A Study to Learn About How Well the Medicine Efgartigimod Works to Treat Autoimmune Encephalitis In Children 12 Years or Older and Adults

Официальное название

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

Обзор

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

Подробное описание

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.

Вмешательства

• Биопрепарат Efgartigimod PH20 (ARGX-113) SC
  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.
• Другое Placebo PH20 SC
  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

Первичные конечные точки

• Change in CASE score in the NMDAR population [Срок оценки: up to week 24]

Вторичные конечные точки (12)

• Change in mRS [Срок оценки: up to week 8]
• Change in CASE score [Срок оценки: up to week 8]
• Change in MoCA total score [Срок оценки: up to week 8]
• Change in NPI-C total score [Срок оценки: up to week 8]
• Change from baseline in CGI-S [Срок оценки: up to week 8]
• Change from baseline in PGI-S [Срок оценки: up to week 8]
• Change from baseline in CGI-C [Срок оценки: up to week 8]
• Change from baseline in PGI-C [Срок оценки: up to week 8]
• Incidence and severity of AEs [Срок оценки: up to week 8]
• Incidence and severity of SAEs [Срок оценки: up to week 8]
• Trough efgartigimod serum concentrations over time [Срок оценки: up to week 8]
• Percent change from baseline in total IgG levels in serum over time [Срок оценки: up to week 8]

Критерии участия

Критерии включения

• 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

Критерии исключения

• 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.

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Рандомизированное

Модель

Параллельные группы

Маскирование

Тройное слепое

Основная цель

Лечение

Центры проведения

Список центров уточняется — проверьте первичный протокол.

Идентификаторы

NCT: NCT07583641 · ARGX-113-22-AIE-2001

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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