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

NCT07298928 | Not Yet Recruiting

• 1 other identifier: KYYS-2025-0347
• Study Type: interventional
• Target: 30
• Locations: 1 country
• Sites: 3

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

Outcome Measures

Primary Outcomes (1)

• Ocular Myasthenia Gravis Rating Scale-q Score

  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.

  baseline, 1 week, 2 week,3 week,4 week,6 week, 8 week, 10 week,12 week, 16 week, 20 week, 24 week

Secondary Outcomes (4)

• 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

• The incidence and severity of adverse events

  baseline, 4 week, 8 week, 12 week, 24 week

Other Outcomes (1)

• Number and proportion of patients who discontinued cholinesterase inhibitors

  8 week, 16 week, 24 week

Study Arms (1)

a single Telitacicept treatment group

EXPERIMENTAL

Drug: Telitacicept

Interventions

Telitacicept 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.

a single Telitacicept treatment group

Eligibility Criteria

• Age: Up to 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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

You may not qualify if:

• 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).

Sponsors & Collaborators

• The Children's Hospital of Zhejiang University School of Medicine: lead

Study Sites (3)

Shenzhen children's Hospital of China Medical University

Shenzhen, Guangdong, China

Location

Children's Hospital Affiliated to Shandong University

Jinan, Shandong, China

Location

The Children's Hospital, Zhejiang University School of Medicine

Hangzhou, Zhejiang, 310052, China

Location

MeSH Terms

Conditions

Myasthenia Gravis

Interventions

telitacicept

Condition Hierarchy (Ancestors)

Paraneoplastic Syndromes, Nervous System; Nervous System Neoplasms; Neoplasms by Site; Neoplasms; Paraneoplastic Syndromes; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Neurodegenerative Diseases; Neuromuscular Junction Diseases; Neuromuscular Diseases; Autoimmune Diseases; Immune System Diseases

Central Study Contacts

Shanshan Mao

CONTACT

15990042668; 6307003@zju.edu.cn

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Directo

Study Record Dates

• First Submitted: December 9, 2025
• First Posted: December 23, 2025
• Study Start: March 10, 2026
• Primary Completion (Estimated): December 1, 2028
• Study Completion (Estimated): December 1, 2028
• Last Updated: March 6, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will not share

Locations

CN (3)