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International Alliance of ALS/MND Associations

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TUDCA Trial

Background

Tauroursodeoxycholic acid (TUDCA) is an acid present in the bile regularly produced in the human liver. TUDCA is commonly used for treatment of chronic liver conditions and for gallstone and it is thought to have a beneficial effect on mitochondria health (ref: Hoffman 1999). TUDCA has also been described as a protective agent for cellular cell death, and therefore, considered for the treatment of neurodegenerative diseases (ref: Amaral et al., 2009).

Trial Design & Results

A phase 2b proof of concept double blind placebo-controlled study was run in Italy from 2008 to 2012 on 34 individuals affected by ALS/MND. The trial enrolled earlystage non severely disabled individuals who were treated with 1g twice daily of TUDCA or placebo for 54 weeks and examined every 6 weeks. The primary outcome measure was the portion of responder individuals, defined as those showing an improvement of at least 15% in the ALS functional rating scale revised (ALSFRS-R) slope during treatment period compared to the lead in period (the stage before the start of the trial). The study reported that TUDCA was well tolerated, and that the portion of responders was higher in the TUDCA treated group compared to placebo, resulting in slower progression in the TUDCA treated group (ref: Elia et al., 2015). However, this study was conducted on a relatively small number of individuals and a phase 3 was necessary to confirm TUDCA efficacy in slowing disease progression.

On the basis of the results of the phase 2b study, a larger multi center phase 3 placebo-controlled trial was designed using the same dosage of TUDCA and placebo. The trial aimed to recruit 440 people newly diagnosed with ALS in 26 centers across Italy, Germany, UK, France, the Netherlands and Ireland. The delivery of the trial was impacted by the COVID-19 pandemic and recruitment was less than planned. The results from this trial were released in March 2024, the study ran for 18 months including a total of 336 people but failed to meet its primary endpoint, which was defined as a difference in responding and nonresponding individuals in month 18 as measured by ALSFRS-R. Additionally, no difference was found between TUDCA and placebo in secondary outcome measures including time of survival and biomarker measurements. Treatment with TUDCA was well tolerated and generally safe (ref: TUDCA-ALS).

TUDCA is also one of the two components of AMX0035, a compound developed by Amylyx Pharmaceuticals that was recently announced to have not met either primary or secondary endpoints in a phase 3 clinical trial (ref: Amylyx press release).

Summary

Considering the available evidence, it is the opinion of the SAC that TUDCA is safe and tolerable. Given the available data, however, it is the opinion of the SAC that there is enough evidence to conclude that at the studied dosage, TUDCA did not show overall benefit in individuals living with ALS.

International Alliance of ALS/MND Associations
September 2024


The original language of communication is English and any translation cannot be guaranteed for accuracy of messaging.

SOURCES

  • Hoffman 1999 – https://jamanetwork.com/journals/jamainternalmedicine/fullarticle/1105662
  • Amaral et al., 2009 – https://www.sciencedirect.com/science/article/pii/S0022227520307252?pes=vor
  • Elia et al., 2015 – https://onlinelibrary.wiley.com/doi/10.1111/ene.12664
  • TUDCA-ALS – https://www.tudca.eu/top-line-results-announcement/
  • Amylyx press release – https://www.amylyx.com/news/amylyx-pharmaceuticalsannounces-topline-results-from-global-phase-3-phoenix-trial-of-amx0035-in-als

Primary Sidebar

  • Marco Antonio Alvarez Mercado, Mexico

    Marco Antonio Alvarez Mercado, Mexico

  • Den Haag, Diagnosed 2016 , The Netherlands

    Den Haag, Diagnosed 2016 , The Netherlands

  • Colm Francis Davis, Ireland

    Colm Francis Davis, Ireland

  • Imelda Arenas, ACELA, Colombia

    Imelda Arenas, ACELA, Colombia

  • Tammy Moore and Eddy Lefrancois

    Tammy Moore and Eddy Lefrancois

  • Anthony (Tony) Lynch, MND New South Wales, Diagnosed 2016, Australia

    Anthony (Tony) Lynch, MND New South Wales, Diagnosed 2016, Australia

  • Charlie “Hark” Dourney, Diagnosed 2007 , Hark ALS, USA

    Charlie “Hark” Dourney, Diagnosed 2007 , Hark ALS, USA

  • Claire Garry, USA

    Claire Garry, USA
    20200117_214643

  • Kris Van Reusel, Belgium

    Kris Van Reusel, Belgium

  • Conny van der Meijden, Diagnosed 2001,  ALS Netherlands

    Conny van der Meijden, Diagnosed 2001, ALS Netherlands

  • Lombana, Spain

    Lombana, Spain

  • Jose Espinosa, Argentina

    Jose Espinosa, Argentina

  • Jon Newsome, USA

    Jon Newsome, USA

  • Fabio Carvalho

    Fabio Carvalho

  • Bruno Leanza Mantegna, Diagnosed 1999 , AISLA Onlus, Italy

    Bruno Leanza Mantegna, Diagnosed 1999 , AISLA Onlus, Italy

  • Bayley, Australia

    Bayley, Australia

  • Calum Ferguson, Diagnosed 2010 , MND Scotland, UK

    Calum Ferguson, Diagnosed 2010 , MND Scotland, UK

  • John and Loretta Russo, USA

    John and Loretta Russo, USA
    final3878

  • Stephanie Christiansen Hall, Canada

    Stephanie Christiansen Hall, Canada

  • Jorge Melo, ABrELA, Brazil

    Jorge Melo, ABrELA, Brazil

  • Margreth Burger-Saile, Diagnosed 2011,  ALS Schweiz,  Switzerland

    Margreth Burger-Saile, Diagnosed 2011, ALS Schweiz, Switzerland

  • Mahmood Anwar, UK

    Mahmood Anwar, UK

  • Carlos Alberto Arango, Colombia

    Carlos Alberto Arango, Colombia

  • Ada Garrido Benavidez, Diagnosed 2016,  FYADENMAC, Mexico

    Ada Garrido Benavidez, Diagnosed 2016, FYADENMAC, Mexico

  • Jo Knowlton and her dog, Scotland

    Jo Knowlton and her dog, Scotland

  • Marcelo Farinelli, Diagnosed 2006, ABrELA, Brazil

    Marcelo Farinelli, Diagnosed 2006, ABrELA, Brazil

  • Malcolm Buck, Australia

    Malcolm Buck, Australia

  • Amparo Muriel Engativa, Colombia

    Amparo Muriel Engativa, Colombia

  • Aida Trzmiel de Guterman, Asociacion ELA Argentina, Diagnosed 2007, Argentina

    Aida Trzmiel de Guterman, Asociacion ELA Argentina, Diagnosed 2007, Argentina

  • Hans Dieter Olszewski, Diagnosed 2010 , DGM, Germany

    Hans Dieter Olszewski, Diagnosed 2010 , DGM, Germany

  • Oliver Juenke, Germany

    Oliver Juenke, Germany

  • Carlos Alberto Báez Murillo, ACELA, Colombia

    Carlos Alberto Báez Murillo, ACELA, Colombia

  • Olga Cosentino, Diagnosed 2013,  Asociación ELA Argentina

    Olga Cosentino, Diagnosed 2013, Asociación ELA Argentina

  • Bjarne Hytjanstorp, ALS Norge, Norway

    Bjarne Hytjanstorp, ALS Norge, Norway

  • Chih Ching Darren Wong, MND Malaysia

    Chih Ching Darren Wong, MND Malaysia

  • JP

    JP

  • Rosie Riley, Les Turner ALS Foundation, USA

    Rosie Riley, Les Turner ALS Foundation, USA

  • João Marcos Andrietta, Diagnosed 2008 , ABrELA, Brazil

    João Marcos Andrietta, Diagnosed 2008 , ABrELA, Brazil

  • Jean

    Jean
    jean

  • Ian Roberts

    Ian Roberts

  • Shay Rishoni

    Shay Rishoni

  • Elisabeth Zahnd, Switzerland

    Elisabeth Zahnd, Switzerland

  • Wilfried Leusing

    Wilfried Leusing

  • 393647_2252248542053_984912751_n

    393647_2252248542053_984912751_n

  • Dad

    Dad

  • Ian and Teresa Roberts

    Ian and Teresa Roberts

  • Marcel R. Wernard, Diagnosed 2016,  ALS Patients Connected,  The Netherlands

    Marcel R. Wernard, Diagnosed 2016, ALS Patients Connected, The Netherlands

  • Elkin Ramiro Gaviria Muñoz, Diagnosed December 2018

    Elkin Ramiro Gaviria Muñoz, Diagnosed December 2018

  • Susan Keldani, Les Turner ALS Foundation, USA

    Susan Keldani, Les Turner ALS Foundation, USA

  • David Solomon, Diagnosed 2015, MND Association of England, Wales and N Ireland

    David Solomon, Diagnosed 2015, MND Association of England, Wales and N Ireland

Learn more about the March of Faces

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  • Orphazyme – Arimoclomol
  • TUDCA Trial

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