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

Project information

Grant agreement ID: 755094

Status

Ongoing project

  • Start date

    1 January 2018

  • End date

    31 December 2021

Funded under:

H2020-EU.3.1.3.

  • Overall budget:

    € 5 596 928

  • EU contribution

    € 5 596 928

Coordinated by:

HUMANITAS MIRASOLE SPA

Italy

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.

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

THE UNIVERSITY OF SHEFFIELD

United Kingdom

EU Contribution

€ 731 906,25

CENTRE HOSPITALIER REGIONAL UNIVERSITAIRE DE TOURS

France

EU Contribution

€ 490 906

KATHOLIEKE UNIVERSITEIT LEUVEN

Belgium

EU Contribution

€ 352 281,25

UNIVERSITAIR MEDISCH CENTRUM UTRECHT

Netherlands

EU Contribution

€ 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

EU Contribution

€ 351 086,25

BRUSCHETTINI SRL

Italy

EU Contribution

€ 516 868,75

ISTITUTO SUPERIORE DI SANITA

Italy

EU Contribution

€ 239 375

MOTOR NEURONE DISEASE ASSOCIATION

United Kingdom

EU Contribution

€ 51 187,50

Project information

Grant agreement ID: 755094

Status

Ongoing project

  • Start date

    1 January 2018

  • End date

    31 December 2021

Funded under:

H2020-EU.3.1.3.

  • Overall budget:

    € 5 596 928

  • EU contribution

    € 5 596 928

Coordinated by:

HUMANITAS MIRASOLE SPA

Italy