- ICH GCP
- Registro degli studi clinici negli Stati Uniti
- Sperimentazione clinica NCT07583641
A Study to Learn About How Well the Medicine Efgartigimod Works to Treat Autoimmune Encephalitis In Children 12 Years or Older and Adults (Polaris)
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
Condizioni
Intervento / Trattamento
Descrizione dettagliata
Tipo di studio
Iscrizione (Stimato)
Fase
- Fase 2
Contatti e Sedi
Contatto studio
- Nome: Sabine Coppieters, MD
- Numero di telefono: 857-350-4834
- Email: clinicaltrials@argenx.com
Criteri di partecipazione
Criteri di ammissibilità
Età idonea allo studio
- Bambino
- Adulto
- Adulto più anziano
Accetta volontari sani
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
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
Sponsor
Studiare le date dei record
Studia le date principali
Inizio studio (Stimato)
Completamento primario (Stimato)
Completamento dello studio (Stimato)
Date di iscrizione allo studio
Primo inviato
Primo inviato che soddisfa i criteri di controllo qualità
Primo Inserito (Effettivo)
Aggiornamenti dei record di studio
Ultimo aggiornamento pubblicato (Effettivo)
Ultimo aggiornamento inviato che soddisfa i criteri QC
Ultimo verificato
Maggiori informazioni
Termini relativi a questo studio
Parole chiave
Termini MeSH pertinenti aggiuntivi
Altri numeri di identificazione dello studio
- ARGX-113-22-AIE-2001
Piano per i dati dei singoli partecipanti (IPD)
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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 .
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