Tauroursodeoxycholic acid in patients with amyotrophic lateral sclerosis: the TUDCA-ALS trial protocol

Abstract

Background: Amyotrophic lateral sclerosis (ALS) is a chronic neurodegenerative rare disease that affects motor neurons in the brain, brainstem, and spinal cord, resulting in progressive weakness and atrophy of voluntary skeletal muscles. Although much has been achieved in understanding the disease pathogenesis, treatment options are limited, and in Europe, riluzole is the only approved drug. Recently, some other drugs showed minor effects.

Methods: The TUDCA-ALS trial is a phase III, multicenter, randomized, double-blind, placebo-controlled, parallel-group clinical trial. The study aims to enroll 320 patients in 25 centers across seven countries in Europe. Enrolled patients are randomized to one of two treatment arms: TUDCA or identical placebo by oral route. The study measures disease progression during the treatment period and compares it to natural progression during a no-treatment run-in phase. Clinical data and specific biomarkers are measured during the trial. The study is coordinated by a consortium composed of leading European ALS centers.

Conclusion: This trial is aimed to determine whether TUDCA has a disease-modifying activity in ALS. Demonstration of TUDCA efficacy, combined with the validation of new biomarkers, could advance ALS patient care.

Clinical trial registration: ClinicalTrials.gov, identifier: NCT03800524.

Metadata

Authors/Creators:

Albanese, A.
Ludolph, A.C.
McDermott, C.J. https://orcid.org/0000-0002-1269-9053
Corcia, P.
Van Damme, P.
Van den Berg, L.H.
Hardiman, O.
Rinaldi, G.
Vanacore, N.
Dickie, B.

© 2022 Albanese, Ludolph, McDermott, Corcia, Van Damme, Van den Berg, Hardiman, Rinaldi, Vanacore, Dickie and TUDCA-ALS Study Group. This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) and the copyright owner(s) are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms. (https://creativecommons.org/licenses/by/4.0/)

Keywords:

amyotrophic; lateral sclerosis; therapy; clinical trial; phase III; bile acids

Dates: