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Treat Iron-Related Childhood-Onset Neurodegeneration

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.
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Coordinator

LUDWIG-MAXIMILIANS-UNIVERSITAET MUENCHEN

Address

Geschwister Scholl Platz 1
80539 Muenchen

Germany

Activity type

Higher or Secondary Education Establishments

EU Contribution

€ 1 346 708,42

Administrative Contact

Thomas Klopstock (Prof.)

Participants (12)

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KLINIKUM RECHTS DER ISAR DER TECHNISCHEN UNIVERSITAT MUNCHEN

Germany

EU Contribution

€ 844 340

INSTYTUT POMNIK CENTRUM ZDROWIA DZIECKA

Poland

EU Contribution

€ 77 600,40

FONDAZIONE IRCCS ISTITUTO NEUROLOGICO CARLO BESTA

Italy

EU Contribution

€ 403 432,95

ACADEMISCH ZIEKENHUIS GRONINGEN

Netherlands

EU Contribution

€ 387 000

OREGON HEALTH AND SCIENCE UNIVERSITY

United States

EU Contribution

€ 281 700

CHILDREN'S HOSPITAL & RESEARCH CENTER AT OAKLAND

United States

EU Contribution

€ 248 388,85

UNIVERSITY OF NEWCASTLE UPON TYNE

United Kingdom

EU Contribution

€ 244 094,92

ACIES BIO BIOTEHNOLOSKE RAZISKAVE IN RAZVOJ DOO

Slovenia

EU Contribution

€ 394 000

NBIA DISORDERS ASSOCIATION

United States

EU Contribution

€ 114 720

HOFFNUNGSBAUM EV VEREIN ZUR FORDERUNG DER ERFORSCHUNG UND BEHANDLUNG VON NBIA (VORMALS: HALLERVORDEN-SPATZ-SYNDROM)*TREE OF HOPE EV

Germany

EU Contribution

€ 153 600

BAYERISCHE FORSCHUNGSALLIANZ BAVARIAN RESEARCH ALLIANCE GMBH

Germany

EU Contribution

€ 246 251,60

APO-PHARMA INC CORP

Canada

EU Contribution

€ 473 382,66

Project information

Grant agreement ID: 277984

Status

Closed project

  • Start date

    1 November 2011

  • End date

    31 October 2015

Funded under:

FP7-HEALTH

  • Overall budget:

    € 6 977 141,23

  • EU contribution

    € 5 215 219,80

Coordinated by:

LUDWIG-MAXIMILIANS-UNIVERSITAET MUENCHEN

Germany