AUTHOR=Albanese Alberto , Ludolph Albert Christian , McDermott Christopher J. , Corcia Philippe , Van Damme Philip , Van den Berg Leonard H. , Hardiman Orla , Rinaldi Gilberto , Vanacore Nicola , Dickie Brian , TUDCA-ALS Study Group , Tornese Paolo , Cocco Antoniangela , Giudice Maria Lo , Matteoli Michela , Lauranzano Eliana , Malosio Maria Luisa , Adriana Elia Chiara , Lombardo Flavia , Mayer Flavia , Puopolo Maria , Alegiani Stefania Spila , Chiò Adriano , Manera Umberto , Moglia Cristina , Calvo Andrea , Salamone Paolina , Fuda Giuseppe , Colosimo Carlo , Spera Cristina , Ranchicchio Prabha Cristina , Stipa Giuseppe , Frondizi Domenico , Lunetta Christian , Sansone Valeria , Tarlarini Claudia , Gerardi Francesca , Silani Vincenzo , Doretti Alberto , Colombo Eleonora , Demirtzidis Gianluca , Tedeschi Gioacchino , Trojsi Francesca , Passaniti Carla , Ballestrero Stefania , Dorst Johannes , Weiland Ulrike , Fromm Andrea , Wiesenfarth Maximilian , Kandler Katharina , Witzel Simon , Otto Markus , Schuster Joachim , Meyer Thomas , Maier André , Kettemann Dagmar , Petri Susanne , Müschen Lars , Wohnrade Camilla , Sarikidi Anastasia , Osmanovic Alma , Grosskreutz Julian , Rödiger Annekathrin , Steinbach Robert , Ilse Benjamin , Smesny Uta , Untucht Robert , Günther René , Vidovic Maximilian , Shaw Pamela , Collins Alexis , Wollff Helen , Walsh Theresa , Tuddenham Lee , Kazoka Mbombe , White David , Young Stacy , Thompson Benjamin , Madarshahian Daniel , Chhetri Suresh K. , Chaouch Amina , Young Carolyn A. , Arndt Heike , Hanemann Oliver C , Lambert Thomas , Beltran Stephane , Couratier Philippe , Esselin Florence , Camu William , De Elisa , Cruz La , Lemasson Gwendal , Masrori Pegah , Maguire Sinead , Fogarty Liz , Atoyebi Toyosi , Obáin Niamh Ní TITLE=Tauroursodeoxycholic acid in patients with amyotrophic lateral sclerosis: The TUDCA-ALS trial protocol JOURNAL=Frontiers in Neurology VOLUME=13 YEAR=2022 URL=https://www.frontiersin.org/journals/neurology/articles/10.3389/fneur.2022.1009113 DOI=10.3389/fneur.2022.1009113 ISSN=1664-2295 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.