Mitochondrial Capacity Boost in ALS (MICABO-ALS) Trial
试验速览
- 阶段
- 2 期
- 状态
- 进行中（未招募）
- 入组人数
- 21
- 试验地点
- 3
- 主要终点
- Measurement of serum NfL
研究概览
简要总结
The purpose of this research is to investigate the validity of a previous clinical trial named EH301, which showed beneficial effects of anti-oxidant therapies in patients with amyotrophic lateral sclerosis (ALS). If validated by this study, providing over-the-counter anti-oxidants would be a simple, low risk, low-cost approach to significantly slow or stop the progression of ALS, for which currently no effective treatment exists. It is currently thought that oxidative stress is a major cause of ALS. The study investigators are therefore planning to expand the original scope of the previous trial by including anti-oxidants at high doses that were not previously used. All of these compounds are considered safe.
详细描述
Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disorder that affects muscle function throughout the body. Weakness and muscle shrinkage begin either in the face, arm, or leg. A clinical ALS hallmark is rapidly progressive weight loss. Also, respiration is usually affected late in the disease, and death typically occurs as a consequence of respiratory failure. The median survival after disease onset is approximately 3-4 years.
While there are now two FDA-approved agents for patients with ALS, there are no interventions that have had a meaningful impact on the natural course of this disease. Riluzole prolongs survival by up to 12 weeks. Edaravone improves some aspects of neurological function in a small subset of patients, but that was ineffective in clinical studies that included ALS patients at all stages of disease.
Past failures to identify effective therapies reflect the complexity of ALS pathogenesis, in that no single therapeutic target has been identified. Thus, single agent or dual combination therapies are unlikely to succeed. Given the truly rapid and devastating nature of ALS and that there are no effective treatments for ALS, one can argue that it is critical to devise a different approach. Until the exact mechanisms that lead to ALS are identified, it is necessary to employ polytherapy which includes new agents that show promise.
This study will lay further groundwork on methodology for performing more definite trials in ALS. The study of secondary biomarkers in ALS is significant because there are currently no molecular or biochemical biomarkers for assessing therapeutic efficacy of drug treatments in ALS clinical trials. By doing this study, the study investigators hope to learn that high-dose anti-oxidants would be a simple, low risk, low-cost approach to significantly slow or stop the progression of ALS, for which currently no effective treatment exists. Participation in this research will last about 13 months
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 21 Years 至 80 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •A clinical diagnosis by a study investigator of laboratory-supported probable, probable, or definite ALS, according to a modified El Escorial criterion (Appendix 2).
- •21 to 80 years of age inclusive.
- •If patients are taking riluzole for ALS, they must be on a stable dose for at least thirty days prior to the baseline visit.
- •Willing and able to give signed informed consent that has been approved by the Institutional Review Board (IRB).
排除标准
- •Diagnosis of other neurodegenerative diseases (Parkinson disease, Alzheimer disease, etc.).
- •Clinically significant history of unstable medical illness (unstable angina, advanced cancer, etc.) over the last 30 days.
- •Infection with the human immunodeficiency virus (HIV)
- •Limited mental capacity such that the patient cannot provide written informed consent or comply with evaluation procedures.
- •History of recent alcohol or drug abuse or noncompliance with treatment or other experimental protocols. 6 Receipt of any investigational drug within the past 30 days.
研究组 & 干预措施
Antioxidants
Eligible patients will receive over-the-counter anti-oxidants, namely vitamin E, NAc cysteine, L-cystine, Nicotinamide and Taurursodiol at defined doses.
干预措施: Antioxidants (Combination Product)
结局指标
主要结局
Measurement of serum NfL
时间窗: 12 months
The change in log serum NfL level from baseline to 12 months will be assessed using a linear mixed model and the differences in NfL level at 12 months from baseline assessed using a paired t-test.
次要结局
- Survival analysis(12 months)
- Frequency of serious adverse events and adverse events.(12 months)
- Measurement of functional decline in ALS(12 months)