Tideglusib for the Treatment of Amyotrophic Lateral Sclerosis

NCT05105958 | Not Yet Recruiting Phase 2 DMC

• 1 other identifier: TIDALS_01
• Study Type: interventional
• Target: 98
• Locations: 1 country
• Sites: 5

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

Outcome Measures

Primary Outcomes (1)

• Increase in Alanine Aminotransferase

  Increase in Alanine Aminotransferase \< 3x of Upper Limit of Normal

  14 weeks

Secondary Outcomes (1)

• Most common side effect

  14 weeks

Other Outcomes (2)

• Exploratory outcome: clinical efficacy

  14 weeks

• Exploratory outcome: vital capacity

  14 weeks

Study Arms (2)

Tideglusib

EXPERIMENTAL

Patients receive 1000 mg Tideglusib once daily per os

Drug: Tideglusib

Placebo

PLACEBO COMPARATOR

Patients receive placebo matching Tideglusib 100 mg once daily per os

Drug: Tideglusib

Interventions

Tideglusib DRUG

1000 mg/day per os

Placebo; Tideglusib

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• University of Zurich: lead
• University of Lausanne Hospitals: collaborator
• University of Bern: collaborator
• Cantonal Hospital of St. Gallen: collaborator
• University Hospital, Geneva: collaborator

Study Sites (5)

University Hospital Bern

Bern, Switzerland

Location

University Hospital Geneva

Geneva, 1205, Switzerland

Location

University Hospital Lausanne

Lausanne, Switzerland

Location

Kantonsspital St. Gallen

Sankt Gallen, Switzerland

Location

University Hospital Zurich

Zurich, Switzerland

Location

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Interventions

tideglusib

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Annemarie Hübers

  University Hospital, Geneva

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Annemarie Hübers

CONTACT

0795531171; annemarie.hubers@hcuge.ch

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: Double-blind
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 4, 2021
• First Posted: November 3, 2021
• Study Start: December 1, 2025
• Primary Completion: December 1, 2025
• Study Completion: March 1, 2026
• Last Updated: January 28, 2025

Record last verified: 2025-01

Data Sharing

• IPD Sharing: Will not share

Locations

CH (5)