A Pilot, Randomized, Double-Blind, Placebo-Controlled Phase I Study to Determine the Safety and Efficacy of Ampicillin in Treating Primary Dystonia Symptoms
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 2
- 试验地点
- 1
- 主要终点
- Safety and tolerability of Ampicillin in treating DYT-1 dystonia
研究概览
简要总结
The purpose of this study is to evaluate if the antibiotic Ampicillin is safe and tolerated in patients that have generalized dystonia caused by the DYT-1 gene mutation, as compared to patients treated with a placebo. A placebo is a pill that looks and tastes the same as the real drug, but without the active ingredient. The second objective of this study is to determine if dystonia symptoms improve while on the study drug.
详细描述
This is a double-blinded and randomized drug study: neither the patient nor the investigator know if patients are taking study drug (Ampicillin) or placebo.
Three study visits will include neurological exams, review of medical history, genetic test results and video-taped BFM-DRS motor scales. Patients will receive medication during baseline visit, consisting of either Ampicillin drug or placebo, which will be consumed twice daily for 28 days. Following a washout period (no drugs) of 7 days, patient will return to clinic for second study visit and receive second set of medication (placebo -vs- Ampicillin). Medication will be consumed twice daily for 28 days, with patient returning to clinic after a washout period of 7 days for final study visit in clinic.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Double (Participant, Investigator)
入排标准
- 年龄范围
- 7 Years 至 80 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •DYT-1 dystonia, confirmed by genetic testing
- •Between ages of 7 and 80 years
- •BFM-DRS score greater than 6
排除标准
- •Negative DYT-1 dystonia gene test
- •Allergy to penicillins or cephalosporins
- •Concurrent bacterial, viral or fungal infection at time of enrollment
- •Pregnancy
- •Inability to follow study protocol
- •Lactose intolerance (placebo contains lactose powder)
研究组 & 干预措施
Ampicillin
Patients who are randomly selected to receive the active drug will receive Ampicillin (2mg daily for adults and 1 mg daily for children).
干预措施: Ampicillin (Drug)
Placebo
Patients who are randomly selected to receive the placebo will be given a sugar pill that resembles the active drug.
干预措施: Sugar pill (Drug)
结局指标
主要结局
Safety and tolerability of Ampicillin in treating DYT-1 dystonia
时间窗: 70 days
The primary objective of this study is to evaluate the safety and tolerability of treating DYT-1 dystonia with the use of ampicillin as compared to placebo.
次要结局
- Burke-Fahn Marsden Dystonia Rating Scale (BFM-DRS)(70 days)