Tideglusib for the Treatment of Amyotrophic Lateral Sclerosis

NCT ID: NCT05105958

Last Updated: 2025-01-28

Basic Information

• Recruitment Status: NOT_YET_RECRUITING
• Clinical Phase: PHASE2
• Total Enrollment: 98 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2025-12-01
• Study Completion Date: 2026-03-01

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.

Conditions

Amyotrophic Lateral Sclerosis

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

Tideglusib

Patients receive 1000 mg Tideglusib once daily per os

Group Type: EXPERIMENTAL

Tideglusib

Intervention Type: DRUG

1000 mg/day per os

Placebo

Patients receive placebo matching Tideglusib 100 mg once daily per os

Group Type: PLACEBO_COMPARATOR

Tideglusib

Intervention Type: DRUG

1000 mg/day per os

Interventions

Tideglusib

1000 mg/day per os

Intervention Type: DRUG

Eligibility Criteria

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

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

University of Lausanne Hospitals

OTHER

Sponsor Role: collaborator

University of Bern

OTHER

Sponsor Role: collaborator

Cantonal Hospital of St. Gallen

OTHER

Sponsor Role: collaborator

University Hospital, Geneva

OTHER

Sponsor Role: collaborator

University of Zurich

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Annemarie Hübers

Role: PRINCIPAL_INVESTIGATOR

University Hospital, Geneva

Locations

University Hospital Bern

Bern, , Switzerland

University Hospital Geneva

Geneva, , Switzerland

University Hospital Lausanne

Lausanne, , Switzerland

Kantonsspital St. Gallen

Sankt Gallen, , Switzerland

University Hospital Zurich

Zurich, , Switzerland

Countries

Switzerland

Central Contacts

Annemarie Hübers

Role: CONTACT

annemarie.hubers@hcuge.ch

0795531171

Facility Contacts

Olivier Scheidegger

Role: primary

Annemarie Hübers

Role: primary

annemarie.hubers@hcuge.ch

0795531171

David Benninger

Role: primary

Markus Weber

Role: primary

Hans Jung

Role: primary

Other Identifiers

TIDALS_01

Identifier Type: -

Identifier Source: org_study_id