전신 중증 근무력증이 있는 2세~18세 미만 소아의 Nipocalimab에 대한 연구
전신 중증 근무력증이 있는 2세~18세 미만 소아에서 니포칼리맙의 약동학, 약력학, 안전성 및 활성을 평가하기 위한 공개 라벨 비통제 다기관 연구
The purpose of this study is to determine the effect of nipocalimab on total serum immunoglobulin G (IgG) in pediatric participants 2 to less than (<) 18 years of age (globally) and 8 to <18 years of age (for Unites Stated (US) sites only), the safety and tolerability of treatment with nipocalimab in children and adolescents and to evaluate the pharmacokinetics (PK) of nipocalimab in children and adolescents with generalized myasthenia gravis (gMG) who have an insufficient clinical response to ongoing, stable standard-of-care therapy.
연구 개요
연구 유형
등록 (추정된)
단계
- 2 단계
- 3단계
확장된 액세스
연락처 및 위치
연구 연락처
- 이름: Study Contact
- 전화번호: 844-434-4210
- 이메일: Participate-In-This-Study1@its.jnj.com
연구 장소
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Leiden, 네덜란드, 2333 ZA
- 모병
- Leiden University Medical Center
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Arizona
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Phoenix, Arizona, 미국, 85016
- 모병
- Phoenix Children's Hospital
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California
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Los Angeles, California, 미국, 90027
- 완전한
- Childrens Hospital Los Angeles
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Palo Alto, California, 미국, 94304
- 모병
- Lucile Packard Children's Hospital Stanford
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San Francisco, California, 미국, 94158
- 모병
- UCSF Benioff Children's Hospital
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Colorado
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Aurora, Colorado, 미국, 80045
- 모병
- Children's Hospital Colorado
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Florida
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Tampa, Florida, 미국, 33613
- 모병
- University of South Florida Morsani Center for Advanced Healthcare
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Kansas
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Lawrence, Kansas, 미국, 66045
- 완전한
- University of Kansas Medical Center
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Michigan
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Ann Arbor, Michigan, 미국, 48109
- 모병
- C.S. Mott Children's Hospital
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Pennsylvania
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Hershey, Pennsylvania, 미국, 17033
- 모병
- Penn State Milton S Hershey Medical Ctr
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Philadelphia, Pennsylvania, 미국, 19106
- 모병
- Childrens Hospital Of Philadelphia
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Pittsburgh, Pennsylvania, 미국, 15224
- 종료됨
- University of Pittsburgh Medical Center
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Azumino-shi, 일본, 399-8288
- 모병
- Nagano Children's Hospital
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Chiba, 일본, 260 8677
- 완전한
- Chiba University Hospital
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Miyazaki, 일본, 889-1692
- 모병
- University of Miyazaki Hospital
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Nishinomiya-Shi, 일본, 663-8501
- 모병
- Hyogo College of Medicine Hospital
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Saitama Shi, 일본, 330-8777
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- Saitama Prefecture Children's Medical Center
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Shinjuku-ku, 일본, 162-8666
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- Tokyo Women's Medical University Hospital
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Gdansk, 폴란드, 80 211
- 모병
- Uniwersyteckie Centrum Kliniczne
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참여기준
자격 기준
공부할 수 있는 나이
건강한 자원 봉사자를 받아들입니다
연구 대상 성별
설명
Key Inclusion Criteria:
- Age: For US sites only: 8 to < 18 years
- Diagnosis of myasthenia gravis (MG) with generalized muscle weakness meeting the clinical criteria for generalized myasthenia gravis (gMG) as defined by the Myasthenia Gravis Foundation of America (MGFA) Clinical Classification Class IIa/b, IIIa/b, or IVa/b at screening
- Has a positive serologic test for acetylcholine receptor (anti-AChR) antibodies or muscle-specific tyrosine kinase (anti-MuSK) antibodies at screening
- A participant using herbal, naturopathic, traditional Chinese remedies, ayurvedic or nutritional supplements, or medical marijuana (with a doctor's prescription) is eligible if the use of these medications is acceptable to the Investigator. These remedies must remain at a stable dose and regimen throughout the study
- Has sufficient venous access to allow drug administration by infusion and blood sampling as per the protocol
- Participants should have a body weight and body mass index between 5th and 95th percentile for age and sex. Obese participants greater than 95th percentile and underweight participants below 5th percentile may participate following medical clearance
- A female of childbearing potential must have a negative highly sensitive serum (beta-human chorionic gonadotropin [beta-hCG]) at Screening and a negative urine pregnancy test at Day 1 prior to administration of study intervention
Key Exclusion Criteria:
- Has a history of severe and/or uncontrolled hepatic (example, viral/alcoholic/ autoimmune hepatitis/ cirrhosis/ and/or metabolic liver disease), gastrointestinal, renal, pulmonary, cardiovascular (including congenital heart diseases), psychiatric, neurological musculoskeletal disorder, any other medical disorder(s) (example, diabetes mellitus), risk factors for thrombosis events (example, a history of venous thromboembolism [VTE] or antiphospholipid syndrome, or a personal or family history of heritable coagulation disorder such as factor V leiden, protein S or protein C deficiency, atrial fibrillation/flutter, major orthopedic surgery or significant trauma that may increase the risk of VTE, is expected to be immobilized for prolonged periods of time), or has clinically significant abnormalities in screening laboratory, that might interfere with participant's full participation in the study, and/ or might jeopardize the safety of the participant or the validity of the study results
- Has any confirmed or suspected clinical immunodeficiency syndrome not related to treatment of his/her generalized myasthenia gravis (gMG), or has a family history of congenital or hereditary immunodeficiency unless confirmed absent in the participant
- Has had a thymectomy within 12 months prior to screening, or thymectomy is planned during the Active treatment Phase of the study
- Has shown a previous severe immediate hypersensitivity reaction, such as anaphylaxis to therapeutic proteins (example, monoclonal antibodies)
- Has experienced myocardial infarction, unstable ischemic heart disease, or stroke within 12 weeks of screening
공부 계획
연구는 어떻게 설계됩니까?
디자인 세부사항
- 주 목적: 치료
- 할당: 해당 없음
- 중재 모델: 단일 그룹 할당
- 마스킹: 없음(오픈 라벨)
무기와 개입
참가자 그룹 / 팔 |
개입 / 치료 |
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실험적: Nipocalimab
Participants age 2 to less than (<) 18 years of age (globally) and 8 to <18 years of age (for US sites only) will be divided into 2 cohorts as per their age-adolescents 12 to <18 years and children 2 to <12 years and will receive nipocalimab once every two weeks for 24 weeks. After Week 24, all participants will have the option to enroll in long term extension (LTE). |
Nipocalimab will be administered as an IV infusion.
다른 이름들:
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연구는 무엇을 측정합니까?
주요 결과 측정
결과 측정 |
측정값 설명 |
기간 |
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Change from Baseline in Total Serum Immunoglobulin-G (IgG) Levels
기간: Up to 3 years
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Change from baseline in total serum IgG levels were reported. |
Up to 3 years
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Number of Participants with Infectious Adverse Events (AEs)
기간: Up to 3 years
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Number of participants with infectious AEs will be reported. An AE is any untoward medical occurrence in a participant participating in a clinical study that does not necessarily have a causal relationship with the pharmaceutical/biological agent under study. |
Up to 3 years
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Number of Participants with Serious AEs (SAEs)
기간: Up to 3 years
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Number of participants with SAEs will be reported. A SAE is any AE that results in: death, persistent or significant disability/incapacity, requires inpatient hospitalization or prolongation of existing hospitalization, is life-threatening experience, is a congenital anomaly/birth defect, is suspected transmission of any infectious agent via a medicinal product, is medically important to prevent one of the outcomes listed above. |
Up to 3 years
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Number of Participants with Adverse Events of Special Interests (AESIs)
기간: Up to 3 years
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Number of participants with AESIs will be reported. Treatment-emergent AEs associated with the following situations are considered an AESI: a) infections that are severe or require intravenous (IV) anti-infective or operative/invasive intervention; b) hypoalbuminemia with albumin less than (<)20 grams per liter (g/L) [<] 2.0 grams per deciliter [g/dL]) c) opportunistic infections and d) Serious and non-serious deep-vein thrombosis (DVT) and/or pulmonary embolism (PE). Any AE occurring at or after the initial administration of study intervention through end of study is treatment emergent. |
Up to 3 years
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Number of Participants with Abnormalities in Clinical Laboratory Tests
기간: Up to 3 years
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Number of participants with abnormalities in clinical laboratory tests (including chemistry, hematology, coagulation, and urinalysis) will be reported. |
Up to 3 years
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Number of Participants with Abnormalities in Vital Signs
기간: Up to 3 years
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Number of participants with abnormalities in vital signs including sitting pulse/heart rate, sitting systolic and diastolic blood pressure, and oral temperature (degrees Celsius) will be reported. |
Up to 3 years
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Number of Participants with Abnormalities in Physical Examination
기간: Up to 3 years
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Number of participants with abnormalities in physical examinations including height, weight, assessments of the skin, head, eyes, ears, nose, throat, neck, thyroid, lungs, heart, abdomen, lymph nodes and extremities will be reported. |
Up to 3 years
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Serum Concentration of Nipocalimab over Time
기간: Up to 3 years
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Serum samples will be analyzed to determine concentrations of nipocalimab using a validated, specific, and sensitive immunoassay method. |
Up to 3 years
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Clearance (CL) of Nipocalimab
기간: Up to 3 years
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CL is defined as the volume of serum from which nipocalimab is completely removed per unit time. |
Up to 3 years
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Volume of Distribution (V) of Nipocalimab
기간: Up to 3 years
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V is defined as the representation of nipocalimab's propensity to either remain in the serum or redistribute to other tissue compartments. |
Up to 3 years
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Half-life (t1/2) of Nipocalimab
기간: Up to 3 years
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t1/2 is defined as the time it takes for nipocalimab's active substance in the body to reduce by half. |
Up to 3 years
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Steady-state Peak Concentration (Cpeak,ss) of Nipocalimab
기간: Up to 3 years
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Cpeak,ss is defined as the peak serum concentration of nipocalimab at steady state. |
Up to 3 years
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Steady-state Trough concentration (Ctrough,ss) of Nipocalimab
기간: Up to 3 years
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Ctrough,ss will be reported. It is defined as the observed serum concentration of nipocalimab just prior to the beginning of a dosing interval at steady state. |
Up to 3 years
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Steady-state Area Under the Curve (AUCss) of Nipocalimab
기간: Up to 3 years
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AUCss is defined as the area under the curve for nipocalimab at steady state. |
Up to 3 years
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2차 결과 측정
결과 측정 |
측정값 설명 |
기간 |
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Change from Baseline in Myasthenia Gravis -Activities of Daily Living (MG-ADL) Score
기간: Up to 3 years
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Change from baseline in MG-ADL score will be reported. The MG-ADL score provides a rapid assessment of the participant's myasthenia gravis (MG) symptom severity. Eight functions (talking, chewing, swallowing, breathing, impairment of ability to brush teeth or comb hair, impairment of ability to arise from a chair, double vision, eyelid droop) are rated on a 4-point scale: 0 (no impairment) to 3 (severe impairment). The total score will be sum of eight function scores and can range from 0 to 24. A higher score indicates greater symptom severity. |
Up to 3 years
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Change in the Quantitative Myasthenia Gravis (QMG) Score
기간: Up to 3 years
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The QMG score is a standardized quantitative strength assessment comprising 13 components (and is administered by a trained qualified healthcare professional [HCP] eg, physician, physician assistant, nurse practitioner, nurse). The quantitative results of each strength component are mapped to the following 4-point scale: 0 equals to (=) none, 1 = mild, 2 = moderate and 3 = severe. The total score will be sum of 13 components scores and can range from 0 to 39. A higher score indicates greater weakness. |
Up to 3 years
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European Quality of Life 5-Dimension Youth (EQ-5D-Y) Tool Score
기간: Up to 3 years
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The EQ-5D-Y is a standardized child friendly instrument for use as a measure of health status, primarily designed for self-completion by children and adolescents, or via a proxy version to be completed by the child's caregiver. The EQ-5D-Y descriptive system comprises the following 5 dimensions: Mobility, looking after myself (washing and dressing), usual activities, pain or discomfort and feeling worried or unhappy. Each of the 5 dimensions is divided into 3 levels of perceived problems (Level 1 indicating no problem, Level 2 indicating some problems, Level 3 a lot of problems). |
Up to 3 years
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Neurological Quality of Life (Neuro-QoL) Pediatric Fatigue Score
기간: Up to 3 years
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The Neuro-QoL pediatric fatigue score will be used to assess the impact of fatigue in participants aged 10 to less than (<) 18 years. The participant will rate each of the 11 items on a 5-point scale. Higher scores indicate greater fatigue. |
Up to 3 years
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Patient Global Impression of Severity (PGI-S) Score
기간: Up to 3 years
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The PGI-S score will be used to assess the severity of fatigue due to generalized myasthenia gravis (gMG) in participants aged 10 to < 18 years. Participants will be asked to rate their fatigue over the past 7 days using the following 5-point scale: 1 = None, 2 = Mild, 3 = Moderate, 4 = Severe, and 5 = Very severe. Higher scores indicate greater severity of fatigue. |
Up to 3 years
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Patient Global Impression of Change (PGI-C) Score
기간: Up to 3 years
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The PGI-C score will be used to assess if there has been an improvement or decline in patient-reported fatigue since the beginning of the treatment in participants aged 10 to <18 years. Participants will be asked to rate their current fatigue as compared to when they started the study, using the following 7-point scale: 1 = Much better, 2 = Moderately better, 3 = A little better, 4 = No change, 5 = A little worse, 6 = Moderately worse, and 7 = Much worse. Higher scores indicate greater change in overall fatigue. |
Up to 3 years
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Number of Participants with Anti-Drug Antibodies [ADAs] to Nipocalimab
기간: Up to 3 years
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Number of participants with ADAs to nipocalimab will be reported. |
Up to 3 years
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Number of Participants with Neutralizing Antibodies (NAbs) to Nipocalimab
기간: Up to 3 years
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Number of participants with NAbs to nipocalimab will be reported. |
Up to 3 years
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Number of Participants with Vaccine Antibody Titers to Diphtheria or Tetanus
기간: Up to 3 years
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Number of participants with vaccine antibody titers to diphtheria or tetanus will be reported. |
Up to 3 years
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공동 작업자 및 조사자
수사관
- 연구 책임자: Janssen Research & Development, LLC Clinical Trial, Janssen Research & Development, LLC
연구 기록 날짜
연구 주요 날짜
연구 시작 (실제)
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
키워드
추가 관련 MeSH 약관
기타 연구 ID 번호
- CR109137
- 2021-002479-20 (EudraCT 번호)
- 80202135MYG2001 (기타 식별자: Janssen Research & Development, LLC)
- 2022-502539-21-00 (레지스트리 식별자: EUCT number)
개별 참가자 데이터(IPD) 계획
개별 참가자 데이터(IPD)를 공유할 계획입니까?
IPD 계획 설명
The data sharing policy of Johnson & Johnson Innovative Medicine is available at www.innovativemedicine.jnj.com/our-innovation/clinical-trials/transparency. As noted on this site, requests for access to the study data can be submitted through Yale Open Data Access (YODA) Project site at yoda.yale.edu
약물 및 장치 정보, 연구 문서
미국 FDA 규제 의약품 연구
미국 FDA 규제 기기 제품 연구
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