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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 maggio 2026 aggiornato da: 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

Panoramica dello studio

Stato

Non ancora reclutamento

Descrizione dettagliata

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.

Tipo di studio

Interventistico

Iscrizione (Stimato)

170

Fase

  • Fase 2

Contatti e Sedi

Questa sezione fornisce i recapiti di coloro che conducono lo studio e informazioni su dove viene condotto lo studio.

Contatto studio

Criteri di partecipazione

I ricercatori cercano persone che corrispondano a una certa descrizione, chiamata criteri di ammissibilità. Alcuni esempi di questi criteri sono le condizioni generali di salute di una persona o trattamenti precedenti.

Criteri di ammissibilità

Età idonea allo studio

  • Bambino
  • Adulto
  • Adulto più anziano

Accetta volontari sani

No

Descrizione

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.

Piano di studio

Questa sezione fornisce i dettagli del piano di studio, compreso il modo in cui lo studio è progettato e ciò che lo studio sta misurando.

Come è strutturato lo studio?

Dettagli di progettazione

  • Scopo principale: Trattamento
  • Assegnazione: Randomizzato
  • Modello interventistico: Assegnazione parallela
  • Mascheramento: Triplicare

Armi e interventi

Gruppo di partecipanti / Arm
Intervento / Trattamento
Sperimentale: 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.
Sperimentale: 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.
Comparatore placebo: 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

Sperimentale: 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.

Cosa sta misurando lo studio?

Misure di risultato primarie

Misura del risultato
Misura Descrizione
Lasso di tempo
Change in CASE score in the NMDAR population
Lasso di tempo: 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

Misure di risultato secondarie

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

Collaboratori e investigatori

Qui è dove troverai le persone e le organizzazioni coinvolte in questo studio.

Sponsor

Studiare le date dei record

Queste date tengono traccia dell'avanzamento della registrazione dello studio e dell'invio dei risultati di sintesi a ClinicalTrials.gov. I record degli studi e i risultati riportati vengono esaminati dalla National Library of Medicine (NLM) per assicurarsi che soddisfino specifici standard di controllo della qualità prima di essere pubblicati sul sito Web pubblico.

Studia le date principali

Inizio studio (Stimato)

13 luglio 2026

Completamento primario (Stimato)

4 dicembre 2030

Completamento dello studio (Stimato)

26 luglio 2031

Date di iscrizione allo studio

Primo inviato

7 maggio 2026

Primo inviato che soddisfa i criteri di controllo qualità

7 maggio 2026

Primo Inserito (Effettivo)

13 maggio 2026

Aggiornamenti dei record di studio

Ultimo aggiornamento pubblicato (Effettivo)

13 maggio 2026

Ultimo aggiornamento inviato che soddisfa i criteri QC

7 maggio 2026

Ultimo verificato

1 maggio 2026

Maggiori informazioni

Termini relativi a questo studio

Altri numeri di identificazione dello studio

  • ARGX-113-22-AIE-2001

Piano per i dati dei singoli partecipanti (IPD)

Hai intenzione di condividere i dati dei singoli partecipanti (IPD)?

NO

Informazioni su farmaci e dispositivi, documenti di studio

Studia un prodotto farmaceutico regolamentato dalla FDA degli Stati Uniti

Studia un dispositivo regolamentato dalla FDA degli Stati Uniti

No

Queste informazioni sono state recuperate direttamente dal sito web clinicaltrials.gov senza alcuna modifica. In caso di richieste di modifica, rimozione o aggiornamento dei dettagli dello studio, contattare register@clinicaltrials.gov. Non appena verrà implementata una modifica su clinicaltrials.gov, questa verrà aggiornata automaticamente anche sul nostro sito web .

Prove cliniche su Encefalite Autoimmune (EA)

Prove cliniche su Efgartigimod PH20 (ARGX-113) SC

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