NCT04431895已完成早期 1 期
A Prospective,Single-center,Single-Arm,Single-Blind Pilot Study To Investigate The Efficacy Of Tofacitinib In Patients With Refractory Myasthenia Gravis
适应症
干预措施
相关药物
试验速览
- 阶段
- 早期 1 期
- 状态
- 已完成
- 入组人数
- 19
- 试验地点
- 1
- 主要终点
- Change From Baseline in Quantitative Myasthenia Gravis (QMG) Scores at Month 6
研究概览
简要总结
This is a 6 month study that will evaluate the efficacy of 5mg tofacitinib tablet taken twice a day in patients with refractory myasthenia gravis. Twenty adult participants will receive a four month treatment of tofacitinib followed by elution for one month.
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 70 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •fluctuating muscle weakness and fatigability classified as the Myasthenia Gravis Foundation of America (MGFA) clinical classification Type IIa-IVa;
- •more than 10% amplitude decrement in low frequency repetitive nerve stimulation, and less than 10% amplitude increment in high frequency repetitive nerve stimulation;
- •understanding and assigning the informed consent form, and having a good compliance with treatment.
- •meet all of the criteria in above as well as at least one of the below:
- •refractory: insufficient response to full dose- and course- steroids treatment (0.75-1mg per kg per day for at least 3-6 months), failure to respond adequately to tacrolimus (no less than 3mg per day) for 3 months;
- •severe or intolerable adverse effects from conventional immunosuppressive therapy.
排除标准
- •positive for muscle-specific kinase (MuSK) antibodies;
- •a tumor history except for thymoma;
- •a history of Type B and Type C hepatitis;
- •a history of tuberculosis or T-SPOT.TB tset positivity;
- •hepatic, renal and cardial insufficiency (baseline: ALT/AST>50U/L;BNP>200pg/ml）;
- •severe allergy or infection, or chronic or recurrent infection;
- •pregnancy;
- •hyperlipidemia;
- •participating other clinical trials
研究组 & 干预措施
tofacitinib 5mg twice a day
Experimental
干预措施: Tofacitinib 5 MG (Drug)
结局指标
主要结局
Change From Baseline in Quantitative Myasthenia Gravis (QMG) Scores at Month 6
时间窗: Baseline, Month 6
A quantitative MG scoring system (QMG Score) is essential in the objective evaluation of therapy for MG.This scoring system is based on quantitative testing of sentinel muscle groups. Total QMG score range from 0 (extreme disease severity) to 39 (none), higher score indicated less disease severity.
次要结局
- Change From Baseline in Myasthenia Gravis-related Activities of Daily Living (MG-ADL) Scores at Month 6(Baseline, Month 6)
- Change From Baseline in Myasthenia Gravis Composite (MGC) Scores at Month 6(Baseline, Month 6)
- Change From Baseline in Myasthenia Gravis-Quality of Life Questionnaire-15 item (MG-QOL15) Scores at Month 6(Baseline, Month 6)
- Change From Baseline in Doses of Steroids at Month 6(Baseline, Month 6)
研究者
研究点 (1)
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