A Phase III Trial of Telitacicept in Patients With Ocular Myasthenia Gravis
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 120
- 试验地点
- 1
- 主要终点
- Change from baseline in the MGII (PRO) ocular score
研究概览
简要总结
This is a Phase III, multicenter, randomized, double-blind, placebo-controlled study designed to evaluate the efficacy and safety of Telitacicept for the treatment of Ocular Myasthenia Gravis (OMG).Approximately 120 eligible subjects aged 12 to 80 years with a diagnosis of OMG (Myasthenia Gravis Foundation of America [MGFA] Clinical Classification Type I) will be randomized in a 1:1 ratio to receive either Telitacicept or a matching placebo. Subjects must be on a stable standard-of-care therapy and have an MG Impairment Index (PRO) ocular score of ≥6 at screening and baseline.The dose is age and weight based.The primary objective is to evaluate the efficacy of Telitacicept compared to placebo in treating OMG.The primary efficacy endpoint is the change from baseline in the MGII (PRO) ocular score at Week 24. Secondary endpoints include changes from baseline in other ocular and total scores from MGII, Myasthenia Gravis-Activities of Daily Living (MG-ADL), MG Clinical Absolute Score, and the 15-item Myasthenia Gravis Quality of Life Revised scale (MG-QOL15r). Safety and tolerability will be monitored throughout the study.
详细描述
This is a Phase III, multicenter, randomized, double-blind, placebo-controlled, parallel-group study to assess the efficacy and safety of Telitacicept in subjects with Ocular Myasthenia Gravis (OMG). The study will be conducted at multiple centers.
Eligible subjects will be randomized in a 1:1 ratio to one of two treatment arms:
Arm 1: Telitacicept Arm 2: Placebo Randomization will be stratified by two factors: Acetylcholine Receptor (AChR) antibody status (positive vs. negative) and age (<18 years vs. ≥18 years).
Primary Objective:
To evaluate the efficacy of Telitacicept compared to placebo in the treatment of subjects with Ocular Myasthenia Gravis.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Double (Participant, Investigator)
入排标准
- 年龄范围
- 12 Years 至 80 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Voluntarily signed the informed consent form.
- •Age 12 to 80 years, inclusive, male or female.
- •Body weight ≥30 kg.
- •Diagnosis of Myasthenia Gravis (MG) with documented clinical features consistent with the disease.
- •Myasthenia Gravis Foundation of America (MGFA) Clinical Classification of Type I.
- •On a stable standard-of-care (SOC) treatment regimen.
排除标准
- •Concomitant autoimmune diseases requiring systemic corticosteroid therapy.
- •Clinically significant laboratory abnormalities.
- •Use of other immunosuppressants (not part of the stable SOC) within 1 month prior to randomization.
- •Presence of an acute or chronic infection requiring treatment.
- •Current active hepatitis or history of severe liver disease.
- •Positive for HIV antibodies.
- •Positive for syphilis antibodies (non-specific or specific).
- •Poorly controlled diabetes mellitus, defined as HbA1c >9.0% or fasting blood glucose ≥11.1 mmol/L.
- •Subjects with thymoma (classified as ≤ Stage II for benign and ≥ Stage III for malignant according to the Masaoka staging system) .
- •Presence of uncontrolled chronic degenerative diseases, psychiatric disorders, or neurological diseases other than MG that could interfere with study assessments.
- •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.).
- •Known allergy to human-derived biological products.
研究组 & 干预措施
Placebo
干预措施: Placebo (Drug)
Telitacicept
干预措施: Telitacicept (Drug)
结局指标
主要结局
Change from baseline in the MGII (PRO) ocular score
时间窗: From enrollment to the end of treatment at 24 weeks
at Week 24
次要结局
- Change from baseline in the total MGII score(From enrollment to the end of treatment at 24 weeks)
- Change from baseline in the MG-QOL15r total score(From enrollment to the end of treatment at 24 weeks)
- Change from baseline in the total MG-ADL score(From enrollment to the end of treatment at 24 weeks)
- Change from baseline in the MGII (PRO + PE) ocular score(From enrollment to the end of treatment at 24 weeks)
- Change from baseline in the MG-ADL ocular score(From enrollment to the end of treatment at 24 weeks)
- Change from baseline in the MG Clinical Absolute Score ocular score(From enrollment to the end of treatment at 24 weeks)
- Incidence and severity of adverse events(From signing of informed consent until 4 weeks after the last dose.)