Double-blind, Randomized, Placebo-controlled Two-period Crossover Study to Assess the Effect of Levocarnitine on Vismodegib-associated Muscle Spasms
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 12
- 试验地点
- 2
- 主要终点
- Percentage difference in muscle spasm frequency (defined as number per week) between levocarnitine and placebo
研究概览
简要总结
This randomized clinical trial studies levocarnitine in treating patients with vismodegib-associated muscle spasms. Levocarnitine may decrease muscle spasms caused by vismodegib.
详细描述
PRIMARY OBJECTIVES:
I. To determine the effect of levocarnitine on muscle spasm frequency compared to placebo (median percentage comparison)
SECONDARY OBJECTIVES:
I. To assess intensity of muscle spasms after levocarnitine compared to placebo.
II. To assess responses related to activities of daily living or psychosocial function after levocarnitine compared to placebo.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Crossover
- 主要目的
- Supportive Care
- 盲法
- Double (Participant, Investigator)
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
- •Taking vismodegib daily
- •Subject answers item #1 of muscle spasms questionnaire as moderate or severe intensity at time of screening
- •At least one muscle spasm per day at time of screening
- •Muscle spasms onset after starting vismodegib
- •Willing and able to understand and sign consent form
排除标准
- •Presence of muscle spasms or active neurologic disease prior to start of vismodegib
- •Use of thyroid medication at the time of screening
- •Use of Coumadin or acenocoumarol at time of screening
- •Change in regimen of muscle relaxant medications within four weeks of enrollment
- •If on stable muscle relaxant medication regimen for 4 weeks prior to enrolling, not willing to maintain muscle relaxant regimen without change during course of the study
- •Presence of significant renal disease or hemodialysis which would result in dramatic reductions of systemic levocarnitine levels
- •History of seizures
- •Known deficiency in carnitine (genetic, etc.)
- •Any uncontrolled medical condition which may place the patient at increased risk during study participation (at the discretion of the clinical investigator)
- •Unable or unwilling to comply with study procedures
- •Pregnant or lactating
- •All female patients of childbearing potential including those who are within 1 year of last menstrual period will be required to take a pregnancy test during screening, enrollment and at week 0, 4, 8 and 12
- •If female of reproductive age, or male partner of female of reproductive age, unwilling to use two medically reliable forms of birth control while on vismodegib
- •Unwilling to refrain from donation of bodily fluid (blood, platelets, etc.) within 7 months of last vismodegib dose
研究组 & 干预措施
Arm I (levocarnitine start)
Patients receive levocarnitine PO BID during weeks 1-4. Washout is weeks 5-8. Patients then cross-over to placebo for weeks 9-12.
干预措施: levocarnitine (Dietary Supplement)
Arm I (levocarnitine start)
Patients receive levocarnitine PO BID during weeks 1-4. Washout is weeks 5-8. Patients then cross-over to placebo for weeks 9-12.
干预措施: questionnaire administration (Other)
Arm II (placebo start)
Patients receive placebo PO BID during weeks 1-4. Washout is weeks 5-8. Patients then cross-over to levocarnitine for weeks 9-12.
干预措施: placebo (Other)
Arm II (placebo start)
Patients receive placebo PO BID during weeks 1-4. Washout is weeks 5-8. Patients then cross-over to levocarnitine for weeks 9-12.
干预措施: questionnaire administration (Other)
结局指标
主要结局
Percentage difference in muscle spasm frequency (defined as number per week) between levocarnitine and placebo
时间窗: 12 weeks
Wilcoxon rank-sum test, paired
次要结局
- Severity of adverse events after levocarnitine and placebo(12 weeks)
- Change in severity of muscle spasms after levocarnitine and placebo(12 weeks)
- Social/emotional impacts of muscle spasms after levocarnitine and placebo(12 weeks)
- Change in number of body locations affected by muscle spasms after levocarnitine and placebo(12 weeks)
- Change in impact of spasms on activities of daily living (such as sleeping or driving) after levocarnitine and placebo(12 weeks)
- Frequency of adverse events after levocarnitine and placebo(12 weeks)
研究者
Anne Chang
Associate Professor of Dermatology
Stanford University