Testing tideglusib for amyotrophic lateral sclerosis, a progressive muscle-weakening disease
Tests whether tideglusib is safe and tolerable in adults with ALS.
Plain-language title generated from the registry record
Registry title: Tideglusib for the Treatment of Amyotrophic Lateral Sclerosis
Key facts
- Where
- Bern, Geneva, Lausanne, Sankt Gallen, and Zurich, SwitzerlandSee the 5 sites
- Phase
- Phase 2
Where in Switzerland
- University Hospital BernBern, SwitzerlandNo longer taking part
- University Hospital GenevaGeneva, SwitzerlandNo longer taking part
- University Hospital LausanneLausanne, SwitzerlandNo longer taking part
- Kantonsspital St. GallenSankt Gallen, SwitzerlandNo longer taking part
- University Hospital ZurichZurich, SwitzerlandNo longer taking part
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- University of Zurich
- Trial code
- NCT05105958
- Record updated
- 28 January 2025
- Original description
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.Read full original descriptionShow less
Tideglusib for the Treatment of Amyotrophic Lateral Sclerosis (TIDALS): a Randomized Placebo-controlled Phase II Trial
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.
Inclusion: 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: Previous participation in another clinical study within the preceding 12 weeks; Presence of any concomitant life-threatening disease or impairment likely to interfere with functional assessment; Any medical condition known to have an association with motor neuron dysfunction which might confound or obscure the diagnosis of ALS; Evidence of a major psychiatric disorder or clinically evident dementia precluding evaluation of symptoms; 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; Proven SOD1- or FUS - mutation; Tracheostomy or assisted ventilation of any type during the preceding three months; Pregnancy or breast-feeding females; Alcoholism; 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.