Randomized, Double-blind, Double-dummy Trial of Mycophenolic Acid Versus Azathioprine in the Treatment of Corticosteroid-refractory Myasthenia Gravis
试验速览
- 阶段
- 不适用
- 发起方
- 入组人数
- 40
- 主要终点
- The ratio of two arms patients achieve minimal manifestation (MM, i.e. complete remission)
研究概览
简要总结
This is an randomized, double-blind, double-dummy trial, and the objective is to compare the efficacy and safety of Mycophenolic acid (MA) and Azathioprine (AZA), immunosuppressive drugs, in myasthenia gravis patients. This prospective study will enroll 40 myasthenia gravis (MG) patients who are poor controlled under prior steroid therapy. All subjects should be randomly assigned to MA group and AZA group that will receive routine pyridostigmine and prednisolone in combination with MA or AZA.
详细描述
This will be a double-dummy study to keep the blinded quality.
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MA group: 1 tablet AZA placebo and 4 tables MA (180 mg/tab,720 mg/day) twice daily.
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AZA group: 1 tablet AZA (50mg/tab) and 4 tables MA placebo twice daily.
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When patients achieve minimal manifestation (MM, i.e. complete remission), which lead to normal daily routine, the dose of pyridostigmine should reduce to 240 mg/day (4 tablets) or less. The dose of steroid should be stepped down by 10 mg qod (every other day) for every 2 weeks until the dose achieves 40 mg qod. After that, the dose should be stepped down by 5 mg qod for every month.
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When disease progresses and is no longer maintaining minimal manifestation, the dose of steroid will be stepped up by 10 mg qod for every 2 weeks until achieve clinical stable remission. The taper rule of steroid could start again 1 month after stabilization.
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Every patient will be treated for 1 year. If the patient could not achieve MM within 1 year, the blind of individual patient will be opened and the patients will be crossed over to another medical treatment. The efficacy and safety of second medication will be observed openly until the end of study.
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When the muscle weakness worsens under established study schedule, plasmapheresis could be conducted to improve the condition rapidly.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Crossover
- 主要目的
- Treatment
- 盲法
- Double (Participant, Investigator)
入排标准
- 年龄范围
- 20 Years 至 70 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female age between 20-70 (including 20 and 70 years old).
- •Osserman II and III Myasthenia Gravis.
- •Positive serum anti-acetylcholine receptor antibodies.
- •Poor control of disease with daily dose of prednisone ≥ 30 mg or 0.5 mg/kg at 3 months before enrollment.
- •Without immunosuppressive therapy other than steroid.
排除标准
- •Ocular MG or minimal clinical syndrome that would not require the therapy of steroids.
- •Negative serum anti-acetylcholine receptor antibodies.
- •Use immunosuppressants other than steroids in the preceding year.
- •Previous use other investigational medication within 3 months or current participate other clinical study.
- •Poor renal function: serum creatinine > 3.0 mg/dl or estimated creatinine clearance < 30 ml/min
- •Females who are pregnancy or breast-feeding.
- •Recent history, within 5 years, of malignancy
- •Unwilling or unable to participate the necessary continuous visits and examinations.
研究组 & 干预措施
MA
MA group: 1 tablet AZA placebo and 4 tablets MA (180mg/tab,720 mg/day) twice daily
干预措施: Mycophenolic acid (Drug)
AZA
AZA group: 1 tablet AZA (50mg/tab) and 4 tablets MA placebo twice daily
干预措施: AZA (Drug)
结局指标
主要结局
The ratio of two arms patients achieve minimal manifestation (MM, i.e. complete remission)
时间窗: One year after treatment
次要结局
- Osserman clinical classification(One year after treatment)
- Myasthenia gravis (MG) score(One year after treatment)