The Efficacy and Safety of Telitacicept Combined With Low-dose Steroids in Patients With Refractory Myasthenia Gravis
试验速览
- 阶段
- 4 期
- 状态
- 尚未招募
- 入组人数
- 30
- 主要终点
- The change in patients' ADL scores
研究概览
简要总结
This study is designed to explore the efficacy and safety of Telitacicept combined with low-dose steroids for the treatment of refractory MG, and to investigate related biomarkers such as immunoglobulins, BlyS/APRIL, and AChR-Ab titers, in order to clarify whether Telitacicept can rapidly and effectively help achieve MG treatment goals and assist in steroid reduction.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 85 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age between 18-85 years, both genders included；
- •Meet the diagnostic criteria of the 2020 Chinese MG guidelines, with positive serological testing for AChR-Ab;
- •Clinical classification of Type I to Type IVa according to the MGFA;
- •Meet the criteria for refractory MG in the 2022 Japanese MG guidelines: poor response to standard treatment, intolerance to standard treatment drugs due to adverse reactions, frequent relapses/exacerbations after reduction of standard treatment drugs, frequent need for rescue treatment due to disease fluctuations, frequent myasthenic crises, and comorbidities that limit the use of standard treatment;
- •Patients with unstable symptoms (MG-ADL score ≥6 or QMG ≥8) despite treatment with standard therapeutic regimens before enrollment, defined as follows:
- •Patients on monotherapy with corticosteroids: a corticosteroid dose ≤60mg/d, and a stable dose for at least 1 month before enrollment;
- •Patients on combination therapy with corticosteroids and other immunosuppressants: a corticosteroid dose ≤60mg/d, and a stable dose for at least 1 month before enrollment, while other immunosuppressants such as azathioprine, methotrexate, tacrolimus, and mycophenolate mofetil have been stable for 6 months prior to the study start and will remain stable during the study period;
- •Patients must provide written informed consent.
排除标准
- •Patients with active infections, such as herpes zoster, HIV, active pulmonary tuberculosis, or active hepatitis;
- •Patients with thymic tumors or those who have undergone thymectomy within the past 6 months;
- •Patients with coexisting malignant tumors;
- •Patients with severe hepatic or renal insufficiency;
- •Patients who have received intravenous immunoglobulin or undergone plasmapheresis within the last 2 months;
- •Patients who have received any live vaccines within the last 3 months or plan to receive any vaccines during the study period;
- •Women who are currently pregnant or breastfeeding, and patients who plan to conceive during the trial period;
- •Patients with allergies to human-derived biological products;
- •Patients who have participated in any clinical trial within the last 28 days or within 5 half-lives of the study medication (whichever is longer);
- •Any other patients deemed unsuitable for enrollment by the investigator (e.g., severe psychiatric disorders).
研究组 & 干预措施
Patients with refractory MG treated with Telitacicept combined with low-dose steroids
This is an open-label, single-arm exploratory study of Telitacicept (240mg weekly, then every two weeks after achieving MMS or QMG reduction of ≥ 6 points) combined with a gradual reduction of steroids and other immunosuppressants. When the steroid dose is reduced to 5mg/day or 10mg/every other day, Telitacicept can be reduced to 160mg, administered subcutaneously every two weeks.
干预措施: Telitacicept (Drug)
结局指标
主要结局
The change in patients' ADL scores
时间窗: from baseline to week 52
The variation in ADL scores at 52 weeks compared to baseline in patients. Total MG -ADL scores range from 0 (normal) to 24 (severe).
The change in patients' QMG scores
时间窗: from baseline to week 52
The variation in QMG scores at 52 weeks compared to baseline in patients. Total QMG scores range from 0 (none) to 39 (severe).
次要结局
- MGFA-PIS(from baseline to week 52)
- the average daily corticosteroid usage(from baseline to week 24；from baseline to week 52)
- the usage of traditional non-steroidal immunosuppressants(from baseline at weeks 24 and 52)
- Number of relapses(week 52)
- AUDTC(week 52)
- Proportion of patients with a corticosteroid dose ≤10mg/d(at weeks 24 and 52)
- Proportion of patients achieving MMS with a corticosteroid dose ≤ 5mg/d(week 24)
- MG QoL15r scores(from baseline at weeks 24 and 52)
- safety（Incidence, severity, and outcome of adverse events）(52 weeks)
- relevant biomarkers(from baseline at week 52)