Efgartigimod in Anti-NMDA Receptor Encephalitis.
ConditionEncephalitis Autoimmune
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age18+
SponsorBeijing Tongren Hospital
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Age ≥18 years old, male or female;
Abnormal (mental) behavior or cognitive dysfunction
Speech dysfunction (verbal urgency, hypospeech, mutism)
Seizures
Disqualifiers
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.
Trial design
Design model
Single group
Treatments tested in this trial
Efgartigimod Alfa
DrugEfgartigimod will be intravenously injected at a dose of 10mg/kg per week, lasting for 4 weeks.
Treatment groups
20 Participants
are divided into 1 treatment groupGroup A: Efgartigimod treatment groupExperimental treatment 1 intervention
Trial outcomes
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.
Time frame
from baseline to week 4Secondary outcomes
1
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.
Time frame
from baseline to week 122
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.
Time frame
from baseline to week 123
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.
Time frame
from baseline to week 124
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.
Time frame
from baseline to week 12Sponsors and contacts
Click on the lead sponsor to view all of their trials.
Beijing Tongren Hospital
Lead sponsor
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
Source ClinicalTrials.gov