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 Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
30
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.
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
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.
Time frame: baseline, 1 week, 2 week,3 week,4 week,6 week, 8 week, 10 week,12week, 16 week, 20 week, 24 week, 28 week, 32 week, 36 week, 40 week, 44 week, 48 week
quantitative Myasthenia Gravis scale
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.
Time frame: baseline, 1 week, 2 week,3 week,4 week,6 week, 8 week, 10 week,12week, 16 week, 20 week, 24 week, 28 week, 32 week, 36 week, 40 week, 44 week, 48 week
Myasthenia Gravis⁃activity of daily living scale
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.
Time frame: baseline, 1 week, 2 week,3 week,4 week,6 week, 8 week, 10 week,12week, 16 week, 20 week, 24 week, 28 week, 32 week, 36 week, 40 week, 44 week, 48 week
The proportion of children patients who have their hormone therapy discontinued
Time frame: 12week, 48 week
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