Encephalitis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Efgartigimod AlfaDrug
Also called No other Intervention Names
Efgartigimod will be intravenously injected at a dose of 10mg/kg per week, lasting for 4 weeks.
| Study group | Role | What they receive |
|---|---|---|
| Efgartigimod treatment group | Experimental | Efgartigimod will be intravenously injected at a dose of 10mg/kg per week, lasting for 4 weeks. Receives: Drug: Efgartigimod Alfa |
What is being measured
The primary outcome is the main question the study is designed to answer.
the change of modified Rankin Scale (mRS) score
the change in modified Rankin Scale (mRS) score at week 4 (day 28) post-treatment compared to baseline.
modified Rankin Scale (mRS): from 0 to 6 The higher the patient's mRS score, the more severe the neurological deficit.
Measured: from baseline to week 4
Secondary outcomes (8)
the change of Clinical Assessment Scale for Autoimmune Encephalitis (CASE) score from baseline to week 12
Changes from baseline in Clinical Assessment Scale for Autoimmune Encephalitis (CASE) at 12 weeks post-treatment.
Clinical Assessment Scale for Autoimmune Encephalitis (CASE) : from 0 to 27. The higher the patient's CASE score, the more severe the neurological deficit.
Measured: from baseline to week 12
the change of Clinical Global Impression Scale (CGI) score
Changes from baseline in Clinical Global Impression Scale (CGI) at 12 weeks post-treatment.
Subscale scores: Severity Index (SI) (0-7) and Global Improvement Index (GI) (0-7). Efficacy Index (EI) (0-4.00). In pharmacological studies, only drug formulations with an Efficacy Index of 1.0 or higher are considered valuable.
Measured: from baseline to week 12
the change of Glasgow Coma Scale (GCS) score
Changes from baseline in Glasgow Coma Scale (GCS) at 12 weeks post-treatment. Glasgow Coma Scale (GCS) : from 3 to 15. The lower the patient's mRS score, the more severe the Impaired consciousness.
Measured: from baseline to week 12
the change of Anti-NMDAR Encephalitis One-Year Functional Status Score (NEOS) score
Changes from baseline in Anti-NMDAR Encephalitis One-Year Functional Status Score (NEOS) at 12 weeks post-treatment.
Anti-NMDAR Encephalitis One-Year Functional Status Score (NEOS): from 0 to 5. The higher the NEOS score, the poorer the patient's 1-year prognosis.
Measured: from baseline to week 12
Time of clinical improvement
Time to baseline improvement of ≥1 point in mRS score without rescue therapy
Measured: from baseline to week 4
Changes in anti-NMDAR antibody levels at 12 weeks post-treatment
detection of changes in anti-NMDAR antibody levels at 12 weeks post-treatment
Measured: from baseline to week 12
Changes in total IgG levels at 12 weeks post-treatment
detection of changes in total IgG levels at 12 weeks post-treatment
Measured: from baseline to week 12
the change of Clinical Assessment Scale for Autoimmune Encephalitis (CASE) score
Changes from baseline in Clinical Assessment Scale for Autoimmune Encephalitis (CASE) at 12 weeks post-treatment.
Clinical Assessment Scale for Autoimmune Encephalitis (CASE) : from 0 to 27. The higher the patient's CASE score, the more severe the neurological deficit.
Measured: from baseline to week 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Sep 2026
- Study start25 Jun 2026
- Primary completion31 Dec 2027 (estimated)
- Study completion30 Mar 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Encephalitis AutoimmuneStudies 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Beijing Tongren Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- Henan Provincial People's Hospital, First Affiliated Hospital of Zhejiang University, Shandong Provincial Hospital, Peking Union Medical College, The Second Hospital of Hebei Medical University, Xijing Hospital, Beijing Huaxin Hospital
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07822776
- Sponsor's protocol ID
- MR-11-24-042362
- Other IDs
- CN-NEU-003
- Acronym
- ANSWER
- Status last verified
- Sep 2025
- First submitted
- 28 Apr 2026
- Last updated
- 16 Sep 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 16 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Efficacy and Safety of Efgartigimod in Anti-N-methyl-D-aspartate Receptor Encephalitis: a Phase II Multicenter Single-arm Prospective Pilot Study
- ClinicalTrials.gov ID
- NCT07822776
- Acronym / sponsor ID
- ANSWER / MR-11-24-042362
- Conditions
- Encephalitis Autoimmune
- Keywords
- Anti-NMDAR encephalitis, Treatment, Antibody clearance, FcRn, Efgartigimod
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- Beijing Tongren HospitalUniversity, hospital or other
- Collaborators
- Henan Provincial People's Hospital, First Affiliated Hospital of Zhejiang University, Shandong Provincial Hospital, Peking Union Medical College, The Second Hospital of Hebei Medical University, Xijing Hospital, Beijing Huaxin Hospital
- Study start
- 25 Jun 2026 (actual)
- Primary completion
- 31 Dec 2027 (estimated)
- Study completion
- 30 Mar 2028 (estimated)
- First posted
- 16 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.
This study is a multicenter, open-label, single-arm exploratory trial designed to enroll 20 eligible patients with anti-NMDAR encephalitis. It aims to evaluate the efficacy and safety of efgartigimod in the acute phase of anti-NMDAR encephalitis. The study drug is efgartigimod, α injection administered intravenously at a dose of 10 mg/kg (maximum dose 1200 mg). Each infusion lasts approximately 1 hour, administered weekly for a total of 4 doses. The primary endpoint is the change in modified Rankin Scale (mRS) score at week 4 (day 28) post-treatment compared to baseline.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Efgartigimod treatment group | Experimental | Drug: Efgartigimod AlfaEfgartigimod will be intravenously injected at a dose of 10mg/kg per week, lasting for 4 weeks. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (21 listed)
- Age ≥18 years old, male or female;
- Diagnosed as anti-NMDAR encephalitis, diagnostic criteria as follows:
- c．Reasonable exclusion of other etiologies.
- Patients with newly diagnosed or relapsed anti-NMDAR encephalitis who scored ≥2 on the mRS (5 patients each with mRS scores of 2-5);
- Study subjects had received at least 3 days of glucocorticoid therapy at a dose of 500-1000 mg MP daily within 2 weeks prior to enrollment (baseline visit). They had transitioned to oral corticosteroid therapy and stable doses of non-steroidal immunosuppressive agents (NISIT) (limited to relapsed patients), and had not received IVIG or plasma exchange.
Key exclusion criteria (9 listed)
- Presence of any untreated teratoma or thymoma at baseline visit. Detection of teratoma or thymoma prior to or during the screening period is permitted if the disease is considered cured following treatment (typically surgical resection) within 1 week before baseline.
- Known allergy to any component of the study drug or any other anti-FcRn drug.
- Received IVIG or PE therapy within 2 weeks prior to screening.
- Research participants with clinically significant active infections (including unresolved or inadequately treated infections) as assessed by the investigator.
- Malignancies requiring chemotherapy.
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 AlfaAlso: No other Intervention NamesEfgartigimod will be intravenously injected at a dose of 10mg/kg per week, lasting for 4 weeks. | Drug | Efgartigimod treatment group |
Primary outcome
the change of modified Rankin Scale (mRS) score
Time frame: from baseline to week 4
the change in modified Rankin Scale (mRS) score at week 4 (day 28) post-treatment compared to baseline. modified Rankin Scale (mRS): from 0 to 6 The higher the patient's mRS score, the more severe the neurological deficit.
8 secondary outcomes
the change of Clinical Assessment Scale for Autoimmune Encephalitis (CASE) score from baseline to week 12
Time frame: from baseline to week 12
the change of Clinical Global Impression Scale (CGI) score
Time frame: from baseline to week 12
the change of Glasgow Coma Scale (GCS) score
Time frame: from baseline to week 12
the change of Anti-NMDAR Encephalitis One-Year Functional Status Score (NEOS) score
Time frame: from baseline to week 12
Time of clinical improvement
Time frame: from baseline to week 4
Changes in anti-NMDAR antibody levels at 12 weeks post-treatment
Time frame: from baseline to week 12
Changes in total IgG levels at 12 weeks post-treatment
Time frame: from baseline to week 12
the change of Clinical Assessment Scale for Autoimmune Encephalitis (CASE) score
Time frame: from baseline to week 12
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 16 Sep 2026.
- Individual participant dataSharing planned: No
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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.
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 in people with Encephalitis Autoimmune, run by Beijing Tongren Hospital at 1 site in China.
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 21 inclusion and 9 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: looking for participants now, as of the sponsor's last update on 16 Sep 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 China. 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?
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 Jiawei Wang (010-58266091, wangjwcq@163.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
the change of modified Rankin Scale (mRS) score (time frame: from baseline to week 4). 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 16 Sep 2026.