Tauroursodeoxycholic acid in patients with amyotrophic lateral sclerosis: The TUDCA-ALS trial protocol
TUDCA-ALS Study Group
(2022) Frontiers in Neurology, volume 13
(Article)
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
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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.
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Keywords: amyotrophic lateral sclerosis, bile acids, clinical trial, phase III, therapy, Neurology, Clinical Neurology
ISSN: 1664-2295
Publisher: Frontiers Media S. A.
Note: Funding Information: This is an investigator-initiated study funded by the European Union's Horizon 2020 research and innovation program (grant agreement 755094). Funding Information: Author AL declares participation in Advisory Boards of Roche Pharma AG, Biogen, Alector, and Amylyx. Author CJM reports consultancy with Biogen, Amylyx, and Cytokinetics. Author PV declares participation in Advisory Board meetings for Biogen, UCB, argenx, Cytokinetics, Ferrer, Muna Therapeutics, Augustine Therapeutics, Alector, Alexion, QurAlis, and VectorY (paid to institution) and grant from CSL Behring (E. von Behring Chair for Neuromuscular and Neurodegenerative Disorders; paid to institution). Author OH declares participation in Advisory Boards for Accelsiors, Biogen Idec, Cytokinetics, NeuroSense Therapeutics, Neuropath Therapeutics, Novartis, Orion, Denali, and Wave Pharmaceuticals; role as Principal Investigator on the PRECISION ALS Project; Academic/Industry Collaboration funded by Science Foundation Ireland; Research collaboration with Biogen, Cytokinetics, and Ionis in delivering the IMPACT ALS survey and with Cytokinetics in delivering the REVEALS study of respiratory decline in ALS; Editor-in-Chief of ALS and the Frontotemporal Degeneration; Editorial board member of the Journal of Neurology, Neurosurgery, and Psychiatry. Author GR declares that Bruschettini S.R.L is the pharmaceutical company providing the investigational medicinal product and industrial partner of the project, in which he is an employee as Scientific Director. The remaining authors declare that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest. Publisher Copyright: Copyright © 2022 Albanese, Ludolph, McDermott, Corcia, Van Damme, Van den Berg, Hardiman, Rinaldi, Vanacore, Dickie and TUDCA-ALS Study Group.
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