Double-Blind, Placebo Controlled Pilot-Study of Octanoic Acid in Essential Tremor
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 29
- 试验地点
- 1
- 主要终点
- Normalized Accelerometric Tremor Power, Dominant Hand, 80min After Administration, Weighted Condition
研究概览
简要总结
Background:
- Essential tremor (ET) is a neurological disorder characterized by uncontrollable shaking. Several medications are used to treat ET; however, they are often only partly effective and can have side effects.
- Research studies have shown that octanol, a food additive similar to alcohol, can improve tremor in animals. Octanol is less likely to make people drunk than alcohol. Two earlier NIH studies found that one form of octanol, called 1-octanol, did improve tremor in some people and had few side effects.
- In the body, 1-octanol is converted to octanoic acid. Researchers are interested in finding out whether octanoic acid can help people with ET.
Objectives:
- To find out if octanoic acid can improve hand tremor in people with essential tremor.
- To measure levels of octanoic acid in the blood after it is taken.
Eligibility:
- Patients 21 years of age and older with ET, who are willing to abstain from alcohol, caffeine, and all medications as required by the study and who are willing and able to fast for up to 12 hours at a time.
- Participants may not be of Asian or Native American ancestry because of genetic susceptibilities to the intoxicating effects of the study drug.
Design:
- This study requires a 3-day hospital admission as well as two outpatient visits.
- Visit 1 (outpatient): Screening visit and blood alcohol level test
- Medical history, physical and neurological examination, a blood test, and an electrocardiogram to measure heart function. Women who are able to get pregnant will have a urine pregnancy test.
- Patients will consume 1.5 ounces of alcohol per drink (up to three drinks at least 30 minutes apart), and be tested to evaluate how the tremor responds. Researchers will draw blood to measure blood alcohol level about 1 hour after the first drink and closely monitor patients for signs of intoxication.
- Inpatient examination
- Preparation: Researchers will prepare a schedule to stop any tremor medications that patients might be on. Patients may not drink alcohol or eat or drink anything with caffeine, including chocolate, for at least 2 days before admission.
- Day 1: Vital signs, blood (and urine pregnancy) tests, and electrocardiogram. Patients will be asked to wear a tremor monitor, similar to a wristwatch. Patients will also have IV lines inserted for blood draws.
- Days 2 and 3: Randomized study medication (octanoic acid on one day, placebo on the other day). Patients will fast before taking the drug, but will be allowed to eat and drink after the tests are completed (around noon).
- Blood will be drawn before taking the study drug and again (a total of nine times) after taking the drug.
- Tremor will be measured during the study, before and after taking the drug.
- Visit 2 (outpatient): 4 to 7 days after discharge
- Blood test and an electrocardiogram, and a series of questionnaires regarding the study.
详细描述
OBJECTIVE:
We propose a study to examine the safety and efficacy of octanoic acid in essential tremor (ET).
STUDY POPULATION:
We will study 19 adult subjects with ethanol-responsive ET.
DESIGN:
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
- 21 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- 未提供
研究组 & 干预措施
Octanoic Acid
干预措施: Octanoic Acid (Drug)
Placebo
干预措施: Placebo (Drug)
结局指标
主要结局
Normalized Accelerometric Tremor Power, Dominant Hand, 80min After Administration, Weighted Condition
时间窗: 80 min after administration of the study drug on day 1 and 2 of Visit 2
Postural tremor was measured using accelerometry with a motion sensor (accelerometer) placed at the dorsum of each hand, and tremor recorded simultaneously with surface-electromyography of wrist flexors and extensors for 2 minutes at each time-point. The recording was repeated with 1 lbs weight added to each wrist, which was described to record the central tremor component. The primary outcome measure was defined as tremor power of the central tremor component (after the addition of weight) 80 minutes after administration, measured at the dominant hand, normalized to baseline (baseline = 1), and comparing octanoic acid vs. placebo. Ratio of tremor power at 80 min divided by tremor power at baseline used for outcome measure calculation.
次要结局
- TMax Octanoic Acid(between 5 and 300 min post dose)
- Normalized Tremor Power, 300 Min After Administration, Weighted Condition, Dominant Hand, OA vs Placebo(300 min post dose)
- PK: AUC After OA(5 to 300 min post dose)
研究者
Mark Hallett
Chief, Human Motor Control Section
National Institute of Neurological Disorders and Stroke (NINDS)