Beta-agonist Efficacy and Tolerability as Adjuvant Therapy in Myasthenia Gravis
试验速览
- 阶段
- 2 期
- 入组人数
- 30
- 试验地点
- 2
- 主要终点
- Myasthenia Gravis Quality of Life 15-items (MG-QOL15)
研究概览
简要总结
This study examines the effect of adjuvant therapy with the oral beta-agonist Salbutamol in patients with generalized myasthenia gravis on stable standard of care having residual symptoms.
详细描述
Myasthenia Gravis (MG) causes various degrees of increased muscular fatigue and ocular, bulbar, respiratory and extremity symptoms.
Residual symptoms often remain despite treatment with acetylcholinesterase inhibitors and immunosuppressive agents. Escalation of immunosuppressive treatment may provide additional benefit but is associated with potentially severe side effects, and high economic costs.
Treatment with beta-agonists has been investigated in animal models of MG, and in small, randomized pilot studies of generalized MG. Adjuvant therapy with oral beta-agonists in MG may be safe and cheap and may improve symptoms.
The trial will examine the tolerability and efficacy of adjuvant therapy with the oral beta-agonist Salbutamol in patients with generalized myasthenia gravis on stable standard of care having residual symptoms.
Present study is an investigator-initiated, randomized, placebo-controlled, rater and subject-blinded crossover study.
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 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Generalized myasthenia gravis (MGFA IIa-IVb) at screening, verified by ≥ 1 of the following: 1) AchR-antibodies in medical history, 2) Abnormal decrement on repetitive nerve stimulation in medical history
- •Disease duration of ≥ 1 year
- •Stable dose of antimyasthenic medications at screening
- •Residual symptoms with a MG-QOL15 score of ≥ 10
- •Age ≥ 18 years
- •Ability to understand the requirements of the trial and provide written, informed consent
排除标准
- •Evidence of malignancy ≤ 3 years prior to screening, unless deemed completely cured
- •Thymectomy ≤ 6 months prior to screening
- •Impending MG crisis or respiratory insufficiency
- •Worsening of MG symptoms due to other diseases or medications (e.g. infection, beta-blockers, aminoglycosides, etc.)
- •Other factor(s) or medical condition(s) that may explain residual symptoms
- •Pregnancy or breast-feeding
- •Treatment with beta-agonists
- •Uncontrolled diabetes
- •Ischemic Heart Disease, Cardiac Arrhythmia or Heart Failure (including hypertrophic cardiomyopathy)
- •Uncontrolled Hypertension (≥ 160/110)
- •Known hypersensitivity to any of the study drug components
- •Treatment with tricyclic antidepressants, monoamineoxidase inhibitors, digoxine, or methylxanthines.
研究组 & 干预措施
Active
Active intervention arm. Treatment for 8 weeks per treatment period.
干预措施: Salbutamol 4Mg Tablet (Drug)
Placebo
Placebo arm. Treatment for 8 weeks per treatment period.
干预措施: Placebo oral capsule (Drug)
结局指标
主要结局
Myasthenia Gravis Quality of Life 15-items (MG-QOL15)
时间窗: 8 weeks (Treatment Period 1), 8 weeks (Treatment Period 2)
Validated patient reported outcome-questionnaire consisting of 15 items and their impact on quality of life. Results are reported as change from baseline to 8 weeks of treatment in both treatment periods (Visit 4 compared to Visit 2, and Visit 7 compared to Visit 5).
Treatment Tolerability
时间窗: 8 weeks (Treatment Period 1), 8 weeks (Treatment Period 2)
Tolerability assessed by rate of adverse events and drug discontinuation in both treatment periods.
次要结局
- Myasthenia Gravis Composite (MG-Composite)(8 weeks (Treatment Period 1), 8 weeks (Treatment Period 2))
- Myasthenia Gravis Activity of Daily Living (MG-ADL)(8 weeks (Treatment Period 1), 8 weeks (Treatment Period 2))
- Quantitative Myasthenia Gravis (QMG)(8 weeks (Treatment Period 1), 8 weeks (Treatment Period 2))
- Neuro QOL(8 weeks (Treatment Period 1), 8 weeks (Treatment Period 2))
研究者
Jan Lykke Scheel Thomsen
MD, PhD Fellow
University of Aarhus