Randomized Controlled Trial of N-acetyl Cysteine as a Neuroprotective Agent in Progressive Multiple Sclerosis
试验速览
- 阶段
- 2 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 98
- 试验地点
- 1
- 主要终点
- Safety and tolerability
研究概览
简要总结
This study evaluates the effectiveness of N-acetyl cysteine (NAC) in the treatment of progressive multiple sclerosis. Half of the patients will receive NAC, while the other half will receive a placebo.
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
- 40 Years 至 70 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •- 40-70 (inclusive) years in age,
- •meet 2017 McDonald criteria (Thompson 2018),
- •patients with primary or secondary progressive MS (Thompson 2018),
- •at least 2 years since progressive symptom onset,
- •evidence of clinical changes over the previous 2 years unrelated to relapses: increased EDSS or 20% slowing on 25-foot walk, change of ambulatory support, cognitive change documented on cognitive testing. Progression defined by patients in terms of ambulation perimeter or type of support to ambulate are acceptable if aforementioned physician-based measure changes are not available.
- •EDSS score 3.0 to 7.0 (inclusive),
- •can be on a stable disease-modifying treatment initiated > 3 months prior to screening,
- •can be on stable doses of dalfampridine initiated at least one month before screening.
排除标准
- •- MS relapses in the previous 6 months
- •oral glucocorticosteroid treatment within the prior 3 months
- •patient with issues undergoing MRI scans
- •pregnancy or breastfeeding
- •women of child-bearing potential not able to utilize an effective form of contraception for the duration of the study
- •history of bleeding disorders
- •active gastrointestinal ulcers
- •abnormal liver function testing (aminotransferase (AST) or alanine aminotransferase (ALT) >2 times upper limit of normal)
- •current treatment for active malignancy or metastatic malignancy treated in the past year
- •alcohol or substance use disorder
- •allergy to NAC
- •planned surgery or move within 15 months
- •use of medications/supplements with antioxidant properties (including over-the-counter NAC)
研究组 & 干预措施
N-acetyl cysteine
N-acetyl cysteine (NAC) 1200mg t.i.d.
干预措施: N-acetyl cysteine (Drug)
Placebo
Placebo 1200mg t.i.d.
干预措施: Placebo (Drug)
结局指标
主要结局
Safety and tolerability
时间窗: 15 months
Number of adverse events recorded by system, severity, and by relationship to treatment arm.
Effect of NAC on on progression of brain, thalamic and cervical cord atrophy
时间窗: 15 months
The primary endpoint is brain, thalamic and cord atrophy measured by brain and cervical spine MRI at month 3 and month 15.
次要结局
- Clinical effects of NAC(15 months)
研究者
Emmanuelle Waubant, MD PhD
Professor, Neurology UCSF Weill Institute for Neurosciences
University of California, San Francisco