The Effect of Betaine in Refractory Syringomyelia（RS）
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 30
- 试验地点
- 1
- 主要终点
- ASIA Score
研究概览
简要总结
Purpose: This clinical trial aims to evaluate the indications, therapeutic effects and side effects of betaine in refractory syringomyelia.
Primary outcome measure: The primary endpoint is the change of ASIA at week 12. The clinical efficacy is defined as ASIA increase ≥ 1 at week 12, as compared with that before betaine usage.
详细描述
The pathogenesis of syringomyelia is poorly understood and duraplasty or shunting is not always effective. Although it is generally thought that syringomyelia is simply an accumulation of CSF from the subarachnoid space, the pathogenesis is likely to be more complex and may involve cellular and molecular processes.
The investigators supposed that betaine might play a key role in the pathogenesis of syringomyelia, especially post-traumatic syringomyelia(PTS), and that betaine, as an Osmotic homeostasis-related drug, would protect Osmotic homeostasis in syringomyelia.
Primary objectives: This clinical trial aims to evaluate the indications, therapeutic effects and safety of betaine in refractory syringomyelia.
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
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
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
- •Contraindication to duraplasty and shunting treatment due to history or high risk of severe adverse effects,
- •non-effective response to duraplasty and shunting treatment in 12 months prior to study entry.
- •Estimated life expectancy must be greater than 12 months.
- •Ability to understand and willingness to sign a written informed consent document, or constant caregivers who well understand and willingness to sign a written informed consent document.
- •Must be able to swallow tablets
排除标准
- •Participants have a cardiometabolic disease for which they take prescribed medications
- •Evidence of tumor metastasis, recurrence, or invasion;
- •History of psychiatric diseases ;
- •History of seizures;
- •History of arteriosclerotic cardiovascular diseases (ASCVD), e.g. stroke, myocardial infaction, unstable angina, within 6 months;
- •New York Heart Association Grade II or greater congestive heart failure;
- •Serious and inadequately controlled cardiac arrhythmia;
- •Significant vascular disease, e.g. moderate or severe carotid stenosis, aortic aneurysm, -history of aortic dissection;
- •Severe infection;
- •History of allergy to relevant drugs;
- •Pregnancy, lactation, or fertility program in the following 12 months;
- •History or current diagnosis of peripheral nerve disease;
- •Abnormal in liver and renal function;
- •Active tuberculosis;
- •Transplanted organs;
- •Human immunodeficiency virus;
- •Participation in other experimental studies.
研究组 & 干预措施
betaine
oral betaine
干预措施: Betaine (Drug)
placebo
rice-flour as a placebo
干预措施: Placebo (Drug)
结局指标
主要结局
ASIA Score
时间窗: 1 day before and 3 days, 3 months, after drug treatment
American Spinal Injury Association(ASIA) Score for evaluating the spinal cord function， degree of the spinal cord function, motor1-100, sensory 1-224, higher scores mean a better outcome
次要结局
- syringomyelia remission(1 day before and 3 days, 3 months, after drug treatment)
- Visual Analog Scale (VAS)(1 day before and 3 days, 3 months, after drug treatment)
- Klekamp and Sammi syringomyelia scale(1 day before and 3 days, 3 months, after drug treatment)
- xuanwu syringomyelia scale(3 days, 3 months, after drug treatment)
- Electrophysiology results(1 day before and 3 days, 3 months, after drug treatment)
- modified Japanese Orthopaedic Association Scores (mJOA)(1 day before and 3 days, 3 months, after drug treatment)
- Incidence of complications(3 days, 3 months, after drug treatment)