A Study to Learn About How Well the Medicine Efgartigimod Works to Treat Autoimmune Encephalitis In Children 12 Years or Older and Adults (Polaris)
21 août 2026 mis à jour par: 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
Aperçu de l'étude
Statut
Recrutement
Les conditions
Intervention / Traitement
Description détaillée
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.
Type d'étude
Interventionnel
Inscription (Estimé)
170
Phase
- Phase 2
Contacts et emplacements
Cette section fournit les coordonnées de ceux qui mènent l'étude et des informations sur le lieu où cette étude est menée.
Coordonnées de l'étude
- Nom: Sabine Coppieters, MD
- Numéro de téléphone: 857-350-4834
- E-mail: clinicaltrials@argenx.com
Lieux d'étude
-
-
Arizona
-
Tucson, Arizona, États-Unis, 85718
- Recrutement
- Center For Neurosciences
-
Chercheur principal:
- Francisco Valdivia
-
Contact:
- Gwendolyn Grabiec
- Numéro de téléphone: (520) 529-5234
- E-mail: ggrabiec@neurotucson.com
-
-
Texas
-
Sherman, Texas, États-Unis, 75092-7371
- Recrutement
- Texas Institute for Neurological Disorders
-
Chercheur principal:
- Bharathy Sundaram
-
Contact:
- Laurie Laurie
- Numéro de téléphone: (903) 893-5141 x4358
- E-mail: ljarvis@texasneurologyinstitute.com
-
-
Critères de participation
Les chercheurs recherchent des personnes qui correspondent à une certaine description, appelée critères d'éligibilité. Certains exemples de ces critères sont l'état de santé général d'une personne ou des traitements antérieurs.
Critère d'éligibilité
Âges éligibles pour étudier
- Enfant
- Adulte
- Adulte plus âgé
Accepte les volontaires sains
Non
La description
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.
Plan d'étude
Cette section fournit des détails sur le plan d'étude, y compris la façon dont l'étude est conçue et ce que l'étude mesure.
Comment l'étude est-elle conçue ?
Détails de conception
- Objectif principal: Traitement
- Répartition: Randomisé
- Modèle interventionnel: Affectation parallèle
- Masquage: Tripler
Armes et Interventions
Groupe de participants / Bras |
Intervention / Traitement |
|---|---|
|
Expérimental: 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.
|
|
Expérimental: 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.
|
|
Comparateur 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 |
|
Expérimental: 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.
|
Que mesure l'étude ?
Principaux critères de jugement
Mesure des résultats |
Description de la mesure |
Délai |
|---|---|---|
|
Change in CASE score in the NMDAR population
Délai: 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
|
Mesures de résultats secondaires
Mesure des résultats |
Description de la mesure |
Délai |
|---|---|---|
|
Change in mRS
Délai: 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
Délai: 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
Délai: up to week 8
|
MoCA= Montreal Cognitive Assessment
|
up to week 8
|
|
Change in NPI-C total score
Délai: 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
Délai: 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
Délai: 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
Délai: 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
Délai: 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
Délai: up to week 8
|
AEs= Adverse Effects
|
up to week 8
|
|
Incidence and severity of SAEs
Délai: up to week 8
|
SAEs = Serious Adverse Effects
|
up to week 8
|
|
Trough efgartigimod serum concentrations over time
Délai: up to week 8
|
up to week 8
|
|
|
Percent change from baseline in total IgG levels in serum over time
Délai: up to week 8
|
IgG= Immunoglobulin G
|
up to week 8
|
|
Incidence and prevalence of ADA against efgartigimod in serum over time
Délai: up to week 8
|
ADA = antidrug antibody(ies)
|
up to week 8
|
|
Incidence and prevalence of antibodies against rHuPH20 in plasma over time
Délai: up to week 8
|
rHuPH20 = Recombinant Human Hyaluronidase PH20
|
up to week 8
|
|
Change in mRS in the NMDAR population
Délai: 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
Délai: 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
Délai: 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.
Délai: 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
Délai: 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
Délai: 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
Délai: 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
Délai: 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.
Délai: 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
Délai: week 24 onwards
|
AEs = Adverse Effects
|
week 24 onwards
|
|
Incidence and severity of SAEs
Délai: 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
Délai: 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
Délai: 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
Délai: 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
Délai: 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
Délai: 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
Délai: 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
Délai: 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
Délai: up to 24 weeks
|
up to 24 weeks
|
|
|
Time to first occurrence of seizure freedom.
Délai: 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
Délai: up to 24 weeks
|
up to 24 weeks
|
|
|
Time to use of rescue therapy after randomization
Délai: up to 24 weeks
|
up to 24 weeks
|
|
|
Trough efgartigimod serum concentrations over time
Délai: up to 24 weeks
|
up to 24 weeks
|
|
|
Percent change in total IgG levels in serum
Délai: up to 24 weeks
|
IgG = Immunoglobulin G
|
up to 24 weeks
|
|
Incidence and prevalence of ADA against efgartigimod in serum over time
Délai: up to 24 weeks
|
ADA = anti drug antibodies
|
up to 24 weeks
|
|
Incidence and prevalence of antibodies against rHuPH20 in plasma over time
Délai: 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
Délai: 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
Délai: 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)
Délai: 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)
Délai: 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
Délai: 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
Délai: week 24 to week 48
|
AIE = Auto-Immune Encephalitis
|
week 24 to week 48
|
|
Time to participants requiring rescue or second-line AIE therapies
Délai: week 24 to week 48
|
AIE = Auto-Immune Encephalitis
|
week 24 to week 48
|
|
Change in CASE
Délai: 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
Délai: 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
Délai: 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
Délai: 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
Délai: week 24 to week 48
|
mRS=modified Rankin Scale
|
week 24 to week 48
|
|
Incidence and prevalence of ADA against efgartigimod in serum
Délai: week 24 to week 48
|
ADA = antidrug antibody(ies)
|
week 24 to week 48
|
|
Percent change in total IgG levels in serum
Délai: week 24 to week 48
|
IgG = Immunoglobulin G
|
week 24 to week 48
|
Collaborateurs et enquêteurs
C'est ici que vous trouverez les personnes et les organisations impliquées dans cette étude.
Parrainer
Dates d'enregistrement des études
Ces dates suivent la progression des dossiers d'étude et des soumissions de résultats sommaires à ClinicalTrials.gov. Les dossiers d'étude et les résultats rapportés sont examinés par la Bibliothèque nationale de médecine (NLM) pour s'assurer qu'ils répondent à des normes de contrôle de qualité spécifiques avant d'être publiés sur le site Web public.
Dates principales de l'étude
Début de l'étude (Estimé)
15 août 2026
Achèvement primaire (Estimé)
20 janvier 2029
Achèvement de l'étude (Estimé)
12 mai 2029
Dates d'inscription aux études
Première soumission
7 mai 2026
Première soumission répondant aux critères de contrôle qualité
7 mai 2026
Première publication (Réel)
13 mai 2026
Mises à jour des dossiers d'étude
Dernière mise à jour publiée (Réel)
24 août 2026
Dernière mise à jour soumise répondant aux critères de contrôle qualité
21 août 2026
Dernière vérification
1 mai 2026
Plus d'information
Termes liés à cette étude
Mots clés
Termes MeSH pertinents supplémentaires
Autres numéros d'identification d'étude
- ARGX-113-22-AIE-2001
Plan pour les données individuelles des participants (IPD)
Prévoyez-vous de partager les données individuelles des participants (DPI) ?
NON
Informations sur les médicaments et les dispositifs, documents d'étude
Étudie un produit pharmaceutique réglementé par la FDA américaine
Oui
Étudie un produit d'appareil réglementé par la FDA américaine
Non
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