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
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 发起方
- argenx
- 入组人数
- 170
- 试验地点
- 3
研究概览
简要总结
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
详细描述
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Triple (Participant, Care Provider, Investigator)
入排标准
- 年龄范围
- 12 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Is at least 12 years of age.
- •Meeting at least the criteria for possible AIE according to the Graus criteria.
- •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
排除标准
- •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.
研究组 & 干预措施
Part A (Open-Label Lead-in Period): Efgartigimod PH20 SC
All participants will receive efgartigimod PH20 SC open label for 8 weeks
干预措施: Efgartigimod PH20 (ARGX-113) SC (Biological)
Part B (Double-blinded treatment period): Efgartigimod PH20 SC
Participants will receive efgartigimod PH20 SC for 24 weeks
干预措施: Efgartigimod PH20 (ARGX-113) SC (Biological)
Part B (Maintenance double-blinded treatment period): Placebo PH20 SC
Participants will receive placebo for 24 weeks
干预措施: Placebo PH20 SC (Other)
Part C (Open-Label Extension Period): Efgartigimod PH20 SC
Participants who complete Part B will receive efgartigimod PH20 SC for 24 weeks
干预措施: Efgartigimod PH20 (ARGX-113) SC (Biological)