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Conservative iron chelation as a disease-modifying strategy in Parkinson’s disease: a multicentric, parallel-group, placebo-controlled, randomized clinical trial of deferiprone

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.

Field of science

  • /social sciences/economics and business/economics
  • /natural sciences/chemical sciences/inorganic chemistry/inorganic compounds
  • /social sciences/economics and business
  • /medical and health sciences/basic medicine/neurology/parkinson

Call for proposal

H2020-PHC-2014-two-stage
See other projects for this call

Funding Scheme

RIA - Research and Innovation action

Coordinator

CENTRE HOSPITALIER REGIONAL ET UNIVERSITAIRE DE LILLE
Address
Avenue Oscar Lambret 2
59037 Lille
France
Activity type
Higher or Secondary Education Establishments
EU contribution
€ 2 662 169,75

Participants (15)

INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE
France
EU contribution
€ 642 888
Address
Rue De Tolbiac 101
75654 Paris
Activity type
Research Organisations
ECRIN EUROPEAN CLINICAL RESEARCH INFRASTRUCTURE NETWORK
France
EU contribution
€ 331 848
Address
5 Rue Watt
75013 Paris
Activity type
Research Organisations
INSERM TRANSFERT SA
France
EU contribution
€ 389 491
Address
Rue Watt 7
75013 Paris
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
APO-PHARMA INC CORP
Canada
EU contribution
€ 0
Address
Signet Drive 150
M9L 1T9 Toronto
Activity type
Other
INSTITUTO DE MEDICINA MOLECULAR JOAO LOBO ANTUNES
Portugal
EU contribution
€ 187 083
Address
Avenida Prof Egas Moniz
1649 028 Lisboa
Activity type
Research Organisations
FUNDACIO CLINIC PER A LA RECERCA BIOMEDICA
Spain
EU contribution
€ 196 773,25
Address
Carrer Rossello 149
08036 Barcelona
Activity type
Research Organisations
UNIVERSITY OF NEWCASTLE UPON TYNE
United Kingdom
EU contribution
€ 313 370
Address
Kings Gate
NE1 7RU Newcastle Upon Tyne
Activity type
Higher or Secondary Education Establishments
EBERHARD KARLS UNIVERSITAET TUEBINGEN

Participation ended

Germany
EU contribution
€ 48 078,38
Address
Geschwister-scholl-platz
72074 Tuebingen
Activity type
Higher or Secondary Education Establishments
MEDIZINISCHE UNIVERSITAT INNSBRUCK
Austria
EU contribution
€ 226 735
Address
Christoph Probst Platz 1
6020 Innsbruck
Activity type
Higher or Secondary Education Establishments
UNIVERZITA KARLOVA
Czechia
EU contribution
€ 145 000
Address
Ovocny Trh 560/5
116 36 Praha 1
Activity type
Higher or Secondary Education Establishments
STICHTING KATHOLIEKE UNIVERSITEIT
Netherlands
EU contribution
€ 256 250
Address
Geert Grooteplein Noord 9
6525 EZ Nijmegen
Activity type
Higher or Secondary Education Establishments
PHILIPPS UNIVERSITAET MARBURG
Germany
EU contribution
€ 74 875
Address
Biegenstrasse 10
35037 Marburg
Activity type
Higher or Secondary Education Establishments
SORBONNE UNIVERSITE
France
EU contribution
€ 82 550
Address
21 Rue De L'ecole De Medecine
75006 Paris
Activity type
Higher or Secondary Education Establishments
UNIVERSITAET ULM
Germany
EU contribution
€ 90 000
Address
Helmholtzstrasse 16
89081 Ulm
Activity type
Higher or Secondary Education Establishments
CHRISTIAN-ALBRECHTS-UNIVERSITAET ZU KIEL
Germany
EU contribution
€ 351 882,62
Address
Olshausenstrasse 40
24118 Kiel
Activity type
Higher or Secondary Education Establishments