Skip to main content

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

Objectif

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.

Appel à propositions

H2020-PHC-2014-two-stage
Voir d’autres projets de cet appel

Régime de financement

RIA - Research and Innovation action

Coordinateur

CENTRE HOSPITALIER REGIONAL ET UNIVERSITAIRE DE LILLE
Adresse
Avenue Oscar Lambret 2
59037 Lille
France
Type d’activité
Higher or Secondary Education Establishments
Contribution de l’UE
€ 2 604 799,55

Participants (16)

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

Participation terminée

Allemagne
Contribution de l’UE
€ 48 078,38
Adresse
Geschwister-scholl-platz
72074 Tuebingen
Type d’activité
Higher or Secondary Education Establishments
MEDIZINISCHE UNIVERSITAT INNSBRUCK
Autriche
Contribution de l’UE
€ 226 735
Adresse
Christoph Probst Platz 1
6020 Innsbruck
Type d’activité
Higher or Secondary Education Establishments
UNIVERZITA KARLOVA
Tchéquie
Contribution de l’UE
€ 145 000
Adresse
Ovocny Trh 560/5
116 36 Praha 1
Type d’activité
Higher or Secondary Education Establishments
STICHTING RADBOUD UNIVERSITEIT
Pays-Bas
Contribution de l’UE
€ 256 250
Adresse
Houtlaan 4
6525 XZ Nijmegen
Type d’activité
Higher or Secondary Education Establishments
PHILIPPS UNIVERSITAET MARBURG
Allemagne
Contribution de l’UE
€ 74 875
Adresse
Biegenstrasse 10
35037 Marburg
Type d’activité
Higher or Secondary Education Establishments
SORBONNE UNIVERSITE
France
Contribution de l’UE
€ 82 550
Adresse
21 Rue De L'ecole De Medecine
75006 Paris
Type d’activité
Higher or Secondary Education Establishments
UNIVERSITAET ULM
Allemagne
Contribution de l’UE
€ 90 000
Adresse
Helmholtzstrasse 16
89081 Ulm
Type d’activité
Higher or Secondary Education Establishments
CHRISTIAN-ALBRECHTS-UNIVERSITAET ZU KIEL

Participation terminée

Allemagne
Contribution de l’UE
€ 378 071,50
Adresse
Olshausenstrasse 40
24118 Kiel
Type d’activité
Higher or Secondary Education Establishments
UNIVERSITATSKLINIKUM SCHLESWIG-HOLSTEIN
Allemagne
Contribution de l’UE
€ 31 181,32
Adresse
Ratzeburger Allee 160
23562 Lübeck
Type d’activité
Higher or Secondary Education Establishments