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Nipocalimab 在 2 至 18 岁以下患有全身性重症肌无力儿童中的研究

2026年11月2日 更新者：Janssen Research & Development, LLC

一项开放标签非对照多中心研究，旨在评估 Nipocalimab 在 2 至 18 岁以下患有全身性重症肌无力的儿童中的药代动力学、药效学、安全性和活性

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.

研究概览

地位

招聘中

条件

干预/治疗

研究类型

介入性

注册 (估计的)

12

阶段

  • 阶段2
  • 第三阶段

扩展访问

暂时不可用 查看扩展访问记录。

联系人和位置

本节提供了进行研究的人员的详细联系信息，以及有关进行该研究的地点的信息。

学习联系方式

学习地点

  • 日本
      • Azumino-shi、日本、399-8288
        • 招聘中
        • Nagano Children's Hospital
      • Chiba、日本、260 8677
        • 完全的
        • Chiba University Hospital
      • Miyazaki、日本、889-1692
        • 招聘中
        • University of Miyazaki Hospital
      • Nishinomiya-Shi、日本、663-8501
        • 招聘中
        • Hyogo College of Medicine Hospital
      • Saitama Shi、日本、330-8777
        • 招聘中
        • Saitama Prefecture Children's Medical Center
      • Shinjuku-ku、日本、162-8666
        • 招聘中
        • Tokyo Women's Medical University Hospital
  • 波兰
      • Gdansk、波兰、80 211
        • 招聘中
        • Uniwersyteckie Centrum Kliniczne
  • 美国
    • Arizona
      • Phoenix、Arizona、美国、85016
        • 招聘中
        • Phoenix Children's Hospital
    • California
      • Los Angeles、California、美国、90027
        • 完全的
        • Childrens Hospital Los Angeles
      • Palo Alto、California、美国、94304
        • 招聘中
        • Lucile Packard Children's Hospital Stanford
      • San Francisco、California、美国、94158
        • 招聘中
        • UCSF Benioff Children's Hospital
    • Colorado
      • Aurora、Colorado、美国、80045
        • 招聘中
        • Children's Hospital Colorado
    • Florida
      • Tampa、Florida、美国、33613
        • 招聘中
        • University of South Florida Morsani Center for Advanced Healthcare
    • Kansas
      • Lawrence、Kansas、美国、66045
        • 完全的
        • University of Kansas Medical Center
    • Michigan
      • Ann Arbor、Michigan、美国、48109
        • 招聘中
        • C.S. Mott Children's Hospital
    • Pennsylvania
      • Hershey、Pennsylvania、美国、17033
        • 招聘中
        • Penn State Milton S Hershey Medical Ctr
      • Philadelphia、Pennsylvania、美国、19106
        • 招聘中
        • Childrens Hospital Of Philadelphia
      • Pittsburgh、Pennsylvania、美国、15224
        • 终止
        • University of Pittsburgh Medical Center
  • 荷兰
      • Leiden、荷兰、2333 ZA
        • 招聘中
        • Leiden University Medical Center

参与标准

研究人员寻找符合特定描述的人，称为资格标准。这些标准的一些例子是一个人的一般健康状况或先前的治疗。

资格标准

适合学习的年龄

2年 至 17年 (孩子)

接受健康志愿者

不

有资格学习的性别

全部

描述

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

学习计划

本节提供研究计划的详细信息，包括研究的设计方式和研究的衡量标准。

研究是如何设计的？

设计细节

  • 主要用途：治疗
  • 分配：不适用
  • 介入模型：单组作业
  • 屏蔽：无（打开标签）

武器和干预

参与者组/臂
干预/治疗
实验性的：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).
药品：Nipocalimab

Nipocalimab will be administered as an IV infusion.

其他名称：
  • M281/JNJ-80202135

研究衡量的是什么？

主要结果指标

结果测量
措施说明
大体时间
Change from Baseline in Total Serum Immunoglobulin-G (IgG) Levels
大体时间：Up to 3 years

Change from baseline in total serum IgG levels were reported.
Up to 3 years
Number of Participants with Infectious Adverse Events (AEs)
大体时间：Up to 3 years

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
Number of Participants with Serious AEs (SAEs)
大体时间：Up to 3 years

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
Number of Participants with Adverse Events of Special Interests (AESIs)
大体时间：Up to 3 years

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
Number of Participants with Abnormalities in Clinical Laboratory Tests
大体时间：Up to 3 years

Number of participants with abnormalities in clinical laboratory tests (including chemistry, hematology, coagulation, and urinalysis) will be reported.
Up to 3 years
Number of Participants with Abnormalities in Vital Signs
大体时间：Up to 3 years

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
Number of Participants with Abnormalities in Physical Examination
大体时间：Up to 3 years

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
Serum Concentration of Nipocalimab over Time
大体时间：Up to 3 years

Serum samples will be analyzed to determine concentrations of nipocalimab using a validated, specific, and sensitive immunoassay method.
Up to 3 years
Clearance (CL) of Nipocalimab
大体时间：Up to 3 years

CL is defined as the volume of serum from which nipocalimab is completely removed per unit time.
Up to 3 years
Volume of Distribution (V) of Nipocalimab
大体时间：Up to 3 years

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
Half-life (t1/2) of Nipocalimab
大体时间：Up to 3 years

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
Steady-state Peak Concentration (Cpeak,ss) of Nipocalimab
大体时间：Up to 3 years

Cpeak,ss is defined as the peak serum concentration of nipocalimab at steady state.
Up to 3 years
Steady-state Trough concentration (Ctrough,ss) of Nipocalimab
大体时间：Up to 3 years

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
Steady-state Area Under the Curve (AUCss) of Nipocalimab
大体时间：Up to 3 years

AUCss is defined as the area under the curve for nipocalimab at steady state.
Up to 3 years

次要结果测量

结果测量
措施说明
大体时间
Change from Baseline in Myasthenia Gravis -Activities of Daily Living (MG-ADL) Score
大体时间：Up to 3 years

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
Change in the Quantitative Myasthenia Gravis (QMG) Score
大体时间：Up to 3 years

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
European Quality of Life 5-Dimension Youth (EQ-5D-Y) Tool Score
大体时间：Up to 3 years

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
Neurological Quality of Life (Neuro-QoL) Pediatric Fatigue Score
大体时间：Up to 3 years

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
Patient Global Impression of Severity (PGI-S) Score
大体时间：Up to 3 years

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
Patient Global Impression of Change (PGI-C) Score
大体时间：Up to 3 years

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
Number of Participants with Anti-Drug Antibodies [ADAs] to Nipocalimab
大体时间：Up to 3 years

Number of participants with ADAs to nipocalimab will be reported.
Up to 3 years
Number of Participants with Neutralizing Antibodies (NAbs) to Nipocalimab
大体时间：Up to 3 years

Number of participants with NAbs to nipocalimab will be reported.
Up to 3 years
Number of Participants with Vaccine Antibody Titers to Diphtheria or Tetanus
大体时间：Up to 3 years

Number of participants with vaccine antibody titers to diphtheria or tetanus will be reported.
Up to 3 years

合作者和调查者

在这里您可以找到参与这项研究的人员和组织。

赞助

Janssen Research & Development, LLC

调查人员

  • 研究主任：Janssen Research & Development, LLC Clinical Trial、Janssen Research & Development, LLC

研究记录日期

这些日期跟踪向 ClinicalTrials.gov 提交研究记录和摘要结果的进度。研究记录和报告的结果由国家医学图书馆 (NLM) 审查，以确保它们在发布到公共网站之前符合特定的质量控制标准。

研究主要日期

学习开始 (实际的)

2022年7月20日

初级完成 (估计的)

2027年6月30日

研究完成 (估计的)

2029年7月2日

研究注册日期

首次提交

2022年2月22日

首先提交符合 QC 标准的

2022年2月22日

首次发布 (实际的)

2022年3月3日

研究记录更新

最后更新发布 (实际的)

2026年11月4日

上次提交的符合 QC 标准的更新

2026年11月2日

最后验证

2026年11月1日

更多信息

与本研究相关的术语

关键字

其他相关的 MeSH 术语

其他研究编号

  • CR109137
  • 2021-002479-20 (EudraCT编号)
  • 80202135MYG2001 (其他标识符：Janssen Research & Development, LLC)
  • 2022-502539-21-00 (注册表标识符：EUCT number)

计划个人参与者数据 (IPD)

计划共享个人参与者数据 (IPD)？

是的

IPD 计划说明

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

药物和器械信息、研究文件

研究美国 FDA 监管的药品

是的

研究美国 FDA 监管的设备产品

不

此信息直接从 clinicaltrials.gov 网站检索，没有任何更改。如果您有任何更改、删除或更新研究详细信息的请求，请联系 register@clinicaltrials.gov. clinicaltrials.gov 上实施更改，我们的网站上也会自动更新.

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