Telitacicept for ocular myasthenia gravis

A Phase III Trial of Telitacicept in Patients With Ocular Myasthenia Gravis

This trial will test whether telitacicept can reduce eye muscle weakness in people aged 12 to 80 with ocular myasthenia gravis who are on stable standard treatment.

Quick facts

• Phase: Phase 3
• Study type: Interventional
• Enrollment: 120 (estimated)
• Ages: 12 Years to 80 Years
• Sex: All
• Sponsor: RemeGen Co., Ltd. (Industry-sponsored)
• Locations: 1 site (Beijing, Beijing Municipality)
• Trial ID: NCT07249632 on ClinicalTrials.gov

What this trial studies

This is a Phase III, multicenter, randomized, double-blind, placebo-controlled study enrolling about 120 participants who will be randomized 1:1 to telitacicept or matching placebo with dosing based on age and body weight. Randomization is stratified by acetylcholine receptor antibody status and by age (<18 vs ≥18). The primary endpoint is change from baseline in the patient-reported MGII ocular score at Week 24, with additional ocular and total MG outcome measures and safety assessments as secondary endpoints. Safety will be monitored through adverse event reporting, labs, vital signs, physical exams, and oversight by an independent Data Monitoring Committee.

Who should consider this trial

Good fit: Ideal candidates are people aged 12–80 with MGFA Type I ocular myasthenia gravis, body weight ≥30 kg, an MGII (PRO) ocular score of ≥6, and who are on a stable standard-of-care regimen.

Not a fit: Patients with generalized myasthenia gravis, thymoma, active or chronic infections, uncontrolled diabetes, positive HIV or syphilis serology, other autoimmune disease requiring systemic corticosteroids, recent non-stable immunosuppressant use, or those unable to be on a stable SOC regimen are unlikely to qualify or benefit.

Why it matters

Potential benefit: If successful, telitacicept could reduce ocular symptoms and improve daily functioning for people with ocular myasthenia gravis, potentially lowering the need for additional immunosuppressive therapies.

How similar studies have performed: Other B-cell–targeting biologic therapies have shown benefit in myasthenia gravis and related autoimmune conditions, but telitacicept's effect specifically in ocular-only MG has not been proven in prior late-phase trials.

Eligibility criteria

Inclusion Criteria:

1. Voluntarily signed the informed consent form.
2. Age 12 to 80 years, inclusive, male or female.
3. Body weight ≥30 kg.
4. Diagnosis of Myasthenia Gravis (MG) with documented clinical features consistent with the disease.
5. Myasthenia Gravis Foundation of America (MGFA) Clinical Classification of Type I.
6. On a stable standard-of-care (SOC) treatment regimen.

Exclusion Criteria:

1. Concomitant autoimmune diseases requiring systemic corticosteroid therapy.
2. Clinically significant laboratory abnormalities.
3. Use of other immunosuppressants (not part of the stable SOC) within 1 month prior to randomization.
4. Presence of an acute or chronic infection requiring treatment.
5. Current active hepatitis or history of severe liver disease.
6. Positive for HIV antibodies.
7. Positive for syphilis antibodies (non-specific or specific).
8. Poorly controlled diabetes mellitus, defined as HbA1c \>9.0% or fasting blood glucose ≥11.1 mmol/L.
9. Subjects with thymoma (classified as ≤ Stage II for benign and ≥ Stage III for malignant according to the Masaoka staging system) .
10. Presence of uncontrolled chronic degenerative diseases, psychiatric disorders, or neurological diseases other than MG that could interfere with study assessments.
11. Other diseases causing ptosis, peripheral muscle weakness, or diplopia (e.g., Graves' ophthalmopathy, blepharospasm, progressive external ophthalmoplegia, muscular dystrophy, brainstem or cranial nerve lesions, etc.).
12. Known allergy to human-derived biological products.

Where this trial is running

Beijing, Beijing Municipality

• Beijing Hospital — Beijing, Beijing Municipality, China (Recruiting)

Conditions Myasthenia Gravis, Ocular