Objective Neurodegeneration with brain iron accumulation (NBIA) is a heterogeneous group of rare hereditary neurodegenerative disorders characterized by high levels of brain iron. The most common form is pantothenate kinase-associated neurodegeneration (PKAN). Classic PKAN and most other NBIA cases are characterised by early childhood onset and rapid progression. Currently, there is no proven therapy to halt or reverse PKAN or any other NBIA. This is especially unfortunate as both the iron accumulation in NBIA and the biochemical defect in PKAN are predicted to be amenable to drug-based treatment. Thus, the current absence of clinical trials is not due to lack of therapeutic options but to rarity of the disease, lack of patient registries and fragmentation of therapeutic research worldwide. For example, the iron-chelating drug deferiprone has been administered to PKAN patients on an individual basis or in pilot trials, both precluding firm conclusions about its efficacy. With TIRCON, we will address this urgent and unmet need for NBIA/PKAN therapy with an ambitious and highly collaborative plan that leverages worldwide expertise. We propose a large investigator-driven randomized clinical trial of deferiprone in PKAN, bringing together leading centres and patient advocacy groups from Europe and the US to reach the required patient cohort size. In addition, together with a European SME, we propose to pursue preclinical development of pantethine and its derivatives which have shown promising efficacy in a Drosophila PKAN model. To facilitate future research, we will develop a harmonized patient registry and biomaterial bank to allow for natural history studies and biomarker development, two critical needs in NBIA research. TIRCON partners, apart from their unique clinical and basic science expertise in NBIA, have longstanding experience in investigator-driven and industry-driven randomized clinical trials. Importantly, they have been closely collaborating in recent years. Fields of science natural sciencesbiological sciencesneurobiologyhumanitieshistory and archaeologyhistoryengineering and technologyindustrial biotechnologybiomaterials Programme(s) FP7-HEALTH - Specific Programme "Cooperation": Health Topic(s) HEALTH-2011.2.2.1-1 - Investigator-driven clinical trials for childhood-onset neurodegenerative diseases Call for proposal FP7-HEALTH-2011-two-stage See other projects for this call Funding Scheme CP-FP - Small or medium-scale focused research project Coordinator LUDWIG-MAXIMILIANS-UNIVERSITAET MUENCHEN Address Geschwister scholl platz 1 80539 Muenchen Germany See on map Region Bayern Oberbayern München, Kreisfreie Stadt Activity type Higher or Secondary Education Establishments Administrative Contact Thomas Klopstock (Prof.) Links Contact the organisation Opens in new window Website Opens in new window EU contribution No data Participants (12) Sort alphabetically Sort by EU Contribution Expand all Collapse all KLINIKUM RECHTS DER ISAR DER TECHNISCHEN UNIVERSITAT MUNCHEN Germany EU contribution € 844 340,00 Address Ismaninger strasse 22 81675 Muenchen See on map Region Bayern Oberbayern München, Kreisfreie Stadt Activity type Higher or Secondary Education Establishments Administrative Contact Renate Raedlein (Ms.) Links Contact the organisation Opens in new window Website Opens in new window Other funding No data INSTYTUT POMNIK CENTRUM ZDROWIA DZIECKA Poland EU contribution € 77 600,40 Address Aleja dzieci polskich 20 04730 Warszawa See on map Region Makroregion województwo mazowieckie Warszawski stołeczny Miasto Warszawa Activity type Research Organisations Administrative Contact Tomasz Kmiec (Dr.) Links Contact the organisation Opens in new window Website Opens in new window Other funding No data FONDAZIONE IRCCS ISTITUTO NEUROLOGICO CARLO BESTA Italy EU contribution € 403 432,95 Address Via giovanni celoria 11 20133 Milano See on map Region Nord-Ovest Lombardia Milano Activity type Research Organisations Administrative Contact Francesca Grechi (Dr.) Links Contact the organisation Opens in new window Website Opens in new window Other funding No data ACADEMISCH ZIEKENHUIS GRONINGEN Netherlands EU contribution € 387 000,00 Address Hanzeplein 1 9713 GZ Groningen See on map Region Noord-Nederland Groningen Overig Groningen Activity type Higher or Secondary Education Establishments Administrative Contact Johanna M Van Apeldoorn (Ms.) Links Contact the organisation Opens in new window Website Opens in new window Other funding No data OREGON HEALTH AND SCIENCE UNIVERSITY United States EU contribution € 281 700,00 Address 3181 sw sam jackson park road 97239-3098 Portland, oregon See on map Administrative Contact Susan Hayflick (Prof.) Links Contact the organisation Opens in new window Other funding No data CHILDREN'S HOSPITAL & RESEARCH CENTER AT OAKLAND United States EU contribution € 248 388,85 Address 52nd street 747 94609 Oakland ca See on map Activity type Research Organisations Administrative Contact Rebecca Rosales (Ms.) Links Contact the organisation Opens in new window Website Opens in new window Other funding No data UNIVERSITY OF NEWCASTLE UPON TYNE United Kingdom EU contribution € 244 094,92 Address Kings gate NE1 7RU Newcastle upon tyne See on map Region North East (England) Northumberland and Tyne and Wear Tyneside Activity type Higher or Secondary Education Establishments Administrative Contact Amanda Gregory (Dr.) Links Contact the organisation Opens in new window Website Opens in new window Other funding No data ACIES BIO BIOTEHNOLOSKE RAZISKAVE IN RAZVOJ DOO Slovenia EU contribution € 394 000,00 Address Tehnoloski park 21 1000 Ljubljana See on map Region Slovenija Zahodna Slovenija Osrednjeslovenska Activity type Private for-profit entities (excluding Higher or Secondary Education Establishments) Administrative Contact Enej Kuscer (Dr.) Links Contact the organisation Opens in new window Website Opens in new window Other funding No data NBIA DISORDERS ASSOCIATION United States EU contribution € 114 720,00 Address Monaco court 2082 92019-4235 El cajon ca See on map Activity type Other Administrative Contact Patricia Wood (Ms.) Links Contact the organisation Opens in new window Website Opens in new window Other funding No data HOFFNUNGSBAUM EV VEREIN ZUR FORDERUNG DER ERFORSCHUNG UND BEHANDLUNG VON NBIA (VORMALS: HALLERVORDEN-SPATZ-SYNDROM)*TREE OF HOPE EV Germany EU contribution € 153 600,00 Address Hardenberger strasse 73 42549 Velbert See on map Activity type Other Administrative Contact Angelika Klucken (Mrs.) Links Contact the organisation Opens in new window Website Opens in new window Other funding No data BAYERISCHE FORSCHUNGSALLIANZ BAVARIAN RESEARCH ALLIANCE GMBH Germany EU contribution € 246 251,60 Address Prinzregentenstrasse 52 80538 Munchen See on map Region Bayern Oberbayern München, Kreisfreie Stadt Activity type Other Administrative Contact Florence Gauzy (Dr.) Links Contact the organisation Opens in new window Website Opens in new window Other funding No data APO-PHARMA INC CORP Canada EU contribution € 473 382,66 Address Signet drive 150 M9L 1T9 Toronto See on map Activity type Other Administrative Contact Michael Spino (Dr.) Links Contact the organisation Opens in new window Website Opens in new window Other funding No data
