Pilot Study on the Response of Inherited Erythromelalgia Patients With NaV1.7 Mutations to Carbamazepine: Clinical Imaging Study
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 发起方
- 入组人数
- 2
- 试验地点
- 2
- 主要终点
- Carbamazepine Affects Pain in Patients With S241T NaV1.7 IEM Mutation
研究概览
简要总结
This research study is designed to investigate brain response using fMRI scan, and behavioral responses, to treatment with the drug carbamazepine (CBZ) in patients with the painful sodium channelopathy inherited Erythromelalgia (IEM). This study is designed to identify the central nervous system (CNS) regions that are activated during ongoing or evoked pain attacks, and the altered CNS response to CBZ treatment. This will advance our understanding of how IEM affects the brain. We also hope to validate a pharmacogenic approach to the study of IEM by use of an FDA approved drug. We hope, but cannot be sure, that subjects will directly benefit from this 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, Care Provider)
入排标准
- 年龄范围
- 18 Years 至 80 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •diagnosis/symptoms of EM
- •specific NaV1.7 sodium channel mutations (including S241T)
排除标准
- •patients with no identified NaV1.7 mutation
研究组 & 干预措施
Carbamazepine
Administered in gradual doses from 200 mg twice a day increasing to no more than 800 mg twice a day if symptoms do not reduce. After 2 weeks of steady dose drug will be tapered down every 3 days.
干预措施: Carbamazepine (Drug)
Placebo
Administered in gradual doses from 200 mg twice a day increasing to no more than 800 mg twice a day if symptoms do not reduce to match dosing with carbamazepine. After 2 weeks of steady dose drug will be tapered down every 3 days.
干预措施: Placebo (Drug)
结局指标
主要结局
Carbamazepine Affects Pain in Patients With S241T NaV1.7 IEM Mutation
时间窗: 15 days
次要结局
- Carbamazepine Affects Mean Duration of Pain Episode(15 days)