NCT07822776招募中2 期
Efficacy and Safety of Efgartigimod in Anti-N-methyl-D-aspartate Receptor Encephalitis: a Phase II Multicenter Single-arm Prospective Pilot Study
Beijing Tongren Hospital1 个研究点 分布在 1 个国家目标入组 20 人开始时间: 2026年6月25日最近更新:
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 发起方
- 入组人数
- 20
- 试验地点
- 1
- 主要终点
- the change of modified Rankin Scale (mRS) score
研究概览
简要总结
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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)
- •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.
排除标准
- •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.
- •Patients with severe underlying conditions such as cardiac insufficiency, arrhythmia, or coagulation disorders.
研究组 & 干预措施
Efgartigimod treatment group
Experimental
Efgartigimod will be intravenously injected at a dose of 10mg/kg per week, lasting for 4 weeks.
干预措施: Efgartigimod Alfa (Drug)
结局指标
主要结局
the change of modified Rankin Scale (mRS) score
时间窗: 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.
次要结局
- 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)
- Changes in anti-NMDAR antibody levels at 12 weeks post-treatment(from baseline to week 12)
- Changes in total IgG levels at 12 weeks post-treatment(from baseline to week 12)
- the change of Clinical Assessment Scale for Autoimmune Encephalitis (CASE) score(from baseline to week 12)
研究者
研究点 (1)
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