Efgartigimod in Anti-NMDA Receptor Encephalitis.

NCT07822776 | Recruiting Phase 2

• 2 other identifiers: MR-11-24-042362CN-NEU-003
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 1

Brief 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. 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

Keywords

Anti-NMDAR encephalitis; Treatment; Antibody clearance; FcRn; Efgartigimod

Outcome Measures

Primary Outcomes (1)

• 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.

  from baseline to week 4

Secondary Outcomes (8)

• the change of Clinical Assessment Scale for Autoimmune Encephalitis (CASE) score from baseline to week 12

  from baseline to week 12

• the change of Clinical Global Impression Scale (CGI) score

  from baseline to week 12

• the change of Glasgow Coma Scale (GCS) score

  from baseline to week 12

• the change of Anti-NMDAR Encephalitis One-Year Functional Status Score (NEOS) score

  from baseline to week 12

• Time of clinical improvement

  from baseline to week 4

Study Arms (1)

Efgartigimod treatment group

EXPERIMENTAL

Efgartigimod will be intravenously injected at a dose of 10mg/kg per week, lasting for 4 weeks.

Drug: Efgartigimod Alfa

Interventions

Efgartigimod Alfa DRUG

Efgartigimod will be intravenously injected at a dose of 10mg/kg per week, lasting for 4 weeks.

Also known as: No other Intervention Names

Efgartigimod treatment group

Eligibility Criteria

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

You may qualify if:

• 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
• 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.
• 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.

You may not qualify if:

• 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.
• 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.

Sponsors & Collaborators

• Beijing Tongren Hospital: lead
• Henan Provincial People's Hospital: collaborator
• First Affiliated Hospital of Zhejiang University: collaborator
• Shandong Provincial Hospital: collaborator
• Peking Union Medical College: collaborator
• The Second Hospital of Hebei Medical University: collaborator
• Xijing Hospital: collaborator
• Beijing Huaxin Hospital: collaborator

Study Sites (1)

Beijing Tongren Hospital,Capital Medical University, Beijing, Beijing 100730

Beijing, 102600, China

RECRUITING

MeSH Terms

Conditions

Autoimmune Diseases of the Nervous System; Anti-N-Methyl-D-Aspartate Receptor Encephalitis

Interventions

efgartigimod alfa

Condition Hierarchy (Ancestors)

Nervous System Diseases; Autoimmune Diseases; Immune System Diseases; Paraneoplastic Syndromes, Nervous System; Nervous System Neoplasms; Neoplasms by Site; Neoplasms; Paraneoplastic Syndromes; Encephalitis; Brain Diseases; Central Nervous System Diseases; Neurodegenerative Diseases; Neuroinflammatory Diseases

Central Study Contacts

Jiawei Wang

CONTACT

010-58266091; wangjwcq@163.com

Yujing Peng

CONTACT

010-58266091; pengyujing1206@163.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 28, 2026
• First Posted: September 16, 2026
• Study Start: June 25, 2026
• Primary Completion (Estimated): December 31, 2027
• Study Completion (Estimated): March 30, 2028
• Last Updated: September 16, 2026

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)