Exploratory Study of Efficacy of Ocrelizumab in Autoimmune Encephalitis
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 入组人数
- 3
- 试验地点
- 1
- 主要终点
- Number of Participants Who Had Clinical Worsening
研究概览
简要总结
This pilot study is a randomized, double-blind, placebo controlled study of the efficacy of ocrelizumab in autoimmune encephalitis. Subjects with new diagnosis of autoimmune encephalitis will be invited to enroll in this study. Subjects will be randomized to receive ocrelizumab (an anti-CD20 therapy) or matched placebo, and will undergo three infusions over a six month period. Subjects will complete clinical visits over the study period, during which safety monitoring and neuropsychological assessments will be performed to assess for signs of clinical worsening from encephalitis. The primary outcome of this study is the proportion of patients who fail to complete the twelve month period without clinical worsening, as defined by the protocol. Subjects who experience early clinical worsening during the study may be offered open-label treatment with ocrelizumab at the discretion of the investigators.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
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 or greater
- •Able to obtain informed consent from patient or appropriate designee
- •Possible autoimmune encephalitis as defined by Table 1:
- •Reasonable exclusion of alternative causes
- •Subacute onset (< 3 months) of memory deficits, altered consciousness, and/or psychiatric symptoms
- •One or more of the following:
- •CSF (cerebrospinal fluid) pleocytosis (>5 cells/µl corrected, if necessary, for traumatic lumbar puncture)
- •EEG (electroencephalogram) with epileptiform or focal slow wave abnormalities involving temporal lobes
- •Brain abnormalities on T2/FLAIR MRI restricted to the mesial temporal (limbic) lobes
- •Associated dyskinesias (faciobrachial dystonic movements or orofacial dyskinesias)
- •Completed initial treatment with iv steroids (at least 3000mg solumedrol) and plasma exchange (at least 3 exchanges) within the past 8 weeks
- •Presence of one (or more) of the following autoantibodies in serum or CSF
- •NMDA receptor
排除标准
- •Prior immunosuppression treatment in past year (other than steroids, intravenous immunoglobulin and plasma exchange)
- •Active malignancy requiring chemotherapy
- •Evidence of active hepatitis or tuberculosis infection
- •Medical condition that (in investigators opinion) precludes the use of ocrelizumab
研究组 & 干预措施
Treatment Arm
Ocrelizumab will be administered 3 times over a 1 year study period. Subjects will receive a dose of 300 mg at week 0 (baseline) and again at week 2. The final dose of 600 mg will be administered at week 24.
干预措施: Ocrelizumab (Drug)
Treatment Placebo Arm
Saline will be used as the matching placebo
干预措施: Saline (Drug)
结局指标
主要结局
Number of Participants Who Had Clinical Worsening
时间窗: 12 months
The number of participants who had clinical worsening within 12 months.
次要结局
- Change in TFLS T-score (Texas Functional Living Scale) Score at 6 Months(Baseline, 6 month)
- Change in TFLS T Score (Texas Functional Living Scale) Score at 12 Months(Baseline, 12 months)
- Time to Treatment Failure(12 months)
研究者
Steven Vernino
PROFESSOR
University of Texas Southwestern Medical Center