Innovating Works

FAIR-PARK-II

Financiado
Conservative iron chelation as a disease modifying strategy in Parkinson s disea...
Conservative iron chelation as a disease modifying strategy in Parkinson s disease a multicentric parallel group placebo controlled randomized clinical trial of deferiprone Parkinson’s disease (PD) is a major, chronic, non-communicable disease and the 2nd most frequent neurodegenerative disorder worldwide. Excess iron is primarily detected in the substantia nigra pars compacta, where dopaminergic neu... Parkinson’s disease (PD) is a major, chronic, non-communicable disease and the 2nd most frequent neurodegenerative disorder worldwide. Excess iron is primarily detected in the substantia nigra pars compacta, where dopaminergic neurons are exposed to high levels of oxidative stress produced by mitochondrial disorders and dopamine metabolism. Our previous preclinical, translational and pilot clinical studies demonstrated that novel iron chelation therapy with the prototypic drug deferiprone (DFP) (i) induces neuroprotection in cell models of PD via a powerful antioxidant effect, (ii) reduces regional siderosis of the brain, (iii) reduces motor handicap via inhibition of catechol-o-methyl transferase, and (iv) slows the progression of motor handicap in the 1-methyl-4-phenyl-1,2,3,6-tetrahydropyridine mouse model and in early PD patients. This project now seeks to demonstrate that conservative iron chelation therapy with moderate-dose DFP (30 mg/kg/day) slows the progression of handicap in de novo PD patients while not affecting systemic parameters. The 9-month, parallel-group, randomized, placebo-controlled, multicentre trial will be followed by a 1-month wash-out period. The primary efficacy criterion will be the change in motor and non-motor handicap scores on the Total Movement Disorders Society Unified Parkinson’s Disease Rating Scale to identify disease-modifying and symptomatic effects. The secondary efficacy criterion will be the change in score between baseline and 40 weeks (i.e. probing the disease-modifying effect only). Potential surrogate radiological and biological biomarkers, health economics and societal impacts will be assessed. 17 national, European and international research and innovation activities will be linked with the project. The study results should prompt academic and industrial research on iron chelation as a disease-modifying treatment in neurodegenerative diseases. ver más
31/10/2021
RAILENIUM
8M€
Duración del proyecto: 78 meses Fecha Inicio: 2015-04-14
Fecha Fin: 2021-10-31

Línea de financiación: concedida

El organismo H2020 notifico la concesión del proyecto el día 2021-10-31
Línea de financiación objetivo El proyecto se financió a través de la siguiente ayuda:
PHC-13-2014: New therapies for chronic non-communicable diseases
Cerrada hace 10 años
Presupuesto El presupuesto total del proyecto asciende a 8M€
Líder del proyecto
RAILENIUM No se ha especificado una descripción o un objeto social para esta compañía.