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Financement de l’UE (5 596 928 €) : Innocuité et efficacité de l’acide tauroursodésoxycholique (TUDCA) comme traitement d’appoint chez les patients atteints de sclérose latérale amyotrophique (SLA) Hor 01/01/2018 Programme de recherche et d'innovation de l'UE « Horizon »
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Innocuité et efficacité de l’acide tauroursodésoxycholique (TUDCA) comme traitement d’appoint chez les patients atteints de sclérose latérale amyotrophique (SLA)
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.
| Bruschettini Srl | 516 869 € |
| Centre Hospitalier Regional Universitaire de Tours | 490 906 € |
| Humanitas Mirasole S.p.A. | 1 541 620 € |
| Istituto Superiore Di Sanita | 239 375 € |
| Katholieke Universiteit Leuven | 352 281 € |
| Motor Neurone Disease Association | 36 563 € |
| The Provost, Fellows Foundation Scholars & the Other Members of Board, of the College of the Holy & Undivided Trinity of Queen Elizabeth Near Dublin | 351 086 € |
| The University of Sheffield | 731 906 € |
| Universitaet ULM | 647 125 € |
| Universitair Medisch Centrum Utrecht | 689 198 € |
https://cordis.europa.eu/project/id/755094
Cette annonce se réfère à une date antérieure et ne reflète pas nécessairement l’état actuel.