- ICH GCP
- 미국 임상 시험 레지스트리
- 임상시험 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
연구 개요
상세 설명
연구 유형
등록 (추정된)
단계
- 2 단계
연락처 및 위치
연구 연락처
- 이름: Sabine Coppieters, MD
- 전화번호: 857-350-4834
- 이메일: clinicaltrials@argenx.com
참여기준
자격 기준
공부할 수 있는 나이
- 어린이
- 성인
- 고령자
건강한 자원 봉사자를 받아들입니다
설명
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.
공부 계획
연구는 어떻게 설계됩니까?
디자인 세부사항
- 주 목적: 치료
- 할당: 무작위
- 중재 모델: 병렬 할당
- 마스킹: 삼루타
무기와 개입
참가자 그룹 / 팔 |
개입 / 치료 |
|---|---|
|
실험적: 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.
|
|
실험적: 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.
|
|
위약 비교기: 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 |
|
실험적: 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.
|
연구는 무엇을 측정합니까?
주요 결과 측정
결과 측정 |
측정값 설명 |
기간 |
|---|---|---|
|
Change in CASE score in the NMDAR population
기간: 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
|
2차 결과 측정
결과 측정 |
측정값 설명 |
기간 |
|---|---|---|
|
Change in mRS
기간: 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
기간: 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
기간: up to week 8
|
MoCA= Montreal Cognitive Assessment
|
up to week 8
|
|
Change in NPI-C total score
기간: 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
기간: 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
기간: 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
기간: 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
기간: 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
기간: up to week 8
|
AEs= Adverse Effects
|
up to week 8
|
|
Incidence and severity of SAEs
기간: up to week 8
|
SAEs = Serious Adverse Effects
|
up to week 8
|
|
Trough efgartigimod serum concentrations over time
기간: up to week 8
|
up to week 8
|
|
|
Percent change from baseline in total IgG levels in serum over time
기간: up to week 8
|
IgG= Immunoglobulin G
|
up to week 8
|
|
Incidence and prevalence of ADA against efgartigimod in serum over time
기간: up to week 8
|
ADA = antidrug antibody(ies)
|
up to week 8
|
|
Incidence and prevalence of antibodies against rHuPH20 in plasma over time
기간: up to week 8
|
rHuPH20 = Recombinant Human Hyaluronidase PH20
|
up to week 8
|
|
Change in mRS in the NMDAR population
기간: 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
기간: 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
기간: 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.
기간: 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
기간: 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
기간: 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
기간: 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
기간: 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.
기간: 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
기간: week 24 onwards
|
AEs = Adverse Effects
|
week 24 onwards
|
|
Incidence and severity of SAEs
기간: 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
기간: 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
기간: 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
기간: 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
기간: 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
기간: 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
기간: 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
기간: 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
기간: up to 24 weeks
|
up to 24 weeks
|
|
|
Time to first occurrence of seizure freedom.
기간: 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
기간: up to 24 weeks
|
up to 24 weeks
|
|
|
Time to use of rescue therapy after randomization
기간: up to 24 weeks
|
up to 24 weeks
|
|
|
Trough efgartigimod serum concentrations over time
기간: up to 24 weeks
|
up to 24 weeks
|
|
|
Percent change in total IgG levels in serum
기간: up to 24 weeks
|
IgG = Immunoglobulin G
|
up to 24 weeks
|
|
Incidence and prevalence of ADA against efgartigimod in serum over time
기간: up to 24 weeks
|
ADA = anti drug antibodies
|
up to 24 weeks
|
|
Incidence and prevalence of antibodies against rHuPH20 in plasma over time
기간: 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
기간: 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
기간: 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)
기간: 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)
기간: 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
기간: 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
기간: week 24 to week 48
|
AIE = Auto-Immune Encephalitis
|
week 24 to week 48
|
|
Time to participants requiring rescue or second-line AIE therapies
기간: week 24 to week 48
|
AIE = Auto-Immune Encephalitis
|
week 24 to week 48
|
|
Change in CASE
기간: 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
기간: 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
기간: 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
기간: 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
기간: week 24 to week 48
|
mRS=modified Rankin Scale
|
week 24 to week 48
|
|
Incidence and prevalence of ADA against efgartigimod in serum
기간: week 24 to week 48
|
ADA = antidrug antibody(ies)
|
week 24 to week 48
|
|
Percent change in total IgG levels in serum
기간: week 24 to week 48
|
IgG = Immunoglobulin G
|
week 24 to week 48
|
공동 작업자 및 조사자
스폰서
연구 기록 날짜
연구 주요 날짜
연구 시작 (추정된)
기본 완료 (추정된)
연구 완료 (추정된)
연구 등록 날짜
최초 제출
QC 기준을 충족하는 최초 제출
처음 게시됨 (실제)
연구 기록 업데이트
마지막 업데이트 게시됨 (실제)
QC 기준을 충족하는 마지막 업데이트 제출
마지막으로 확인됨
추가 정보
이 연구와 관련된 용어
추가 관련 MeSH 약관
기타 연구 ID 번호
- ARGX-113-22-AIE-2001
개별 참가자 데이터(IPD) 계획
개별 참가자 데이터(IPD)를 공유할 계획입니까?
약물 및 장치 정보, 연구 문서
미국 FDA 규제 의약품 연구
미국 FDA 규제 기기 제품 연구
이 정보는 변경 없이 clinicaltrials.gov 웹사이트에서 직접 가져온 것입니다. 귀하의 연구 세부 정보를 변경, 제거 또는 업데이트하도록 요청하는 경우 register@clinicaltrials.gov. 문의하십시오. 변경 사항이 clinicaltrials.gov에 구현되는 즉시 저희 웹사이트에도 자동으로 업데이트됩니다. .
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Efgartigimod PH20 (ARGX-113) SC에 대한 임상 시험
-
argenx모집하지 않고 적극적으로원발성 면역 혈소판 감소증그루지야, 일본, 폴란드, 미국, 아르헨티나, 호주, 불가리아, 칠레, 중국, 아일랜드, 이탈리아, 요르단, 포르투갈, 루마니아, 태국, 튀니지, 그리스, 멕시코, 뉴질랜드, 노르웨이, 남아프리카, 영국, 터키 (Türkiye), 러시아 제국, 대한민국
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argenx완전한원발성 면역 혈소판 감소증미국, 프랑스, 그루지야, 독일, 이탈리아, 일본, 폴란드, 러시아 연방, 스페인, 칠면조, 영국, 아르헨티나, 호주, 불가리아, 칠레, 중국, 덴마크, 그리스, 아일랜드, 이스라엘, 요르단, 대한민국, 멕시코, 뉴질랜드, 노르웨이, 포르투갈, 루마니아, 세르비아, 남아프리카, 대만, 태국, 튀니지
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argenx모집하지 않고 적극적으로만성 염증성 탈수초성 다발신경병증(CIDP)미국, 오스트리아, 벨기에, 불가리아, 중국, 체코, 덴마크, 프랑스, 그루지야, 독일, 이스라엘, 이탈리아, 일본, 네덜란드, 폴란드, 러시아 연방, 세르비아, 스페인, 우크라이나, 영국, 라트비아, 루마니아, 대만, 칠면조
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argenx완전한원발성 면역 혈소판 감소증미국, 오스트리아, 벨기에, 불가리아, 체코, 프랑스, 그루지야, 독일, 헝가리, 이탈리아, 일본, 네덜란드, 폴란드, 스페인, 영국, 우크라이나, 러시아 제국, 터키 (Türkiye)
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argenx모병만성 염증성 탈수초성 다발신경근병증 | CIDP | CIDP(만성 염증성 탈수초성 다발신경병증)독일, 미국
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argenx완전한
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argenx초대로 등록전신 중증 근무력증 | gMG벨기에, 독일, 스페인, 네덜란드, 폴란드, 이스라엘, 미국, 영국, 프랑스, 캐나다
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argenx모병항체 매개 거부반응미국, 독일, 스페인, 체코, 프랑스, 캐나다, 벨기에, 오스트리아