Objective TUDCA-ALS will focus on amyotrophic lateral sclerosis (ALS) a rare neurodegenerative disorder, that affects motor neurons in the brain, brainstem and spinal cord, resulting in progressive weakness and atrophy of voluntary skeletal muscles. ALS has an estimated prevalence of 5.40 cases per 100.000 population corresponding at about 40.000 patients at European level. Treatments are modestly effective at best, and the majority of patients die within 3-5 years of diagnosis, often from respiratory failure.TUDCA-ALS is based on the results of a successful proof-of-concept trial and has the ambition to develop a novel therapy in patients with ALS, based on administration of tauroursodeoxycholic acid (TUDCA) in addition to riluzole. TUDCA-ALS aims to perform a state-of-art randomized control trial on safety and efficacy of TUDCA in patients with sporadic ALS, without cognitive involvement and to exploit the project results into a novel therapeutic treatment for ALS. The trial primary endpoint is deterioration of function measured with the ALS Functional Rating Scale Revised. The secondary endpoints are: (1) Survival time to invasive ventilation (tracheostomy) or death; (2) changes in quality of life parameters measured by the ALSAQ-40 questionnaire; (3) functional changes measured by Forced Vital Capacity, and the EQ-5D scale. Muscle force will be assessed by the MRC scale and a correlated analysis of function and survival will be performed. Two biomarkers will be measured: neurofilament levels in the CSF and serum and MMP-9 expression in serum. The latter is a possible biomarker of TUDCA efficacy. TUDCA-ALS is grounded on promising phase IIb clinical data and is expected to impact positively on the international research efforts on ALS.TUDCA-ALS aims to obtain concrete benefits for patients with ALS. If the trial will prove successful, the consortium will support the development of this treatment at European level by combining expert knowledge and patient cooperation. Fields of science medical and health sciencesbasic medicinepharmacology and pharmacypharmaceutical drugsmedical and health sciencesbasic medicineneurologyamyotrophic lateral sclerosis Keywords Amyotrophic lateral sclerosis disease modifying treatment neuroprotection deoxycholic acids Programme(s) H2020-EU.3.1. - SOCIETAL CHALLENGES - Health, demographic change and well-being Main Programme H2020-EU.3.1.3. - Treating and managing disease Topic(s) SC1-PM-08-2017 - New therapies for rare diseases Call for proposal H2020-SC1-2016-2017 See other projects for this call Sub call H2020-SC1-2017-Two-Stage-RTD Funding Scheme RIA - Research and Innovation action Coordinator HUMANITAS MIRASOLE SPA Net EU contribution € 1 541 619,50 Address VIA MANZONI 56 20100 Rozzano (Mi) Italy See on map Region Nord-Ovest Lombardia Milano Activity type Private for-profit entities (excluding Higher or Secondary Education Establishments) Links Contact the organisation Opens in new window Website Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Total cost € 1 541 619,50 Participants (9) Sort alphabetically Sort by Net EU contribution Expand all Collapse all UNIVERSITAET ULM Germany Net EU contribution € 647 125,00 Address HELMHOLTZSTRASSE 16 89081 Ulm See on map Region Baden-Württemberg Tübingen Ulm, Stadtkreis Activity type Higher or Secondary Education Establishments Links Contact the organisation Opens in new window Website Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Total cost € 647 125,00 THE UNIVERSITY OF SHEFFIELD United Kingdom Net EU contribution € 731 906,25 Address FIRTH COURT WESTERN BANK S10 2TN Sheffield See on map Region Yorkshire and the Humber South Yorkshire Sheffield Activity type Higher or Secondary Education Establishments Links Contact the organisation Opens in new window Website Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Total cost € 731 906,25 CENTRE HOSPITALIER REGIONAL UNIVERSITAIRE DE TOURS France Net EU contribution € 490 906,00 Address BOULEVARD TONELLE 2 37044 Tours Cedex 9 See on map Region Centre — Val de Loire Centre — Val de Loire Indre-et-Loire Activity type Higher or Secondary Education Establishments Links Contact the organisation Opens in new window Website Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Total cost € 490 906,00 KATHOLIEKE UNIVERSITEIT LEUVEN Belgium Net EU contribution € 352 281,25 Address OUDE MARKT 13 3000 Leuven See on map Region Vlaams Gewest Prov. Vlaams-Brabant Arr. Leuven Activity type Higher or Secondary Education Establishments Links Contact the organisation Opens in new window Website Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Total cost € 352 281,25 UNIVERSITAIR MEDISCH CENTRUM UTRECHT Netherlands Net EU contribution € 689 197,50 Address HEIDELBERGLAAN 100 3584 CX Utrecht See on map Region West-Nederland Utrecht Utrecht Activity type Higher or Secondary Education Establishments Links Contact the organisation Opens in new window Website Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Total cost € 689 197,50 THE PROVOST, FELLOWS, FOUNDATION SCHOLARS & THE OTHER MEMBERS OF BOARD, OF THE COLLEGE OF THE HOLY & UNDIVIDED TRINITY OF QUEEN ELIZABETH NEAR DUBLIN Ireland Net EU contribution € 351 086,25 Address COLLEGE GREEN TRINITY COLLEGE D02 CX56 DUBLIN 2 See on map Region Ireland Eastern and Midland Dublin Activity type Higher or Secondary Education Establishments Links Contact the organisation Opens in new window Website Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Total cost € 351 086,25 BRUSCHETTINI SRL Italy Net EU contribution € 516 868,75 Address VIA ISONZO 6 16147 Genova See on map SME The organization defined itself as SME (small and medium-sized enterprise) at the time the Grant Agreement was signed. Yes Region Nord-Ovest Liguria Genova Activity type Private for-profit entities (excluding Higher or Secondary Education Establishments) Links Contact the organisation Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Total cost € 516 868,75 ISTITUTO SUPERIORE DI SANITA Italy Net EU contribution € 239 375,00 Address Viale Regina Elena 299 00161 Roma See on map Region Centro (IT) Lazio Roma Activity type Research Organisations Links Contact the organisation Opens in new window Website Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Total cost € 239 375,00 MOTOR NEURONE DISEASE ASSOCIATION United Kingdom Net EU contribution € 36 562,50 Address 10 15 NOTRE DAME MEWS NN1 2BG Northampton See on map Region East Midlands (England) Leicestershire, Rutland and Northamptonshire West Northamptonshire Activity type Other Links Contact the organisation Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Total cost € 36 562,50