A Study to Learn About How Well the Medicine Efgartigimod Works to Treat Autoimmune Encephalitis In Children 12 Years or Older and Adults (Polaris)
2026年8月21日 更新者：argenx
A Global, Multicenter, Randomized, Double-Blinded, Placebo-Controlled, Phase 2 Study to Evaluate the Efficacy, Safety, and Tolerability of Efgartigimod PH20 SC in Adult and Adolescent Participants With Autoimmune Encephalitis
The POLARIS study is designed to evaluate how well efgartigimod PH20 SC may work (called "efficacy") and how safe it is for people diagnosed with Autoimmune Encephalitis (AIE). The study consists of 4 parts: in part A participants will receive efgartigimod SC; in part B, participants will be randomized to receive either efgartigimod SC or placebo; in part C, participants who completed part B will receive efgartigimod SC; in part D, participants who completed part C will be observed after their last dose of efgartigimod SC. If AIE symptoms return, efgartigimod SC treatment may be restarted during this time.
The maximum overall study duration for participants is up to 3 years. More information can be found in clinicaltrials.argenx.com/polaris
研究概览
详细说明
The study is designed to address the unmet need for effective immunomodulatory therapy in AIE, enrolling patients across multiple antibody-defined subgroups, with the anti-NMDAR encephalitis group serving as the primary cohort for statistical analysis.
研究类型
介入性
注册 (估计的)
170
阶段
- 阶段2
联系人和位置
本节提供了进行研究的人员的详细联系信息，以及有关进行该研究的地点的信息。
学习联系方式
- 姓名：Sabine Coppieters, MD
- 电话号码：857-350-4834
- 邮箱：clinicaltrials@argenx.com
学习地点
-
-
Arizona
-
Tucson、Arizona、美国、85718
- 招聘中
- Center For Neurosciences
-
首席研究员：
- Francisco Valdivia
-
接触：
- Gwendolyn Grabiec
- 电话号码：(520) 529-5234
- 邮箱：ggrabiec@neurotucson.com
-
-
Texas
-
Sherman、Texas、美国、75092-7371
- 招聘中
- Texas Institute for Neurological Disorders
-
首席研究员：
- Bharathy Sundaram
-
接触：
- Laurie Laurie
- 电话号码：(903) 893-5141 x4358
- 邮箱：ljarvis@texasneurologyinstitute.com
-
-
参与标准
研究人员寻找符合特定描述的人，称为资格标准。这些标准的一些例子是一个人的一般健康状况或先前的治疗。
资格标准
适合学习的年龄
- 孩子
- 成人
- 年长者
接受健康志愿者
不
描述
Inclusion Criteria:
- Is at least 12 years of age.
- Meeting at least the criteria for possible AIE according to the Graus criteria.
- Part A:
Must not have received prior treatment for AIE with PLEX or Ig (participants may have received glucocorticoids); and must not have received PLEX or Ig for any other medical condition in the last 3 months
- Part B: Either completing Part A, or If directly entering Part B, must have received first-line treatment for AIE (i.e. corticosteroids, PLEX, and/or Ig) and have a CASE score of 3 or higher, or a score of 2 or higher in a single sub-item
Exclusion Criteria:
- Known anti-myelin oligodendrocyte glycoprotein (anti-MOG) antibody positivity.
- Any medical condition that would interfere with an accurate assessment of clinical symptoms of AIE.
- Recent major surgery (within 3 months of screening) or intention to have major surgery during the study, except for surgeries for AIE-related teratomas and thymomas.
- History (within 12 months before screening) of current alcohol, drug (including recreational or prescribed cannabinoids), or medication abuse.
- Psychiatric or cognitive impairment unrelated to AIE.
学习计划
本节提供研究计划的详细信息，包括研究的设计方式和研究的衡量标准。
研究是如何设计的？
设计细节
- 主要用途：治疗
- 分配：随机化
- 介入模型：并行分配
- 屏蔽：三倍
武器和干预
参与者组/臂 |
干预/治疗 |
|---|---|
|
实验性的：Part A (Open-Label Lead-in Period): Efgartigimod PH20 SC
All participants will receive efgartigimod PH20 SC open label for 8 weeks
|
subcutaneous administrations of efgartigimod PH20 SC given by prefilled syringe (PFS).
For participants aged 12 to <18 years with body weight ≤50 kg, the study drug will be administered by vial and syringe.
|
|
实验性的：Part B (Double-blinded treatment period): Efgartigimod PH20 SC
Participants will receive efgartigimod PH20 SC for 24 weeks
|
subcutaneous administrations of efgartigimod PH20 SC given by prefilled syringe (PFS).
For participants aged 12 to <18 years with body weight ≤50 kg, the study drug will be administered by vial and syringe.
|
|
安慰剂比较：Part B (Maintenance double-blinded treatment period): Placebo PH20 SC
Participants will receive placebo for 24 weeks
|
subcutaneous administrations of placebo PH20 SC given by prefilled syringe (PFS). For participants aged 12 to <18 years with body weight ≤50 kg, the study drug will be administered by vial and syringe |
|
实验性的：Part C (Open-Label Extension Period): Efgartigimod PH20 SC
Participants who complete Part B will receive efgartigimod PH20 SC for 24 weeks
|
subcutaneous administrations of efgartigimod PH20 SC given by prefilled syringe (PFS).
For participants aged 12 to <18 years with body weight ≤50 kg, the study drug will be administered by vial and syringe.
|
研究衡量的是什么？
主要结果指标
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
|
Change in CASE score in the NMDAR population
大体时间：up to week 24
|
CASE= Clinical Assessment Scale in Autoimmune Encephalitis; NMDAR=N-methyl-D-aspartate receptor; Neuropsychological Status.
The CASE (Clinical Assessment Scale in AIE) includes an assessment of 9 items: seizure, memory dysfunction, psychiatric symptoms, consciousness, language problems, dyskinesia/dystonia, gait instability and ataxia, brainstem dysfunction, weakness.
This overall CASE total score ranges from 0 to 27, with a higher score indicating a greater degree of disability.
|
up to week 24
|
次要结果测量
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
|
Change in mRS
大体时间：up to week 8
|
The mRS (modified Rankin Scale) is commonly used to measure the degree of disability or dependence in the daily activities of people with neurological disabilities.
Scores range from 0 (no symptoms) to 6(dead).
|
up to week 8
|
|
Change in CASE score
大体时间：up to week 8
|
The CASE (Clinical Assessment Scale in AIE) includes an assessment of 9 items: seizure, memory dysfunction, psychiatric symptoms, consciousness, language problems, dyskinesia/dystonia, gait instability and ataxia, brainstem dysfunction, weakness.
This overall CASE total score ranges from 0 to 27, with a higher score indicating a greater degree of disability.
|
up to week 8
|
|
Change in MoCA total score
大体时间：up to week 8
|
MoCA= Montreal Cognitive Assessment
|
up to week 8
|
|
Change in NPI-C total score
大体时间：up to week 8
|
The NPI-C (Neuropsychiatric Inventory--Clinician) total score will be used as a global measure of neuropsychiatric symptoms.Total score is calculated by summing the scores of all the individual domains.Each domain score is determined by summing the item scores within that domain.
The NPI-C uses a clinician rating method, where ratings for frequency, severity, and caregiver distress are provided for each item.These item scores are then summed to create a total domain score.
|
up to week 8
|
|
Change from baseline in CGI-S
大体时间：up to week 8
|
Expression of Change. CGI is a clinician-rated scale that measures illness severity (CGI-S) and global improvement or change (CGI-C).It is rated on a 7-point scale, from 1 (normal) to 7 (amongst the most severely ill patients) for CGI-S and from 1 (very much improved) to 7 (very much worse) for CGI-C. PGI-S and PGI-C are the patient-reported counterparts to CGI-S and CGI-C, respectively. |
up to week 8
|
|
Change from baseline in PGI-S
大体时间：up to week 8
|
A Impression of Severity; PGI-S= Patient Global Impression Scale.
PGI-S and PGI-C are the patient-reported counterparts to CGI-S and CGI-C, respectively.
|
up to week 8
|
|
Change from baseline in CGI-C
大体时间：up to week 8
|
CGI-C= Clinical Global Expression of Change . CGI is a clinician-rated scale that measures illness severity (CGI-S) and global improvement or change (CGI-C).It is rated on a 7-point scale, from 1 (normal) to 7 (amongst the most severely ill patients) for CGI-S and from 1 (very much improved) to 7 (very much worse) for CGI-C. PGI-S and PGI-C are the patient-reported counterparts to CGI-S and CGI-C, respectively. |
up to week 8
|
|
Change from baseline in PGI-C
大体时间：up to week 8
|
A Impression of Severity; PGI-C= Patient Global Expression of Change .
PGI-S and PGI-C are the patient-reported counterparts to CGI-S and CGI-C, respectively.
|
up to week 8
|
|
Incidence and severity of AEs
大体时间：up to week 8
|
AEs= Adverse Effects
|
up to week 8
|
|
Incidence and severity of SAEs
大体时间：up to week 8
|
SAEs = Serious Adverse Effects
|
up to week 8
|
|
Trough efgartigimod serum concentrations over time
大体时间：up to week 8
|
up to week 8
|
|
|
Percent change from baseline in total IgG levels in serum over time
大体时间：up to week 8
|
IgG= Immunoglobulin G
|
up to week 8
|
|
Incidence and prevalence of ADA against efgartigimod in serum over time
大体时间：up to week 8
|
ADA = antidrug antibody(ies)
|
up to week 8
|
|
Incidence and prevalence of antibodies against rHuPH20 in plasma over time
大体时间：up to week 8
|
rHuPH20 = Recombinant Human Hyaluronidase PH20
|
up to week 8
|
|
Change in mRS in the NMDAR population
大体时间：up to week 24
|
The mRS (modified Rankin Scale) is commonly used to measure the degree of disability or dependence in the daily activities of people with neurological disabilities.
Scores range from 0 (no symptoms) to 6(dead)
|
up to week 24
|
|
Change in NPI-C total score in the NMDAR population
大体时间：up to week 24
|
NPI-C=Neuropsychiatric Inventory-Clinician; NMDAR=N-methyl-D-aspartate receptor.
The NPI-C (Neuropsychiatric Inventory--Clinician) total score will be used as a global measure of neuropsychiatric symptoms.
Total score is calculated by summing the scores of all the individual domains.
Each domain score is determined by summing the item scores within that domain.
The NPI-C uses a clinician rating method, where ratings for frequency, severity, and caregiver distress are provided for each item.These item scores are then summed to create a total domain score.
|
up to week 24
|
|
Change in RBANS in the NMDAR population
大体时间：up to week 24
|
RBANS=Repeatable Battery for the Assessment of Neuropsychological Status; NMDAR=N-methyl-D-aspartate receptor.
The RBANS (Repeatable Battery for the Assessment of Neuropsychological Status) is a performance outcome measure developed to identify and characterize cognitive impairment by assessing an individual's current level of cognitive performance.
|
up to week 24
|
|
Percentage of CASE responders in the NMDAR population.
大体时间：at week 24
|
CASE = Clinical Assessment Scale in AIE ; NMDAR=N-methyl-D-aspartate receptor
|
at week 24
|
|
Change in CASE score in the non-NMDAR population
大体时间：up to week 24
|
CASE = Clinical Assessment Scale in AIE; NMDAR=N-methyl.
The CASE (Clinical Assessment Scale in AIE) includes an assessment of 9 items: seizure,memory dysfunction, psychiatric symptoms, consciousness, language problems, dyskinesia/dystonia, gait instability and ataxia, brainstem dysfunction, weakness.
This overall CASE total score ranges from 0 to 27, with a higher score indicating a greater degree of disability.
|
up to week 24
|
|
Change in RBANS in the non-NMDAR population
大体时间：up to week 24
|
RBANS= Repeatable Battery for the Assessment of Neuropsychological Status; NMDAR=N-methyl-D-aspartate receptor.
The RBANS (Repeatable Battery for the Assessment of Neuropsychological Status) is a performance outcome measure developed to identify and characterize cognitive impairment by assessing an individual's current level of cognitive performance.
|
up to week 24
|
|
Change in NPI-C total score in the non-NMDAR population
大体时间：up to week 24
|
NPI-C= Neuropsychiatric Inventory-Clinician; NMDAR=N-methyl-D-aspartate receptor.
The NPI-C Neuropsychiatric Inventory--Clinician) total score will be used as a global measure of neuropsychiatric symptoms.
Total score is calculated by summing the scores of all the individual domains.
Each domain score is determined by summing the item scores within that domain.
The NPI-C uses a clinician rating method, where ratings for frequency, severity, and caregiver distress are provided for each item.
|
up to week 24
|
|
Change in mRS in the non-NMDAR population
大体时间：up to week 24
|
mRS= modified Rankin Scale; NMDAR=N-methyl-D-aspartate receptor.
The mRS (modified Rankin Scale) is commonly used to measure the degree of disability or dependence in the daily activities of people with neurological disabilities.Scores range from 0 (no symptoms) to 6 (dead).
|
up to week 24
|
|
Percentage of CASE responders in the non-NMDAR population.
大体时间：at week 24
|
CASE = Clinical Assessment Scale in AIE ; NMDAR=N-methyl-D-aspartate receptor.
The CASE (Clinical Assessment Scale in AIE) includes an assessment of 9 items: seizure, memory dysfunction, psychiatric symptoms, consciousness, language problems, dyskinesia/dystonia, gait instability and ataxia, brainstem dysfunction, weakness.
This overall CASE total score ranges from 0 to 27, with a higher score indicating a greater degree of disability.
|
at week 24
|
|
Incidence and severity of AEs
大体时间：week 24 onwards
|
AEs = Adverse Effects
|
week 24 onwards
|
|
Incidence and severity of SAEs
大体时间：week 24 onwards
|
SAEs = Serious Adverse Effects
|
week 24 onwards
|
|
Proportion of participants with presence of neuropsychiatric symptoms, defined by NPI-C total score of at least 1 point
大体时间：at week 24
|
NPI-C= Neuropsychiatric Inventory-Clinician.
The NPI-C (Neuropsychiatric Inventory--Clinician) total score will be used as a global measure of neuropsychiatric symptoms.
Total score is calculated by summing the scores of all the individual domains.
Each domain score is determined by summing the item scores within that domain.
The NPI-C uses a clinician rating method, where ratings for frequency, severity, and caregiver distress are provided for each item.
|
at week 24
|
|
Change in MoCA total score
大体时间：up to week 24
|
MoCA= Montreal Cognitive Assessment
|
up to week 24
|
|
Proportion of participants with a favorable outcome in mRS where favorable outcome is defined as no worsening for participants with a baseline mRS score of ≤2 or improvement of ≥1 point for participants with a baseline mRS score of >2
大体时间：up to week 24
|
mRS=modified Rankin Scale.
The mRS (modified Rankin Scale) is commonly used to measure the degree of disability or dependence in the daily activities of people with neurological disabilities.
Scores range from 0 (no symptoms) to 6 (dead).
|
up to week 24
|
|
Change in CGI-S
大体时间：up to week 24
|
CGI-S= Clinical Global Impression of Severity.
CGI is a clinician-rated scale that measures illness severity (CGI-S) and global improvement or change (CGI-C).It is rated on a 7-point scale, from 1 (normal) to 7 (amongst the most severely ill patients) for CGI-S and from 1 (very much improved) to 7 (very much worse) for CGI-C.
|
up to week 24
|
|
Change in CGI-C
大体时间：up to week 24
|
CGI-C= Clinical Global Impression of Change.
CGI is a clinician-rated scale that measures illness severity (CGI-S) and global improvement or change (CGI-C).It is rated on a 7-point scale, from 1 (normal) to 7 (amongst the most severely ill patients) for CGI-S and from 1 (very much improved) to 7 (very much worse) for CGI-C.
|
up to week 24
|
|
Change in PGI-C
大体时间：week 0 to week 24
|
PGI-C =Patient Global Expression of Change.
PGI-S and PGI-C are the patient-reported counterparts to CGI-S and CGI-C, respectively.
|
week 0 to week 24
|
|
Change in PGI-S
大体时间：week 0 to week 24
|
PGI-S= Patient Global Expression of Severity.
PGI-S and PGI-C are the patient-reported counterparts to CGI-S and CGI-C, respectively.
|
week 0 to week 24
|
|
Time to resolution of status epilepticus
大体时间：up to 24 weeks
|
up to 24 weeks
|
|
|
Time to first occurrence of seizure freedom.
大体时间：up to 24 weeks
|
Seizure freedom is defined as no seizures for at least 28 consecutive days
|
up to 24 weeks
|
|
Proportion of participants with seizure freedom for at least the 28 consecutive days
大体时间：up to 24 weeks
|
up to 24 weeks
|
|
|
Time to use of rescue therapy after randomization
大体时间：up to 24 weeks
|
up to 24 weeks
|
|
|
Trough efgartigimod serum concentrations over time
大体时间：up to 24 weeks
|
up to 24 weeks
|
|
|
Percent change in total IgG levels in serum
大体时间：up to 24 weeks
|
IgG = Immunoglobulin G
|
up to 24 weeks
|
|
Incidence and prevalence of ADA against efgartigimod in serum over time
大体时间：up to 24 weeks
|
ADA = anti drug antibodies
|
up to 24 weeks
|
|
Incidence and prevalence of antibodies against rHuPH20 in plasma over time
大体时间：up to 24 weeks
|
rHuPH20 = Recombinant Human Hyaluronidase PH20
|
up to 24 weeks
|
|
Change in CASE score in the NMDAR population compared with the non-NMDAR population
大体时间：up to 24 weeks
|
CASE = Clinical Assessment Scale in AIE ; NMDAR=N-methyl-D-aspartate receptor. .
The CASE (Clinical Assessment Scale in AIE) includes an assessment of 9 items: seizure, memory dysfunction, psychiatric symptoms, consciousness, language problems, dyskinesia/dystonia, gait instability and ataxia, brainstem dysfunction, weakness.
This overall CASE total score ranges from 0 to 27, with a higher score indicating a greater degree of disability.
|
up to 24 weeks
|
|
Change in RBANS total score
大体时间：week 24 to week 48
|
The RBANS (Repeatable Battery for the Assessment of Neuropsychological Status) is a performance outcome measure developed to identify and characterize cognitive impairment by assessing an individual's current level of cognitive performance.
|
week 24 to week 48
|
|
Percentage of participants with maintained change in the CASE total score (defined as stable or improving)
大体时间：week 24 to week 48
|
The CASE (Clinical Assessment Scale in AIE) includes an assessment of 9 items: seizure, memory dysfunction, psychiatric symptoms, consciousness, language problems, dyskinesia/dystonia, gait instability and ataxia, brainstem dysfunction, weakness.
This overall CASE total score ranges from 0 to 27, with a higher score indicating a greater degree of disability.
|
week 24 to week 48
|
|
Percentage of participants with maintained mRS score (defined as stable or improving)
大体时间：week 24 to week 48
|
The mRS (modified Rankin Scale) is commonly used to measure the degree of disability or dependence in the daily activities of people with neurological disabilities.
Scores range from 0 (no symptoms) to 6 (dead).
|
week 24 to week 48
|
|
Change in NPI-C total score
大体时间：week 24 to week 48
|
The NPI-C (Neuropsychiatric Inventory--Clinician) total score will be used as a global measure of neuropsychiatric symptoms.Total score is calculated by summing the scores of all the individual domains.
Each domain score is determined by summing the item scores within that domain.
The NPI-C uses a clinician rating method, where ratings for frequency, severity, and caregiver distress are provided for each item.
These item scores are then summed to create a total domain score.
|
week 24 to week 48
|
|
Proportion of participants requiring rescue or second-line AIE therapies
大体时间：week 24 to week 48
|
AIE = Auto-Immune Encephalitis
|
week 24 to week 48
|
|
Time to participants requiring rescue or second-line AIE therapies
大体时间：week 24 to week 48
|
AIE = Auto-Immune Encephalitis
|
week 24 to week 48
|
|
Change in CASE
大体时间：week 24 to week 48
|
The CASE (Clinical Assessment Scale in AIE) includes an assessment of 9 items: seizure, memory dysfunction, psychiatric symptoms, consciousness, language problems, dyskinesia/dystonia, gait instability and ataxia, brainstem dysfunction, weakness.
This overall CASE total score ranges from 0 to 27, with a higher score indicating a greater degree of disability.
|
week 24 to week 48
|
|
Change in mRs
大体时间：week 24 to week 48
|
The mRS (modified Rankin Scale) is commonly used to measure the degree of disability or dependence in the daily activities of people with neurological disabilities.
Scores range from 0 (no symptoms) to 6 (dead).
|
week 24 to week 48
|
|
Change in RBANS
大体时间：week 24 to week 48
|
The RBANS (Repeatable Battery for the Assessment of Neuropsychological Status) is a performance outcome measure developed to identify and characterize cognitive impairment by assessing an individual's current level of cognitive performance.
|
week 24 to week 48
|
|
Time to resolution of status epilepticus
大体时间：week 24 to week 48
|
week 24 to week 48
|
|
|
Proportion of participants with seizure freedom for at least the 28 consecutive days preceding the participants in final Part of trial
大体时间：week 24 to week 48
|
mRS=modified Rankin Scale
|
week 24 to week 48
|
|
Incidence and prevalence of ADA against efgartigimod in serum
大体时间：week 24 to week 48
|
ADA = antidrug antibody(ies)
|
week 24 to week 48
|
|
Percent change in total IgG levels in serum
大体时间：week 24 to week 48
|
IgG = Immunoglobulin G
|
week 24 to week 48
|
合作者和调查者
在这里您可以找到参与这项研究的人员和组织。
赞助
研究记录日期
这些日期跟踪向 ClinicalTrials.gov 提交研究记录和摘要结果的进度。研究记录和报告的结果由国家医学图书馆 (NLM) 审查，以确保它们在发布到公共网站之前符合特定的质量控制标准。
研究主要日期
学习开始 (估计的)
2026年8月15日
初级完成 (估计的)
2029年1月20日
研究完成 (估计的)
2029年5月12日
研究注册日期
首次提交
2026年5月7日
首先提交符合 QC 标准的
2026年5月7日
首次发布 (实际的)
2026年5月13日
研究记录更新
最后更新发布 (实际的)
2026年8月24日
上次提交的符合 QC 标准的更新
2026年8月21日
最后验证
2026年5月1日
更多信息
与本研究相关的术语
其他相关的 MeSH 术语
其他研究编号
- ARGX-113-22-AIE-2001
计划个人参与者数据 (IPD)
计划共享个人参与者数据 (IPD)？
不
药物和器械信息、研究文件
研究美国 FDA 监管的药品
是的
研究美国 FDA 监管的设备产品
不
此信息直接从 clinicaltrials.gov 网站检索，没有任何更改。如果您有任何更改、删除或更新研究详细信息的请求，请联系 register@clinicaltrials.gov. clinicaltrials.gov 上实施更改，我们的网站上也会自动更新.