Objective
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.
Fields of science
Programme(s)
Call for proposal
H2020-PHC-2014-two-stage
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Funding Scheme
RIA - Research and Innovation actionCoordinator
59037 Lille
France
Participants (18)
75654 Paris
75013 Paris
75013 Paris
Participation ended
M9L 1T9 Toronto
1649 028 Lisboa
08036 Barcelona
NE1 7RU Newcastle Upon Tyne
Participation ended
72074 Tuebingen
6020 Innsbruck
116 36 Praha 1
Participation ended
6525 XZ Nijmegen
35037 Marburg
75006 Paris
89081 Ulm
Participation ended
24118 Kiel
23562 Lübeck
6525 GA Nijmegen
43122 Parma