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

Trial ID: NCT05105958

Interventional - Phase 2 - Not yet recruiting

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.

Conditions

• Amyotrophic Lateral Sclerosis

Interventions

• Tideglusib

Eligibility

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

Sponsor

University of Zurich, University of Lausanne Hospitals, University of Bern, Cantonal Hospital of St. Gallen, University Hospital, Geneva

Source: ClinicalTrials.gov

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