EffectivenessNCT07298928
A Study on the Efficacy and Safety of Telitacicept in the Treatment of Children Ocular Myasthenia Gravis
A study testing Telitacicept in people with Effectiveness, run by The Children's Hospital of Zhejiang University School of Medicine at 3 sites in China.
Trial 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. Read more about this study
- Who is it for?
- Ages Up to 18 years
- Conditions the study is for
- Effectiveness
- What is being tested?
- Telitacicept
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 3 sitesChina
- Study dates
- 10 Mar 2026to Dec 2028
- Phase
- Interventional
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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
Who cannot join
- 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).
Trial locations and contacts
3 sites in 1 country. Contact the study team or a site near you to ask about taking part.
China3 sites
The Children's Hospital, Zhejiang University School of Medicine
Hangzhou, Zhejiang, 310052
Shanshan Mao, 15990042668, 6307003@zju.edu.cn
Children's Hospital Affiliated to Shandong University
Jinan, Shandong
Kai Ma, 18358628830, shdhmmk@126.com
Shenzhen children's Hospital of China Medical University
Shenzhen, Guangdong
Xufeng Luo, 19012770239, 75172133@qq.com
About this study
Official title: 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.
- Conditions
- Effectiveness
- Being tested
- Telitacicept
- Keywords
- long-term follow-up, Ocular myasthenia gravis, Telitacicept, Children, Biological agent, Targeted treatment
- Medical subject headings
- Myasthenia Gravis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TelitaciceptDrug
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.
| Study group | Role | What they receive |
|---|---|---|
| a single Telitacicept treatment group | Experimental | Not described Receives: Drug: Telitacicept |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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
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.
Measured: 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
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.
Measured: 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
Measured: 8 week, 16 week, 24 week
The incidence and severity of adverse events
Measured: baseline, 4 week, 8 week, 12 week, 24 week
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Dec 2025
- Study start10 Mar 2026 (estimated)
- Primary completionDec 2028 (estimated)
- Study completionDec 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for EffectivenessStudies of the same condition, open and under way.
- More trials run by The Children's Hospital of Zhejiang University School of MedicineThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 30 participants (estimated)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Shanshan Mao, The Children's Hospital of Zhejiang University School of Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07298928
- Sponsor's protocol ID
- KYYS-2025-0347
- Status last verified
- Mar 2026
- First submitted
- 9 Dec 2025
- Last updated
- 6 Mar 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 6 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Children's Hospital, Zhejiang University School of Medicine, National Clinical Research Center for Child Health
- ClinicalTrials.gov ID
- NCT07298928
- Acronym / sponsor ID
- KYYS-2025-0347
- Conditions
- Effectiveness
- Keywords
- long-term follow-up, Ocular myasthenia gravis, Telitacicept, Children, Biological agent, Targeted treatment
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- The Children's Hospital of Zhejiang University School of MedicineUniversity, hospital or other
- Study start
- 10 Mar 2026 (estimated)
- Primary completion
- Dec 2028 (estimated)
- Study completion
- Dec 2028 (estimated)
- First posted
- 23 Dec 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| a single Telitacicept treatment group | Experimental | Drug: Telitacicept |
Who may qualify
- Ages
- Up to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (6 listed)
- 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:
Key exclusion criteria (8 listed)
- 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).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| TelitaciceptAll 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. | Drug | a single Telitacicept treatment group |
Primary outcome
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.
4 secondary outcomes
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
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
Number and proportion of children patients who have their hormone therapy discontinued
Time frame: 8 week, 16 week, 24 week
The incidence and severity of adverse events
Time frame: baseline, 4 week, 8 week, 12 week, 24 week
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Mar 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Telitacicept in people with Effectiveness, run by The Children's Hospital of Zhejiang University School of Medicine at 3 sites in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 6 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Shanshan Mao (15990042668, 6307003@zju.edu.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 Mar 2026.