The Children's Hospital, Zhejiang University School of Medicine, National Clinical Research Center for Child Health
试验速览
- 阶段
- 不适用
- 状态
- 尚未招募
- 发起方
- 入组人数
- 30
- 试验地点
- 3
- 主要终点
- Ocular Myasthenia Gravis Rating Scale-q Score
研究概览
简要总结
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- — 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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
排除标准
- •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).
研究组 & 干预措施
a single Telitacicept treatment group
干预措施: Telitacicept (Drug)
结局指标
主要结局
Ocular Myasthenia Gravis Rating Scale-q Score
时间窗: baseline, 1 week, 2 week,3 week,4 week,6 week, 8 week, 10 week,12 week, 16 week, 20 week, 24 week
This Ocular Myasthenia Gravis Rating Scale (OMGRate) is used to assess the impact of symptoms on the quality of daily life in patients with OMG. The OMGRate-q section of the scale is the patient - reported outcome component for evaluating the improvement of self - perceived symptoms. The questionnaire is completed based on the patient's condition over the past 2 weeks. The total score ranges from 0 to 52 points, with higher scores indicating more severe symptoms and greater impairment of daily quality of life.
次要结局
- The incidence and severity of adverse events(baseline, 4 week, 8 week, 12 week, 24 week)
- quantitative Myasthenia Gravis scale(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(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(8 week, 16 week, 24 week)
研究者
Shanshan Mao
Directo
The Children's Hospital of Zhejiang University School of Medicine