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Исследование нипокалимаба у детей в возрасте от 2 до 18 лет с генерализованной миастенией

2 ноября 2026 г. обновлено: Janssen Research & Development, LLC

Открытое неконтролируемое многоцентровое исследование по оценке фармакокинетики, фармакодинамики, безопасности и активности нипокалимаба у детей в возрасте от 2 до 18 лет с генерализованной миастенией гравис

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.

Обзор исследования

Статус

Рекрутинг

Условия

Вмешательство/лечение

Тип исследования

Интервенционный

Регистрация (Оцененный)

12

Фаза

  • Фаза 2
  • Фаза 3

Расширенный доступ

Временно недоступно вне клинических испытаний. См. запись расширенного доступа.

Контакты и местонахождение

В этом разделе приведены контактные данные лиц, проводящих исследование, и информация о том, где проводится это исследование.

Контакты исследования

Места учебы

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

Критерии участия

Исследователи ищут людей, которые соответствуют определенному описанию, называемому критериям приемлемости. Некоторыми примерами этих критериев являются общее состояние здоровья человека или предшествующее лечение.

Критерии приемлемости

Возраст, подходящий для обучения

От 2 года до 17 лет (Ребенок)

Принимает здоровых добровольцев

Нет

Полы, имеющие право на обучение

Все

Описание

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

Учебный план

В этом разделе представлена ​​подробная информация о плане исследования, в том числе о том, как планируется исследование и что оно измеряет.

Как устроено исследование?

Детали дизайна

  • Основная цель: Уход
  • Распределение: Н/Д
  • Интервенционная модель: Одногрупповое задание
  • Маскировка: Нет (открытая этикетка)

Оружие и интервенции

Группа участников / Армия
Вмешательство/лечение
Экспериментальный: 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).
Лекарство: Nipocalimab

Nipocalimab will be administered as an IV infusion.

Другие имена:
  • М281/ДЖНЖ-80202135

Что измеряет исследование?

Первичные показатели результатов

Мера результата
Мера Описание
Временное ограничение
Change from Baseline in Total Serum Immunoglobulin-G (IgG) Levels
Временное ограничение: 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)
Временное ограничение: 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)
Временное ограничение: 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)
Временное ограничение: 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
Временное ограничение: 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
Временное ограничение: 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
Временное ограничение: 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
Временное ограничение: 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
Временное ограничение: 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
Временное ограничение: 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
Временное ограничение: 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
Временное ограничение: 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
Временное ограничение: 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
Временное ограничение: Up to 3 years

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

Вторичные показатели результатов

Мера результата
Мера Описание
Временное ограничение
Change from Baseline in Myasthenia Gravis -Activities of Daily Living (MG-ADL) Score
Временное ограничение: 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
Временное ограничение: 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
Временное ограничение: 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
Временное ограничение: 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
Временное ограничение: 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
Временное ограничение: 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
Временное ограничение: 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
Временное ограничение: 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
Временное ограничение: Up to 3 years

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

Соавторы и исследователи

Здесь вы найдете людей и организации, участвующие в этом исследовании.

Спонсор

Janssen Research & Development, LLC

Следователи

  • Директор по исследованиям: Janssen Research & Development, LLC Clinical Trial, Janssen Research & Development, LLC

Даты записи исследования

Эти даты отслеживают ход отправки отчетов об исследованиях и сводных результатов на сайт ClinicalTrials.gov. Записи исследований и сообщаемые результаты проверяются Национальной медицинской библиотекой (NLM), чтобы убедиться, что они соответствуют определенным стандартам контроля качества, прежде чем публиковать их на общедоступном веб-сайте.

Изучение основных дат

Начало исследования (Действительный)

20 июля 2022 г.

Первичное завершение (Оцененный)

30 июня 2027 г.

Завершение исследования (Оцененный)

2 июля 2029 г.

Даты регистрации исследования

Первый отправленный

22 февраля 2022 г.

Впервые представлено, что соответствует критериям контроля качества

22 февраля 2022 г.

Первый опубликованный (Действительный)

3 марта 2022 г.

Обновления учебных записей

Последнее опубликованное обновление (Действительный)

4 ноября 2026 г.

Последнее отправленное обновление, отвечающее критериям контроля качества

2 ноября 2026 г.

Последняя проверка

1 ноября 2026 г.

Дополнительная информация

Термины, связанные с этим исследованием

Ключевые слова

Дополнительные соответствующие термины MeSH

Другие идентификационные номера исследования

  • CR109137
  • 2021-002479-20 (Номер EudraCT)
  • 80202135MYG2001 (Другой идентификатор: Janssen Research & Development, LLC)
  • 2022-502539-21-00 (Идентификатор реестра: EUCT number)

Планирование данных отдельных участников (IPD)

Планируете делиться данными об отдельных участниках (IPD)?

ДА

Описание плана IPD

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

Информация о лекарствах и устройствах, исследовательские документы

Изучает лекарственный продукт, регулируемый FDA США.

Да

Изучает продукт устройства, регулируемый Управлением по санитарному надзору за качеством пищевых продуктов и медикаментов США.

Нет

Эта информация была получена непосредственно с веб-сайта clinicaltrials.gov без каких-либо изменений. Если у вас есть запросы на изменение, удаление или обновление сведений об исследовании, обращайтесь по адресу register@clinicaltrials.gov. Как только изменение будет реализовано на clinicaltrials.gov, оно будет автоматически обновлено и на нашем веб-сайте. .

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