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 Telitacicept (80 mg or 160 mg per dose) is as follows: once weekly via subcutaneous injection for the first 12 weeks; subsequently, once every two weeks via subcutaneous injection for the next 12 weeks; followed by once every four weeks via subcutaneous injection thereafter. Corticosteroids will be gradually tapered and discontinued based on changes in the patient's clinical condition.
Study summary
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.
Eligibility
Sex
ALL
Min age
—
Max age
18 Years
Healthy volunteers
No
Inclusion Criteria:
1. The patient and their legal guardian voluntarily sign the informed consent form.
2. Age \< 18 years, male or female.
3. Diagnosis of OMG according to the Chinese Guidelines for the Diagnosis and Treatment of Myasthenia Gravis (2025 Edition).
4. Stable administration of any one or combination of the following standard treatments prior to enrollment:
1. Cholinesterase inhibitors
2. Glucocorticoids
Exclusion Criteria:
1. 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.
2. Severe hepatic or renal insufficiency.
3. Patients with malignant tumors other than thymoma.
4. Patients within 3 months post-thymectomy.
5. Hypogammaglobulinemia (IgG \< 400 mg/dL) or IgA deficiency (IgA \< 10 mg/dL).
6. History of allergy to human-derived biological products.
7. 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).
8. Patients deemed unsuitable for participation by the investigator (e.g., patients with severe psychiatric disorders).
Primary outcome measure(s)
Ocular Myasthenia Gravis Rating Scale-q Score — 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.
Trial sites (3)
Facility
City
Region
Status
Shenzhen children's Hospital of China Medical University
Shenzhen
Guangdong
Children's Hospital Affiliated to Shandong University
Jinan
Shandong
The Children's Hospital, Zhejiang University School of Medicine
Hangzhou
Zhejiang
More The Children's Hospital of Zhejiang University School of Medicine trials in China
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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