Open Label Trial of Ranolazine in Myotonia Congenita, Paramyotonia Congenita, & Myotonic Dystrophy Type 1
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 35
- 试验地点
- 1
- 主要终点
- Questionnaires: Short Form Health Survey (SF-36) and Individualized Neuromuscular Quality of Life Questionnaire (INQoL)
研究概览
简要总结
The purpose of this study is to gather preliminary data to determine if ranolazine is a safe and effective treatment for the symptoms of myotonia congenital, paramyotonia congenita, and myotonic dystrophy type 1. The duration of the study is 5 weeks.
详细描述
Recent advances in the understanding of myotonia congenita have identified potential areas that could possibly respond to treatment in a drug study. The drug ranolazine (trade name Ranexa) is a FDA-approved medication to treat chest pain in patients with heart disease. Ranolazine has been studied in mice with myotonia congenita. The data from this animal model suggest that ranolazine may improve the symptoms and signs of myotonia. All individuals that participate will be placed on active drug. The investigators want to see if this drug is safe to take without causing too many side effects for people with myotonia congenita, paramyotonia congenital and myotonic dystrophy type 1. Participants will go to The Ohio State University for study visits. Participants will take ranolazine for four weeks. Participants can expect a total of 4 study visits and 2 phone calls over the 5 week period.
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 100 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of myotonia congenital, paramyotonia congenital or Myotonic Dystrophy Type 1 established by genetic testing in the subject or in a first-degree relative.
- •Clinically evident myotonia
排除标准
- •Contraindications to ranolazine use:
- •for fungus infection: ketoconazole (Nizoral), itraconazole (Sporanox, Onmel)
- •for infection: clarithromycin (Biaxin)
- •for depression: nefazodone
- •for HIV: nelfinavir (Viracept), ritonavir (Norvir), lopinavir and ritonavir (Kaletra), indinavir (Crixivan), saquinavir (Invirase).
- •for tuberculosis (TB): rifampin (Rifadin), rifabutin (Mycobutin), rifapentine (Priftin)
- •for seizures: phenobarbital, phenytoin (Phenytek, Dilantin, Dilantin-125), carbamazepine (Tegretol)
- •the herbal supplement St. John's wort
- •you have scarring (cirrhosis) of your liver
- •Concurrent use of mexiletine, lacosamide, acetazolamide, phenytoin, quinine, procainamide, Saint John wort or tocainide. Patients who were previously treated with these medications may participate. They need to be off of the medication for at least a week prior to enrollment.
- •QTc >470 ms for men and >480 ms for women.
- •Women who are pregnant or breastfeeding
- •Direct family history of sudden cardiac death
研究组 & 干预措施
ranolazine
ranolazine 500mg, twice daily for two weeks; 1000mg twice daily for 2 weeks
干预措施: Ranolazine (Drug)
结局指标
主要结局
Questionnaires: Short Form Health Survey (SF-36) and Individualized Neuromuscular Quality of Life Questionnaire (INQoL)
时间窗: 1 month
quality of life measurements for overall health and neuromuscular disease
Muscle tasks
时间窗: 1 month
The subject is observed and timed while rising from an arm chair, walking 3 meters, turning, walking back, and sitting down again
Electromyography (EMG) Myotonia
时间窗: 1 month
To see if the electrical potentials produced by the muscle fibers change.
次要结局
- Electrocardiogram (ECG)(1 month)
研究者
William Arnold
Associate Professor
Ohio State University