Tideglusib for the Treatment of Amyotrophic Lateral Sclerosis

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.

NCT Number	NCT05105958
Lead Sponsor	University of Zurich Collaborators: University of Lausanne Hospitals, University of Bern, Cantonal Hospital of St. Gallen, University Hospital, Geneva
Conditions	Amyotrophic Lateral Sclerosis
Enrollment	98 participants
Start Date	2025-12-01
Primary Completion	2025-12-01 (estimated)
Study Completion	2026-03-01 (estimated)
Updated on ClinicalTrials.gov	2025-01-28