A Study of Telitacicept for the Treatment of Generalized Myasthenia Gravis

Phase 3

Recruiting

• Conditions: Generalized Myasthenia Gravis

• Registration Number: 2024-512126-29-00
• Lead Sponsor: Remegen Co. Ltd.

Brief Summary

To evaluate the efficacy of telitacicept versus placebo in patients with generalized myasthenia gravis (gMG)

Detailed Description

Myasthenia gravis (MG) is an autoimmune disease that affects the neuromuscular junction on the postsynaptic membrane. The predominant manifestation is muscle weakness, which typically worsens with repeated muscle exertion, such that function is usually the best in the morning with more pronounced weakness at the end of the day. A major challenge in MG is the lack of therapies that cure the disease.

Telitacicept is a fully human TACI-Fc fusion protein that targets B-lymphocyte stimulator (BLyS) and A proliferating-inducing ligand (APRIL), neutralizing their interactions with receptors on B cells. The blockage of BLyS and APRIL interaction with their respective cell membrane receptors (transmembrane activator and CAML interactor \[TACI\], B-cell maturation antigen, and BLyS receptors) by telitacicept would inhibit B-cell proliferation and maturation. This suppression at the proximal portion of the immune response could alleviate autoimmune symptoms.

This study is a randomized, double-blind, placebo-controlled Phase 3 study with an open-label extension to evaluate the efficacy and safety of telitacicept in a global patient population with gMG.

Study Timeline

• Study First Submitted: 2024-06-07
• Study First Submitted QC: 2024-06-07
• Study First Posted: 2024-06-13
• Study Start: 2024-07-17
• Status Verified: 2025-07
• Last Update Submitted: 2025-07-11
• Last Update Posted: 2025-07-15
• Primary Completion: 2026-07
• Study Completion: 2027-10-01

Recruitment & Eligibility

• Status: RECRUITING
• Sex: Not specified
• Target Recruitment: 72

Inclusion Criteria

1. Patients must provide signed informed consent to participate in the study and agreed to compliant with the study procedure.

2. Male or female patient aged ≥18 years at screening.

3. Patients have prior confirmed diagnosis of gMG with generalized muscle weakness (typical pattern of weakness, eg, predominantly proximal, fatigable, fluctuating in severity, more severe in the evening, and improved with rest) meeting the clinical criteria for diagnosis of MG as defined by the Myasthenia Gravis Foundation of America (MGFA) clinical classification II-IV. Patient’s diagnosis should meet at least 1 of the following tests: a. History of abnormal neuromuscular transmission demonstrated by single fiber electromyography or repetitive nerve stimulation, OR b. History of positive edrophonium chloride test, OR c. Improvement in MG signs on oral acetylcholine esterase (AChE) inhibitors as assessed by the treating physician.

4. Patients have positive antibodies against AChR or MuSK at screening.

5. MG‐ADL score ≥6 points at screening and baseline with ocular-related score <50% of the total score.

6. QMG score ≥11 points at screening and baseline.

7. Up to 2 concomitant medications for the treatment of MG are permitted if they meet the stability criteria prior to baseline

8. Patients agree to use highly effective contraception

9. Patients are appropriately vaccinated (e.g., vaccinations for pneumococcus, influenza, and COVID-19) per investigator's clinical judgement considering the patient's risk factors and according to country and local guidelines. Meningococcal vaccination is not required prior to initiating treatment with telitacicept

Exclusion Criteria

1. Patients have been diagnosed with any other autoimmune disease(s), eg, rheumatoid arthritis, Sjogren’s syndrome. Note: Patients with abnormal thyroid function at screening will be excluded from the study. However, patients with a documented history of hypothyroidism or hyperthyroidism who have been adequately treated and whose laboratory results are within the normal range at screening can be included.

2. Patients have current or history of primary immunodeficiency.

3. Patients have history of malignancy within the last 5 years, except for adequately treated nonmelanoma skin cancer (eg, basal or squamous cell carcinoma) or carcinoma in situ of the cervix.

4. Patient have prior or continuing diagnosis of serious cardiovascular disease(s) (including severe arrhythmias), liver, kidney, respiratory system, endocrine (eg, poorly controlled type I or type IIdiabetes mellitus, defined as glycosylated hemoglobin A1c [HbA1c] >8%), or hematologic disease(s), or other medical conditions that, in the opinion of the investigator, Medical Monitor, and/or Study Sponsor, could reasonably prevent the patient from safely participating in the study or from compliance to study procedures.

5. Patients have a known allergy to human biological products or any of the listed excipients.

6. Patients are currently dependent on alcohol/drugs (including marijuana) or have a history of alcohol/drug (including marijuana) dependence or addiction that, in the opinion of the investigator, may adversely affect patient safety or prevent patient compliance with study procedures.

7. History of a suicidal attempt within the past 12 months, or current suicidal, intent or behavior during the screening period according to the Columbia suicide severity rating scales (C-SSRS).

8. Women who are currently breastfeeding or intend to breastfeed during the study period.

9. Patients have abnormal laboratory parameters at screening

10. Patients have received prohibited immunosuppressants other than protocol permitted stable concomitant medication (per Inclusion Criterion 7, see protocol Table 7), biologics or other agents with protocol defined time period prior to randomization.

11. Patients have received intravenous immunoglobulin or plasma exchange therapy ≤4 weeks before randomization.

12. Patients have received live or attenuated vaccine ≤4 weeks prior to screening or during the study.

13. Patients have participated in any interventional clinical trial or received an investigational treatment ≤3 months or within a period of 5 times of study drug’s half-life (whichever is longer).

14. Patients have acute or chronic infection prior to randomization.

15. Patients receiving treatment for any chronic infection (eg, tuberculosis, pneumocystis, cytomegalovirus, herpes simplex virus, herpes zoster, atypical mycobacterium, etc).

16. Patients have thymoma within 5 years or have received thymectomy ≤6 months prior to screening.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Change from baseline in Myasthenia Gravis Activities of Daily Living (MG-ADL) score at Week 24		Change from baseline in Myasthenia Gravis Activities of Daily Living (MG-ADL) score at Week 24

Secondary Outcome Measures

Name	Time	Method
• Change from baseline in MG Quality of Life scale (MG-QOL15r) at Week 24		• Change from baseline in MG Quality of Life scale (MG-QOL15r) at Week 24
• Proportion of patients with a decrease of ≥2 points from baseline in MG-ADL score at Week 24		• Proportion of patients with a decrease of ≥2 points from baseline in MG-ADL score at Week 24
• Proportion of patients with a decrease of ≥3 points from baseline in QMG score at Week 24		• Proportion of patients with a decrease of ≥3 points from baseline in QMG score at Week 24
• Proportion of patients who achieved minimal symptomatic expression (MSE, defined as having MG-ADL score of 0 or 1) at Week 24		• Proportion of patients who achieved minimal symptomatic expression (MSE, defined as having MG-ADL score of 0 or 1) at Week 24
Change from baseline in Quantitative Myasthenia Gravis (QMG) score at Week 24		Change from baseline in Quantitative Myasthenia Gravis (QMG) score at Week 24

Trial Locations

Locations (54)

Los Angeles Site; 🇺🇸 Los Angeles, California, United States

Orange Site; 🇺🇸 Orange, California, United States

San Francisco Site; 🇺🇸 San Francisco, California, United States

Boca Raton, Florida Site; 🇺🇸 Boca Raton, Florida, United States

Miami, Florida Site; 🇺🇸 Miami, Florida, United States

Port Charlotte, Florida Site; 🇺🇸 Port Charlotte, Florida, United States

Augusta Site; 🇺🇸 Augusta, Georgia, United States

Kansas City Site; 🇺🇸 Kansas City, Kansas, United States

Lexington Site; 🇺🇸 Lexington, Kentucky, United States

Louisville Site; 🇺🇸 Louisville, Kentucky, United States

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