A Phase 1/2 Trial of Tauroursodeoxycholic Acid Supplementation in Progressive MS Patients
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 59
- 试验地点
- 1
- 主要终点
- Number of Participants With at Least One Treatment-related Adverse Event
研究概览
简要总结
This study aims to identify the safety and tolerability of bile acid supplementation in patients with progressive Multiple Sclerosis (MS). Participants will also be assessed for an impact of the bile acid on their immune system and gut microbiome. Half of the participants will receive the bile acid tauroursodeoxycholic acid (TUDCA) and half will receive placebo. The investigators believe participants who take TUDCA will have normalization of blood bile acid levels, a normalization of abnormal immune response and a normalization of the gut microbiome.
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, Care Provider)
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
- •Diagnosis of Progressive MS based on Lublin Criteria
- •Low bile acid levels identified using targeted metabolomics analysis
- •On the same therapy for the past 6 months and not expected to switch therapy in the next 6 months
- •No relapse in the past 3 months
排除标准
- •No previous history of liver disease or cholecystectomy
- •No stage IV/V chronic kidney disease or other severe metabolic derangements (e.g. poorly controlled thyroid disease or diabetes)
- •BMI < 15 kg/m2 and BMI > 40 kg/m2
- •Female patients who are pregnant or nursing, or not willing to use contraception
- •Chronic antibiotic use
- •Corticosteroid treatment within the past 30 days
- •Known history of other neuroinflammatory, neurodegenerative or systemic autoimmune disease
研究组 & 干预措施
TUDCA Treatment
Tauroursodeoxycholic acid (Taurolite) 250 mg four capsules by mouth, twice daily for 16 weeks.
干预措施: Tauroursodeoxycholic Acid (Drug)
Placebo oral capsule
Placebo oral capsule four capsules by mouth, twice daily for 16 weeks.
干预措施: Placebo oral capsule (Drug)
结局指标
主要结局
Number of Participants With at Least One Treatment-related Adverse Event
时间窗: 16 weeks
Safety and tolerability will be assessed based on treatment-related adverse events in the two arms.
Number of Total Treatment-related Adverse Events
时间窗: 16 weeks
Safety and tolerability will be assessed based on treatment-related adverse events in the two arms.
Incidence of Treatment-related Adverse Events (AE)
时间窗: 16 weeks
Safety and tolerability will be assessed based on treatment-related adverse events in the two arms. AE incidence will be measured as total number of events per 1000 exposure years.
次要结局
- Change in Quality of Life Based on Multiple Sclerosis Quality of Life (MSQOL)-54 Instrument(Baseline to 16 weeks)
- Change in Microbiome Alpha-diversity Measured by Shannon Index at the End of the Study(Baseline to 16 weeks)
- Change in Fasting Bile Acid Levels in Plasma(Baseline to 16 weeks)
- Change in Flow Cytometric Assessments of Peripheral Blood Mononuclear Cells (PBMCs)(Baseline to 16 weeks)