Tideglusib for the Treatment of Amyotrophic Lateral Sclerosis (TIDALS): a Randomized Placebo-controlled Phase II Trial

TIDALS_01

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.

2025-12-01

2025-12-01

2026-03-01

Not yet recruiting

Early phase I

98

Inclusion Criteria: * 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 Exclusion Criteria: * 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

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2023-06-02