Skip to main content

Safety and efficacy of tauroursodeoxycholic acid (TUDCA) as add-on treatment in patients affected by amyotrophic lateral sclerosis (ALS)

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.

Field of science

  • /medical and health sciences/basic medicine/neurology/amyotrophic lateral sclerosis

Call for proposal

H2020-SC1-2017-Two-Stage-RTD
See other projects for this call

Funding Scheme

RIA - Research and Innovation action
Leaflet | Map data © OpenStreetMap contributors, Credit: EC-GISCO, © EuroGeographics for the administrative boundaries

Coordinator

HUMANITAS MIRASOLE SPA
Address
Via Manzoni 56
20100 Rozzano (Mi)
Italy
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
EU contribution
€ 1 526 994,50

Participants (9)

UNIVERSITAET ULM
Germany
EU contribution
€ 647 125
Address
Helmholtzstrasse 16
89081 Ulm
Activity type
Higher or Secondary Education Establishments
THE UNIVERSITY OF SHEFFIELD
United Kingdom
EU contribution
€ 731 906,25
Address
Firth Court Western Bank
S10 2TN Sheffield
Activity type
Higher or Secondary Education Establishments
CENTRE HOSPITALIER REGIONAL UNIVERSITAIRE DE TOURS
France
EU contribution
€ 490 906
Address
Boulevard Tonelle 2
37044 Tours Cedex 9
Activity type
Higher or Secondary Education Establishments
KATHOLIEKE UNIVERSITEIT LEUVEN
Belgium
EU contribution
€ 352 281,25
Address
Oude Markt 13
3000 Leuven
Activity type
Higher or Secondary Education Establishments
UNIVERSITAIR MEDISCH CENTRUM UTRECHT
Netherlands
EU contribution
€ 689 197,50
Address
Heidelberglaan 100
3584 CX Utrecht
Activity type
Higher or Secondary Education Establishments
THE PROVOST, FELLOWS, FOUNDATION SCHOLARS & THE OTHER MEMBERS OF BOARD OF THE COLLEGE OF THE HOLY & UNDIVIDED TRINITY OF QUEEN ELIZABETH NEAR DUBLIN
Ireland
EU contribution
€ 351 086,25
Address
College Green
2 Dublin
Activity type
Higher or Secondary Education Establishments
BRUSCHETTINI SRL
Italy
EU contribution
€ 516 868,75
Address
Via Isonzo 6
16147 Genova
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
ISTITUTO SUPERIORE DI SANITA
Italy
EU contribution
€ 239 375
Address
Viale Regina Elena 299
00161 Roma
Activity type
Research Organisations
MOTOR NEURONE DISEASE ASSOCIATION
United Kingdom
EU contribution
€ 51 187,50
Address
10 15 Notre Dame Mews
NN1 2BG Northampton
Activity type
Other