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

March 5, 2026 updated by: Shanshan Mao, The Children's Hospital of Zhejiang University School of Medicine

The Children's Hospital, Zhejiang University School of Medicine, National Clinical Research Center for Child Health

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.

Study Overview

Status

Not yet recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

30

Phase

  • Not Applicable

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • China
    • Guangdong
      • Shenzhen, Guangdong, China
        • Shenzhen children's Hospital of China Medical University
        • Contact:
    • Shandong
      • Jinan, Shandong, China
        • Children's Hospital Affiliated to Shandong University
        • Contact:
    • Zhejiang
      • Hangzhou, Zhejiang, China, 310052
        • The Children's Hospital, Zhejiang University School of Medicine
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult

Accepts Healthy Volunteers

No

Description

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).

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: a single Telitacicept treatment group
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 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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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

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 Outcome Measures

Outcome Measure
Measure Description
Time Frame
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

This scale is a standardized tool for the objective measurement of muscle strength and endurance in MG-affected muscle groups. It consists of 13 items covering the following aspects: eyes (onset time of diplopia and ptosis), facial muscles (lip closure strength), pharynx and larynx (swallowing and pronunciation tests), respiration (percentage of forced vital capacity relative to the predicted value), neck (duration of head lifting while lying supine), hand strength (strength reduction measured by a dynamometer), and limbs (duration of posture maintenance).

Each item is scored on a 4-point scale: 0 (normal), 1 (mild), 2 (moderate), and 3 (severe), with a total score ranging from 0 to 39. Higher scores indicate more severe symptoms.
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

This scale is mainly used to assess the impact of symptoms on the quality of daily life in patients with MG and reflect the severity of the disease.

It includes 8 items covering 4 aspects: eyes (frequency of ptosis and diplopia), bulbar region (degree of involvement in speech, chewing, and swallowing), respiration (relationship between breathing and physical activity), and limbs (ability to complete daily movements).

Each item is scored on a scale of 0 (normal) to 3 (most severe), with a total score ranging from 0 to 24. Higher scores indicate greater disease severity.
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
8 week, 16 week, 24 week
The incidence and severity of adverse events
Time Frame: baseline, 4 week, 8 week, 12 week, 24 week
baseline, 4 week, 8 week, 12 week, 24 week

Other Outcome Measures

Outcome Measure
Time Frame
Number and proportion of patients who discontinued cholinesterase inhibitors
Time Frame: 8 week, 16 week, 24 week
8 week, 16 week, 24 week

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

The Children's Hospital of Zhejiang University School of Medicine

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Estimated)

March 10, 2026

Primary Completion (Estimated)

December 1, 2028

Study Completion (Estimated)

December 1, 2028

Study Registration Dates

First Submitted

December 9, 2025

First Submitted That Met QC Criteria

December 20, 2025

First Posted (Actual)

December 23, 2025

Study Record Updates

Last Update Posted (Actual)

March 6, 2026

Last Update Submitted That Met QC Criteria

March 5, 2026

Last Verified

March 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • KYYS-2025-0347

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on Effectiveness

Clinical Trials on Telitacicept

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Gabon

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

Subscribe