TUDCA study (“Safety and efficacy of tauroursodeoxycholic acid (TUDCA) as add-on treatment in patients affected by amyotrophic lateral sclerosis (ALS)”), supported by a Horizon 2020 project.

This phase 3 study aims to investigate the effect of taurodeoxycholic acid (TUDCA) on disease progression in patients with ALS. TUDCA is a bile acid with neuroprotective effects on motor nerve cells. After ingestion, TUDCA is absorbed into the bloodstream, but can also reach the brain because it can cross the blood-brain barrier. TUDCA has few side effects. In a small phase 2 study, there seemed to be an effect of this drug on functional decline (measured via the ALSFRS-R) of ALS patients. It is therefore hoped that a beneficial effect on disease progression could be demonstrated in this study.

The study will compare a dose of 2 x 1000 mg daily with placebo, with a ratio of 1:1. Patients will be followed up for 18 months. To participate, the disease should not be too advanced: patients should be ill for up to 18 months, swallowing function should be normal and lung function (FVC) should be ≥70%. The study is taking place in more than 20 centres in Europe. About 440 patients will be asked to participate. In Belgium, UZ Leuven is participating in this study.

Detailed information and the list of inclusion and exclusion criteria can be found at:
clinicaltrials.gov/ct2/show/NCT03800524

and more information about the project at:
www.tudca.eu/