Effect of Pyridostigmine (Mestinon) on Muscle Strength in Myasthenia Gravis
试验速览
- 阶段
- 4 期
- 入组人数
- 44
- 试验地点
- 1
- 主要终点
- Change in QMG
研究概览
简要总结
A randomized, placebo-controlled, double-blinded cross-over study evaluating and quantifying the effect of pyridostigmine on muscle strength and symptoms in Myasthenia Gravis (MG)
详细描述
The study aims to evaluate and quantify the effect of pyridostigmine on symptoms and muscle strength in newly-diagnosed patients and patients on stable medication.
Pyridostigmine treatment is initiated in the vast majority of MG patients. No studies have quantified the effect in a randomized trial, and no studies have examined the potential difference in effect in newly diagnosed patients as compared to patients on stable, antimyasthenic medications.
The study will investigate the effect in two groups
- Newly diagnosed, treatment-naive patients.
- MG patients on stable antimyasthenic medication.
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
- 18 Years 至 90 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •MG verified by a) anti-body, or b) single-fiber EMG and/or decrement on ENG.
排除标准
- •Anti-MuSK
- •Known cardio-pulmonary disease
- •Known neuropathy
- •Known myopathy
- •Known malignant disease
- •Pregnancy or breastfeeding
- •Mechanic ileus, urinary tract obstruction, peritonitis
- •De-novo MG Eligibility Criteria
- •MG diagnosis < 2 months, no prior antimyasthenic medications
- •Chronic MG Eligibility Criteria
- •MG diagnosis > 1 year, and stable pyridostigmine dosis
研究组 & 干预措施
Active
De-novo: Each capsule contains 60 mg. pyridostigmine. 1 capsule is administered twice within 4 hours.
Chronic: Each capsule contains 60 mg. pyridostigmine. Number of administered capsules per dosage depend on the patient's usual dosage. Study drug is administered twice within 4 hours.
Patients are examined/rated before 1st dose, 1 hour after 1st dose, 1 hour after 2nd dose (Visit 1). After cross-over (Visit 2), patients will be rated open-label at 1 month (Visit 3) and 3 months (Visit 4).
干预措施: Pyridostigmine (Drug)
Placebo
Same as "Active", however capsules contain placebo.
干预措施: Placebo oral capsule (Drug)
结局指标
主要结局
Change in QMG
时间窗: At baseline, 1 hour after 1st dose (administered immediately after baseline assessment), 1 hour after 2nd dose (administered 2 hours after 1st dose). Assessed on Day 1 and Day 2 (cross-over), at Follow-up 1 (1 month) and Follow-up 2 (3 months).
Quantitative Myasthenia Gravis (QMG) scale rate disease severity of myasthenia gravis on 13 items with a total score ranging from 0-39 (higher values representing more severe disease). Total score (0-39), ocular subitems (0-6), bulbar subitems (0-9), extremity/axial subitems (0-21) and respiratory subitem (0-3) will be reported.
次要结局
- Change in muscle strength as assessed by dynamometry (Biodex System 3).(At baseline, 1 hour after 1st dose (administered immediately after baseline assessment), 1 hour after 2nd dose (administered 2 hours after 1st dose). Assessed on Day 1 and Day 2 (cross-over), at Follow-up 1 (1 month) and Follow-up 2 (3 months).)
- Change in MG Composite Score(At baseline, 1 hour after 1st dose (administered immediately after baseline assessment), 1 hour after 2nd dose (administered 2 hours after 1st dose). Assessed on Day 1 and Day 2 (cross-over), at Follow-up 1 (1 month) and Follow-up 2 (3 months).)
- Change in muscle fatigue as assessed by dynamometry (Biodex System 3).(At baseline, 1 hour after 1st dose (administered immediately after baseline assessment), 1 hour after 2nd dose (administered 2 hours after 1st dose). Assessed on Day 1 and Day 2 (cross-over), at Follow-up 1 (1 month) and Follow-up 2 (3 months).)
研究者
Jan Lykke Scheel Thomsen
MD, PhD Fellow
University of Aarhus