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

Zhejiang University

Status: Not yet enrolling

Conditions: Effectiveness

Treatments: Drug: Telitacicept

Study type: Interventional

Funder types: Other

Identifiers

NCT07298928

KYYS-2025-0347

Details and patient eligibility

About

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.

Enrollment: 30 estimated patients

Sex: All

Ages: Under 18 years old

Volunteers: No Healthy Volunteers

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. Receiving corticosteroid therapy prior to enrollment.

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).

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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30 participants in 1 patient group

a single Telitacicept treatment group

Experimental group

Treatment:

Drug: Telitacicept