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

NCT ID: NCT07298928

Last Updated: 2025-12-23

Basic Information

• Recruitment Status: NOT_YET_RECRUITING
• Clinical Phase: NA
• Total Enrollment: 30 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2026-01-15
• Study Completion Date: 2028-12-31

Brief 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.

Conditions

Effectiveness

Keywords

long-term follow-up; Ocular myasthenia gravis; Telitacicept; Children; Biological agent; Targeted treatment

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

a single Telitacicept treatment group

Group Type: EXPERIMENTAL

Telitacicept

Intervention Type: DRUG

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.

Interventions

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.

Intervention Type: DRUG

Eligibility Criteria

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).

• Maximum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

The Children's Hospital of Zhejiang University School of Medicine

OTHER

Sponsor Role: lead

Responsible Party

Shanshan Mao

Directo

Responsibility Role: PRINCIPAL_INVESTIGATOR

Locations

Shenzhen children's Hospital of China Medical University

Shenzhen, Guangdong, China

Children's Hospital Affiliated to Shandong University

Jinan, Shandong, China

The Children's Hospital, Zhejiang University School of Medicine

Hangzhou, Zhejiang, China

Countries

China

Central Contacts

Shanshan Mao

Role: CONTACT

Phone: 15990042668

Email: 6307003@zju.edu.cn

Facility Contacts

Xufeng Luo

Role: primary

Kai Ma

Role: primary

Shanshan Mao

Role: primary

Other Identifiers

KYYS-2025-0347

Identifier Type: -

Identifier Source: org_study_id