1. Tauroursodeoxycholic acid in patients with amyotrophic lateral sclerosis: The TUDCA-ALS trial protocol
- Author
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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, Paolo Tornese, Antoniangela Cocco, Maria Lo Giudice, Michela Matteoli, Eliana Lauranzano, Maria Luisa Malosio, Chiara Adriana Elia, Flavia Lombardo, Flavia Mayer, Maria Puopolo, Stefania Spila Alegiani, Adriano Chiò, Umberto Manera, Cristina Moglia, Andrea Calvo, Paolina Salamone, Giuseppe Fuda, Carlo Colosimo, Cristina Spera, Prabha Cristina Ranchicchio, Giuseppe Stipa, Domenico Frondizi, Christian Lunetta, Valeria Sansone, Claudia Tarlarini, Francesca Gerardi, Vincenzo Silani, Alberto Doretti, Eleonora Colombo, Gianluca Demirtzidis, Gioacchino Tedeschi, Francesca Trojsi, Carla Passaniti, Stefania Ballestrero, Johannes Dorst, Ulrike Weiland, Andrea Fromm, Maximilian Wiesenfarth, Katharina Kandler, Simon Witzel, Markus Otto, Joachim Schuster, Thomas Meyer, André Maier, Dagmar Kettemann, Susanne Petri, Lars Müschen, Camilla Wohnrade, Anastasia Sarikidi, Alma Osmanovic, Julian Grosskreutz, Annekathrin Rödiger, Robert Steinbach, Benjamin Ilse, Uta Smesny, Robert Untucht, René Günther, Maximilian Vidovic, Pamela Shaw, Alexis Collins, Helen Wollff, Theresa Walsh, Lee Tuddenham, Mbombe Kazoka, David White, Stacy Young, Benjamin Thompson, Daniel Madarshahian, Suresh K Chhetri, Amina Chaouch, Carolyn A Young, Heike Arndt, Oliver C Hanemann, Thomas Lambert, Stephane Beltran, Philippe Couratier, Florence Esselin, William Camu, Elisa De, La Cruz, Gwendal Lemasson, Pegah Masrori, Sinead Maguire, Liz Fogarty, Toyosi Atoyebi, Niamh Ní Obáin, 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, Alberto, Albanese, Albert Christian, Ludolph, Christopher J, Mcdermott, Philippe, Corcia, Philip, Van Damme, Leonard H, Van den Berg, Orla, Hardiman, Gilberto, Rinaldi, Nicola, Vanacore, Brian, Dickie, Study Group, TUDCA-ALS, Lo Giudice, Maria, Luisa Malosio, Maria, Spila Alegiani, Stefania, Cristina Ranchicchio, Prabha, Dorst, Johanne, Otto, Marku, Meyer, Thoma, Müschen, Lar, Collins, Alexi, K Chhetri, Suresh, A Young, Carolyn, C Hanemann, Oliver, Lambert, Thoma, and Ní Obáin, Niamh
- Subjects
bile acids ,amyotrophic lateral sclerosis ,therapy ,Neurology ,bile acid ,clinical trial ,phase III ,amyotrophic lateral sclerosi ,Neurology (clinical) ,ddc:610 - Abstract
BackgroundAmyotrophic 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.MethodsThe 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.ConclusionThis 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 registrationClinicalTrials.gov, identifier: NCT03800524.
- Published
- 2022