Open Label Extension of TUDCA-ALS Study

Study Overview
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Brief Summary
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This study will provide extended access to patients and assess longer-term outcomes on patients who have completed the TUDCA-ALS study.

Detailed Description
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The TUDCA-ALS Open label extension study is designed to investigate long term safety, tolerability and efficacy of tauroursodeoxycholic acid in patients with ALS who completed the TUDCA-ALS study

Official Title
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Open Label Extension Study to Investigate Long Term Safety, Tolerability and Efficacy of Tauroursodeoxycholic Acid in Patients With ALS Who Completed the TUDCA-ALS Study

Conditions
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Amyotrophic Lateral Sclerosis

Intervention / Treatment
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* Drug: Tauroursodeoxycholic Acid


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Completion of the visit M18 (Month 18) of the TUDCA-ALS clinical trial. Signed informed consent for participation in the TUDCA-ALS Extension sub-study Exclusion Criteria: Treatment with edaravone or other unaccepted concomitant therapy Consideration by the investigator, for any reason, that the subject is an unsuitable candidate to receive TUDCA or that the subject is unable or unlikely to comply with the dosing schedule or study evaluations The patient of reproductive potential is sexually active and is not willing to use highly effective contraception during the study and up to 90 days after the day of last dose The patient is pregnant or breast feeding

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Active treatment<br> | Drug: Tauroursodeoxycholic Acid<br>* Tauroursodeoxycholic acid (TUDCA) 250 mg capsules Doses: 4 capsules (1 g) twice daily 10-15 minutes after a meal Mode of administration: orally Duration: 18 months<br>* Other names: TUDCA,Tudcabil, Taurolite;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Long-term safety and tolerability of TUDCA during the open-label phase | Long-term safety and tolerability assessed through adverse reaction, concomitant treatment, and routine biochemistry analyses | 18 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Survival time | Survival time measured by death or respiratory insufficiency (defined as tracheostomy or the use of non-invasive ventilation for ≥22 h per day for ≥10 consecutive days). | 18 months | 
| Change in disease progression and functional impairment | Change in disease progression and functional impairment as measured by ALSFRS-R. | 18 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Bile acids, Deoxycholic Acid