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

7. maj 2026 opdateret af: 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

Studieoversigt

Status

Ikke rekrutterer endnu

Betingelser

Intervention / Behandling

Detaljeret beskrivelse

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.

Undersøgelsestype

Interventionel

Tilmelding (Anslået)

170

Fase

  • Fase 2

Kontakter og lokationer

Dette afsnit indeholder kontaktoplysninger for dem, der udfører undersøgelsen, og oplysninger om, hvor denne undersøgelse udføres.

Studiekontakt

Deltagelseskriterier

Forskere leder efter personer, der passer til en bestemt beskrivelse, kaldet berettigelseskriterier. Nogle eksempler på disse kriterier er en persons generelle helbredstilstand eller tidligere behandlinger.

Berettigelseskriterier

Aldre berettiget til at studere

  • Barn
  • Voksen
  • Ældre voksen

Tager imod sunde frivillige

Ingen

Beskrivelse

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.

Studieplan

Dette afsnit indeholder detaljer om studieplanen, herunder hvordan undersøgelsen er designet, og hvad undersøgelsen måler.

Hvordan er undersøgelsen tilrettelagt?

Design detaljer

  • Primært formål: Behandling
  • Tildeling: Randomiseret
  • Interventionel model: Parallel tildeling
  • Maskning: Tredobbelt

Våben og indgreb

Deltagergruppe / Arm
Intervention / Behandling
Eksperimentel: Part A (Open-Label Lead-in Period): Efgartigimod PH20 SC
All participants will receive efgartigimod PH20 SC open label for 8 weeks
Biologisk: 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.
Eksperimentel: Part B (Double-blinded treatment period): Efgartigimod PH20 SC
Participants will receive efgartigimod PH20 SC for 24 weeks
Biologisk: 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.
Placebo komparator: Part B (Maintenance double-blinded treatment period): Placebo PH20 SC
Participants will receive placebo for 24 weeks
Andet: 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
Eksperimentel: Part C (Open-Label Extension Period): Efgartigimod PH20 SC
Participants who complete Part B will receive efgartigimod PH20 SC for 24 weeks
Biologisk: 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.

Hvad måler undersøgelsen?

Primære resultatmål

Resultatmål
Foranstaltningsbeskrivelse
Tidsramme
Change in CASE score in the NMDAR population
Tidsramme: 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

Sekundære resultatmål

Resultatmål
Foranstaltningsbeskrivelse
Tidsramme
Change in mRS
Tidsramme: 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
Tidsramme: 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
Tidsramme: up to week 8
MoCA= Montreal Cognitive Assessment
up to week 8
Change in NPI-C total score
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: up to week 8
AEs= Adverse Effects
up to week 8
Incidence and severity of SAEs
Tidsramme: up to week 8
SAEs = Serious Adverse Effects
up to week 8
Trough efgartigimod serum concentrations over time
Tidsramme: up to week 8
up to week 8
Percent change from baseline in total IgG levels in serum over time
Tidsramme: up to week 8
IgG= Immunoglobulin G
up to week 8
Incidence and prevalence of ADA against efgartigimod in serum over time
Tidsramme: up to week 8
ADA = antidrug antibody(ies)
up to week 8
Incidence and prevalence of antibodies against rHuPH20 in plasma over time
Tidsramme: up to week 8
rHuPH20 = Recombinant Human Hyaluronidase PH20
up to week 8
Change in mRS in the NMDAR population
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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.
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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.
Tidsramme: 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
Tidsramme: week 24 onwards
AEs = Adverse Effects
week 24 onwards
Incidence and severity of SAEs
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: up to 24 weeks
up to 24 weeks
Time to first occurrence of seizure freedom.
Tidsramme: 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
Tidsramme: up to 24 weeks
up to 24 weeks
Time to use of rescue therapy after randomization
Tidsramme: up to 24 weeks
up to 24 weeks
Trough efgartigimod serum concentrations over time
Tidsramme: up to 24 weeks
up to 24 weeks
Percent change in total IgG levels in serum
Tidsramme: up to 24 weeks
IgG = Immunoglobulin G
up to 24 weeks
Incidence and prevalence of ADA against efgartigimod in serum over time
Tidsramme: up to 24 weeks
ADA = anti drug antibodies
up to 24 weeks
Incidence and prevalence of antibodies against rHuPH20 in plasma over time
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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)
Tidsramme: 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)
Tidsramme: 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
Tidsramme: 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
Tidsramme: week 24 to week 48
AIE = Auto-Immune Encephalitis
week 24 to week 48
Time to participants requiring rescue or second-line AIE therapies
Tidsramme: week 24 to week 48
AIE = Auto-Immune Encephalitis
week 24 to week 48
Change in CASE
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: week 24 to week 48
mRS=modified Rankin Scale
week 24 to week 48
Incidence and prevalence of ADA against efgartigimod in serum
Tidsramme: week 24 to week 48
ADA = antidrug antibody(ies)
week 24 to week 48
Percent change in total IgG levels in serum
Tidsramme: week 24 to week 48
IgG = Immunoglobulin G
week 24 to week 48

Samarbejdspartnere og efterforskere

Det er her, du vil finde personer og organisationer, der er involveret i denne undersøgelse.

Sponsor

argenx

Datoer for undersøgelser

Disse datoer sporer fremskridtene for indsendelser af undersøgelsesrekord og resumeresultater til ClinicalTrials.gov. Studieregistreringer og rapporterede resultater gennemgås af National Library of Medicine (NLM) for at sikre, at de opfylder specifikke kvalitetskontrolstandarder, før de offentliggøres på den offentlige hjemmeside.

Studer store datoer

Studiestart (Anslået)

13. juli 2026

Primær færdiggørelse (Anslået)

4. december 2030

Studieafslutning (Anslået)

26. juli 2031

Datoer for studieregistrering

Først indsendt

7. maj 2026

Først indsendt, der opfyldte QC-kriterier

7. maj 2026

Først opslået (Faktiske)

13. maj 2026

Opdateringer af undersøgelsesjournaler

Sidste opdatering sendt (Faktiske)

13. maj 2026

Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier

7. maj 2026

Sidst verificeret

1. maj 2026

Mere information

Begreber relateret til denne undersøgelse

Nøgleord

Yderligere relevante MeSH-vilkår

Andre undersøgelses-id-numre

  • ARGX-113-22-AIE-2001

Plan for individuelle deltagerdata (IPD)

Planlægger du at dele individuelle deltagerdata (IPD)?

INGEN

Lægemiddel- og udstyrsoplysninger, undersøgelsesdokumenter

Studerer et amerikansk FDA-reguleret lægemiddelprodukt

Ja

Studerer et amerikansk FDA-reguleret enhedsprodukt

Ingen

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Kliniske forsøg med Autoimmun Encephalitis (AE)

Kliniske forsøg med Efgartigimod PH20 (ARGX-113) SC

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