Tideglusib for the Treatment of Amyotrophic Lateral Sclerosis (TIDALS)
Primary Purpose
Amyotrophic Lateral Sclerosis
Status
Not yet recruiting
Phase
Phase 2
Locations
Switzerland
Study Type
Interventional
Intervention
Tideglusib
Sponsored by
About this trial
This is an interventional treatment trial for Amyotrophic Lateral Sclerosis
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
Sites / Locations
- University Hospital Bern
- University Hospital Geneva
- University Hospital Lausanne
- Kantonsspital St. Gallen
- University Hospital Zurich
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Placebo Comparator
Arm Label
Tideglusib
Placebo
Arm Description
Patients receive 1000 mg Tideglusib once daily per os
Patients receive placebo matching Tideglusib 100 mg once daily per os
Outcomes
Primary Outcome Measures
Increase in Alanine Aminotransferase
Increase in Alanine Aminotransferase < 3x of Upper Limit of Normal
Secondary Outcome Measures
Most common side effect
Occurence of diarrhea in less then 18 % of patients
Full Information
NCT ID
NCT05105958
First Posted
October 4, 2021
Last Updated
June 1, 2023
Sponsor
University Hospital, Geneva
Collaborators
University of Lausanne Hospitals, University of Zurich, University of Bern, Cantonal Hospital of St. Gallen
1. Study Identification
Unique Protocol Identification Number
NCT05105958
Brief Title
Tideglusib for the Treatment of Amyotrophic Lateral Sclerosis
Acronym
TIDALS
Official Title
Tideglusib for the Treatment of Amyotrophic Lateral Sclerosis (TIDALS): a Randomized Placebo-controlled Phase II Trial
Study Type
Interventional
2. Study Status
Record Verification Date
June 2023
Overall Recruitment Status
Not yet recruiting
Study Start Date
December 1, 2025 (Anticipated)
Primary Completion Date
December 1, 2025 (Anticipated)
Study Completion Date
March 1, 2026 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
University Hospital, Geneva
Collaborators
University of Lausanne Hospitals, University of Zurich, University of Bern, Cantonal Hospital of St. Gallen
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Amyotrophic Lateral Sclerosis
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Masking Description
Double-blind
Allocation
Randomized
Enrollment
98 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Tideglusib
Arm Type
Experimental
Arm Description
Patients receive 1000 mg Tideglusib once daily per os
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
Patients receive placebo matching Tideglusib 100 mg once daily per os
Intervention Type
Drug
Intervention Name(s)
Tideglusib
Intervention Description
1000 mg/day per os
Primary Outcome Measure Information:
Title
Increase in Alanine Aminotransferase
Description
Increase in Alanine Aminotransferase < 3x of Upper Limit of Normal
Time Frame
14 weeks
Secondary Outcome Measure Information:
Title
Most common side effect
Description
Occurence of diarrhea in less then 18 % of patients
Time Frame
14 weeks
Other Pre-specified Outcome Measures:
Title
Exploratory outcome: clinical efficacy
Description
Difference of decline in points on the Revised ALS Functional Rating Scale between the two study arms
Time Frame
14 weeks
Title
Exploratory outcome: vital capacity
Description
slow vital capacity in %
Time Frame
14 weeks
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
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
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Annemarie Hübers
Phone
0795531171
Email
annemarie.hubers@hcuge.ch
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Annemarie Hübers
Organizational Affiliation
University Hospital, Geneva
Official's Role
Principal Investigator
Facility Information:
Facility Name
University Hospital Bern
City
Bern
Country
Switzerland
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Olivier Scheidegger
Facility Name
University Hospital Geneva
City
Genève
ZIP/Postal Code
1205
Country
Switzerland
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Annemarie Hübers
Phone
0795531171
Email
annemarie.hubers@hcuge.ch
Facility Name
University Hospital Lausanne
City
Lausanne
Country
Switzerland
Facility Contact:
First Name & Middle Initial & Last Name & Degree
David Benninger
Facility Name
Kantonsspital St. Gallen
City
Saint-Gall
Country
Switzerland
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Markus Weber
Facility Name
University Hospital Zurich
City
Zürich
Country
Switzerland
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Hans Jung
12. IPD Sharing Statement
Plan to Share IPD
No
Learn more about this trial
Tideglusib for the Treatment of Amyotrophic Lateral Sclerosis
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