NCT03800524

Safety and Efficacy of TUDCA as add-on Treatment in Patients Affected by ALS

Safety and Efficacy of Tauroursodeoxycholic (TUDCA) as add-on Treatment in Patients Affected by Amyotrophic Lateral Sclerosis (ALS)

• Status: Unknown
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 337 (actual)
• Sponsor: Humanitas Mirasole SpA · Academic / Other
• Sex: All
• Age: 18 Years – 80 Years
• Healthy volunteers: Not accepted

Summary

This is a Phase III, multi-centre, randomized, double-blind, placebo-controlled, parallel-group study to evaluate Safety and Efficacy of Tauroursodeoxycholic (TUDCA) as add-on Treatment in Patients Affected by Amyotrophic Lateral Sclerosis (ALS).

Detailed description

Enrolled patients will be randomized to one of two treatment arms: TUDCA or identical placebo by oral route. The randomization will be performed in a ratio one to one for the two arms. TUDCA will be administered orally at the dose of 1 g twice daily (2 g daily) for 18 months. Patients will be taking also riluzole at the dose of 50 mg twice daily (100 mg daily). Patient randomization will take place after a screening (lead-in) period of 12 weeks (3 months) with 3 assessments at 6-week intervals. Clinical assessments during the trial phase will be performed every three months. This will allow measuring the progression rate before and after starting treatment (either active or placebo).

Conditions

• Amyotrophic Lateral Sclerosis (ALS)

Interventions

Type	Name	Description
DRUG	Tauroursodeoxycholic Acid	* 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
DRUG	Placebo	* Placebo 250 mg capsules * Doses: 4 capsules (1 g) twice daily 10-15 minutes after a meal * Mode of administration: orally * Duration: 18 months

Timeline

• Start date: 2019-02-22
• Primary completion: 2023-12-31
• Completion: 2023-12-31
• First posted: 2019-01-11
• Last updated: 2023-07-10

Locations

25 sites across 7 countries: Belgium, France, Germany, Ireland, Italy, Netherlands, United Kingdom