A Study on the Efficacy and Safety of Telitacicept in the Treatment of Children Ocular Myasthenia Gravis
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Telitacicept.
- Who it may be relevant to
- Registry conditions: Effectiveness. Basic parameters: up to 18 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- China
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
The Children's Hospital, Zhejiang University School of Medicine, National Clinical Research Center for Child Health
Overview
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.
Interventions
- Drug 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
Primary outcome measures
- Ocular Myasthenia Gravis Rating Scale-q Score [Time frame: baseline, 1 week, 2 week,3 week,4 week,6 week, 8 week, 10 week,12 week, 16 week, 20 week, 24 week]
Secondary outcome measures (4)
- quantitative Myasthenia Gravis scale [Time frame: 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 [Time frame: 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 [Time frame: 8 week, 16 week, 24 week]
- The incidence and severity of adverse events [Time frame: baseline, 4 week, 8 week, 12 week, 24 week]
Eligibility criteria
Inclusion criteria
- 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
Exclusion criteria
- 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).
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- N/A
- Model
- Single group
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
China · 3 centers
- Shenzhen children's Hospital of China Medical University — Shenzhen
- Children's Hospital Affiliated to Shandong University — Jinan
- The Children's Hospital, Zhejiang University School of Medicine — Hangzhou
Identifiers
NCT: NCT07298928 · KYYS-2025-0347