Treatment of Myotonia - Lamotrigine Versus Namuscla
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- 入组人数
- 32
- 试验地点
- 1
- 主要终点
- Change in Myotonia Behavior Scale (MBS)
研究概览
简要总结
In this clinical study, the aim is to investigate whether there is a difference in treatment of myotonia using two drugs. A difference there can justify the significantly higher cost when treated by Namuscla versus Lamotrigine.
According to the current corona pandemic, the investigators designed an app to use for data collection in the study. The app also ensures that patients who live far from the clinic more easily can participate.
详细描述
On 18 May 2020, the National Board of Health in Denmark approved Namuscla for the treatment of myotonia. Myotonia is a congenital muscle disease that delays muscle relaxation after a muscle contraction. Myotonia can be disabling, but with treatment, most are able to work. Namuscla is the first registered drug to treat myotonia, however, the active part mexiletine is not new. It has been used off-label to treat myotonia for decades. The price for the new preparation is high with a daily cost (approx. 70 EU/dollar). In 2017, the investigators found evidence that treatment with lamotrigine is efficacious against myotonia. Treatment with lamotrigine can reduce the price to 150 EU/dollar per year.
Therefore, the aim of the study is to investigate whether there is a difference in the effects om myotonia and side effects of the two drugs, which can justify the significantly higher cost when treated with Namuscla in a double blinded, inferiority, cross-over study lasting 4 months.
According to the current corona pandemic, the study is designed without need of physical attendance so that inclusion and collection of experimental data can take place, by phone conversations, video-guided testing, and diary registration via a study-App. It also ensures that patients who live far away more easily can participate. Blood tests and ECGs can be performed locally.
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
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
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
- •Capable adult women and men (age ≥ 18 years).
- •Diagnosed with Myotonia Congenita (Becker and Thomsen type), Paramyotonia Congenita, or Hyperkalemic periodic paralysis.
- •Myotonia under treatment or which significantly limits the daily activities (MBS> 2).
排除标准
- •Allergy to lamotrigine, mexiletine, or the inactive ingredients in trial medication.
- •Disease, which is affected by trial medication such as heart disease (ischemia and arrhythmia), epilepsy, and significant renal or hepatic failure.
- •Treatment that, in the opinion of the project manager, can affect the study result - medication with significant interactions with trial medication.
- •In case of smoking, start or cessation during the study.
- •Pregnant or breastfeeding during the study period. Fertile women with a positive pregnancy test at the time of entry into the trial, or who do not use safe contraception during the project period.
研究组 & 干预措施
Lamotrigine
An escalation phase of 28 days:
- tablet Lamotrigine 25 mg once daily in 14 days followed by 50 mg once daily in 14 days.
A treatment phase of 30 days:
- tablet Lamotrigine 100 mg, once daily in 10 days, twice daily in 10 days, followed by third daily in 10 days.
干预措施: Lamotrigine (Drug)
Namuscla
A placebo phase of 28 days:
- tablet placebo 25 mg once daily in 14 days followed by 50 mg once daily in 14 days.
A treatment phase of 30 days:
- tablet Namuscla 167 mg, once daily in 10 days, twice daily in 10 days, followed by third daily in 10 days.
干预措施: Namuscla (Drug)
结局指标
主要结局
Change in Myotonia Behavior Scale (MBS)
时间窗: baseline and week 8
Each participant chose one of six statements about how myotonia affect their daily living. MBS is register once daily in a week. 1 is no symptoms of myotonia, 6 is invalidating symptoms of myotonia. Measured without treatment and in the last week of treatment.
次要结局
- Change in hand-myotonia(baseline and week 8)
- Change in time-up-and-go-test (TUG)(baseline and week 8)
- Change in Individualized Neuromuscular Quality of Life Questionnaire (INQoL)(baseline and week 8)
- Days with Side effects (SE)(through study completion, in total 123 days)
- Change in Side Effect Scale (SE)(baseline and week 8)
- Change in Eye-myotonia(baseline and week 8)
研究者
Grete Andersen, MD
Principal Investigator
Rigshospitalet, Denmark