Tideglusib for the Treatment of Amyotrophic Lateral Sclerosis
- Registration Number
- NCT05105958
- Lead Sponsor
- University Hospital, Geneva
- Brief Summary
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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- NOT_YET_RECRUITING
- Sex
- All
- Target Recruitment
- 98
- 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
- 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
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Tideglusib Tideglusib Patients receive 1000 mg Tideglusib once daily per os Placebo Tideglusib Patients receive placebo matching Tideglusib 100 mg once daily per os
- Primary Outcome Measures
Name Time Method Increase in Alanine Aminotransferase 14 weeks Increase in Alanine Aminotransferase \< 3x of Upper Limit of Normal
- Secondary Outcome Measures
Name Time Method Most common side effect 14 weeks Occurence of diarrhea in less then 18 % of patients
Trial Locations
- Locations (5)
University Hospital Lausanne
🇨🇭Lausanne, Switzerland
University Hospital Zurich
🇨🇭Zürich, Switzerland
University Hospital Bern
🇨🇭Bern, Switzerland
Kantonsspital St. Gallen
🇨🇭Saint-Gall, Switzerland
University Hospital Geneva
🇨🇭Genève, Switzerland