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Набор скоро начнётся NCT07298928

A Study on the Efficacy and Safety of Telitacicept in the Treatment of Children Ocular Myasthenia Gravis

Без фазы С лечением Effectiveness

Ориентир для пациента и семьи

Простыми словами

Автоматическая сводка по структурированным данным реестра. Она помогает сориентироваться, но не заменяет официальный протокол или оценку врача.

Что изучают
В протоколе указаны: Telitacicept.
Кому может быть актуально
Состояния в реестре: Effectiveness. Базовые параметры: до 18 лет · Все.
Что важно проверить
Возраст, диагноз и пол — только базовые ориентиры. Предыдущее лечение, анализы и другие обязательные условия указаны ниже в критериях участия.
Где проводится
Китай
Следующий шаг
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Официальное название

The Children's Hospital, Zhejiang University School of Medicine, National Clinical Research Center for Child Health

Обзор

Under conventional treatment regimens, pediatric ocular myasthenia gravis (OMG) is prone to relapse and is associated with corticosteroid-related adverse effects, indicating an unmet clinical need. In May 2025, the targeted B-cell biologic agent Telitacicept was approved for use in adult patients with acetylcholine receptor (AChR) antibody-positive generalized myasthenia gravis (GMG) and subsequently initiated in national multicenter clinical trials for adult OMG. Our center published a retrospective study in the Chinese Journal of Evidence-Based Pediatrics in August 2025, which was the first report both domestically and internationally on the efficacy and safety of Telitacicept in four pediatric OMG patients. This study plans to conduct a prospective, multicenter, open-label, single-arm clinical trial aimed at evaluating the effectiveness and safety of Telitacicept in pediatric OMG.

Вмешательства

  • Препарат Telitacicept
    All enrolled pediatric patients with OMG who meet the inclusion criteria will receive subcutaneous injections of Telitacicept on top of their existing medication regimen. The dose will be adjusted according to the patient's body weight: 160 mg per dose for those weighing ≥40 kg; 80 mg per dose for those weighing between 20 kg and \<40 kg; for patients weighing \<20 kg or aged \<5 years, a gradual dose reduction may be considered based on individual circumstances. The administration schedule for

Первичные конечные точки

  • Ocular Myasthenia Gravis Rating Scale-q Score [Срок оценки: baseline, 1 week, 2 week,3 week,4 week,6 week, 8 week, 10 week,12 week, 16 week, 20 week, 24 week]
Вторичные конечные точки (4)
  • quantitative Myasthenia Gravis scale [Срок оценки: baseline, 1 week, 2 week,3 week,4 week,6 week, 8 week, 10 week,12 week, 16 week, 20 week, 24 week]
  • Myasthenia Gravis⁃activity of daily living scale [Срок оценки: baseline, 1 week, 2 week,3 week,4 week,6 week, 8 week, 10 week,12 week, 16 week, 20 week, 24 week]
  • Number and proportion of children patients who have their hormone therapy discontinued [Срок оценки: 8 week, 16 week, 24 week]
  • The incidence and severity of adverse events [Срок оценки: baseline, 4 week, 8 week, 12 week, 24 week]

Критерии участия

Критерии включения

  • The patient and their legal guardian voluntarily sign the informed consent form.
  • Age < 18 years, male or female.
  • Diagnosis of OMG according to the Chinese Guidelines for the Diagnosis and Treatment of Myasthenia Gravis (2025 Edition).
  • Stable administration of any one or combination of the following standard treatments prior to enrollment:
  • Cholinesterase inhibitors
  • Glucocorticoids

Критерии исключения

  • Active infection under treatment： Patients who are HBsAg positive must be excluded. Patients who are HBsAg negative but HBcAb positive must undergo quantitative HBV-DNA testing. Patients with a positive quantitative HBV-DNA result must be excluded; those with a negative result may be enrolled.
  • Severe hepatic or renal insufficiency.
  • Patients with malignant tumors other than thymoma.
  • Patients within 3 months post-thymectomy.
  • Hypogammaglobulinemia (IgG < 400 mg/dL) or IgA deficiency (IgA < 10 mg/dL).
  • History of allergy to human-derived biological products.
  • Participation in any other clinical trial within 28 days prior to enrollment or within 5 times the half-life of the investigational drug from the previous trial (whichever is longer).
  • Patients deemed unsuitable for participation by the investigator (e.g., patients with severe psychiatric disorders).

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение
Не применимо
Модель
Одна группа
Маскирование
Открытое
Основная цель
Лечение

Центры проведения

Китай · 3 центра
  • Shenzhen children's Hospital of China Medical University — Шэньчжэнь
  • Children's Hospital Affiliated to Shandong University — Цзинань
  • The Children's Hospital, Zhejiang University School of Medicine — Ханчжоу

Идентификаторы

NCT: NCT07298928 · KYYS-2025-0347

Первоисточники (государственные реестры)

Открыть это исследование на ClinicalTrials.gov ↗