ORCID
- Oliver Hanemann: 0000-0002-1951-1025
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.
DOI
10.3389/fneur.2022.1009113
Publication Date
2022-09-27
Publication Title
Frontiers in Neurology
Volume
13
ISSN
1664-2295
Keywords
amyotrophic lateral sclerosis, bile acids, clinical trial, phase III, therapy
Recommended Citation
Albanese, A., TUDCA-ALS Study Group., Ludolph, A., McDermott, C., Corcia, P., Van Damme, P., Van den Berg, L., Hardiman, O., Rinaldi, G., Vanacore, N., Dickie, B., Tornese, P., Cocco, A., Giudice, M., Matteoli, M., Lauranzano, E., Malosio, M., Elia, C., Lombardo, F., Mayer, F., Puopolo, M., Alegiani, S., Chiò, A., Manera, U., Moglia, C., Calvo, A., Salamone, P., Fuda, G., Colosimo, C., Spera, C., Stipa, G., Frondizi, D., Lunetta, C., Sansone, V., Tarlarini, C., Gerardi, F., Silani, V., Doretti, A., Colombo, E., Demirtzidis, G., Tedeschi, G., Trojsi, F., Passaniti, C., Ballestrero, S., Dorst, J., Weiland, U., Fromm, A., Wiesenfarth, M., Kandler, K., Witzel, S., & Hanemann, O. (2022) 'Tauroursodeoxycholic acid in patients with amyotrophic lateral sclerosis: The TUDCA-ALS trial protocol', Frontiers in Neurology, 13. Available at: https://doi.org/10.3389/fneur.2022.1009113
