Tideglusib for the Treatment of Amyotrophic Lateral Sclerosis (TIDALS): a Randomized Placebo-controlled Phase II Trial
试验速览
- 阶段
- 2 期
- 状态
- 尚未招募
- 入组人数
- 98
- 试验地点
- 5
- 主要终点
- Increase in Alanine Aminotransferase
研究概览
简要总结
Amyotrophic lateral sclerosis (ALS) is a severe neurodegenerative condition, mainly characterized by progressive weakness and wasting of the limbs, the respiratory and bulbar muscles. Respiratory insufficiency leads to a fatal outcome after a mean diseases duration of only three to five years. The disease is characterized by pathological accumulations of a protein called TDP-43, which can be found large cortical and sub-cortical areas of post-mortem ALS brains.
No causal treatment for this condition is known to date, and there is a large unmet need to develop new strategies in order to halt or slow down its progression.
The aim of this study is to test the safety and tolerability of Tideglusib, a treatment that is already in clinical trials for other neuromuscular conditions, in patients with ALS. It is assumed that this drug may have a significant therapeutic benefit in this population due to his mode of action: In the ALS mouse model, Tideglusib decreases significantly the amount of accumulated TDP-43 proteins within the cells.
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)
盲法说明
Double-blind
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
- •Possible, probable (clinically or laboratory supported) or definite ALS according to the revised version of the El Escorial criteria
- •Disease duration < 18 months
- •Vital capacity of more than 60% of normal (defined as slow vital capacity, best of three measurements)
- •Age more than 18 years
- •On a stable dose of riluzole for at least four weeks or not taking riluzole
- •On a stable dose of edaravone for at least four weeks or not taking edaravone
- •Capable of thoroughly understanding all information given and giving full informed consent according to GCP
排除标准
- •Previous participation in another clinical study within the preceding 12 weeks
- •Proven SOD1- or FUS - mutation
- •Tracheostomy or assisted ventilation of any type during the preceding three months
- •Pregnancy or breast-feeding females
- •Any medical condition known to have an association with motor neuron dysfunction which might confound or obscure the diagnosis of ALS
- •Presence of any concomitant life-threatening disease or impairment likely to interfere with functional assessment
- •Evidence of a major psychiatric disorder or clinically evident dementia precluding evaluation of symptoms
- •Alcoholism
- •Cardiovascular disorder/arrhythmia
- •Impaired kidney function, defined as creatinine levels of 2.5 x upper limit of normal (ULN)
- •Impaired liver function, defined as aspartate aminotransferase (AST) or alanine aminotransferase (ALT) of 3 x ULN
- •Liable to be not cooperative or comply with trial requirements as assessed by the investigator, or unable to be reached in the case of emergency
研究组 & 干预措施
Tideglusib
Patients receive 1000 mg Tideglusib once daily per os
干预措施: Tideglusib (Drug)
Placebo
Patients receive placebo matching Tideglusib 100 mg once daily per os
干预措施: Tideglusib (Drug)
结局指标
主要结局
Increase in Alanine Aminotransferase
时间窗: 14 weeks
Increase in Alanine Aminotransferase \< 3x of Upper Limit of Normal
次要结局
- Most common side effect(14 weeks)