DEVELOPAKURE
Clinical Development of Nitisinone for Alkaptonuria
| Coordinatore | ROYAL LIVERPOOL AND BROADGREEN UNIVERSITY HOSPITALS NHS TRUST
Organization address
address: PRESCOT STREET contact info |
| Nazionalità Coordinatore | United Kingdom [UK] |
| Sito del progetto | http://www.developakure.eu |
| Totale costo | 10˙996˙020 € |
| EC contributo | 5˙999˙999 € |
| Programma | FP7-HEALTH
Specific Programme "Cooperation": Health |
| Code Call | FP7-HEALTH-2012-INNOVATION-1 |
| Funding Scheme | CP-FP |
| Anno di inizio | 2012 |
| Periodo (anno-mese-giorno) | 2012-11-01 - 2018-04-30 |
Partecipanti
| # | ||||
|---|---|---|---|---|
| 1 |
ROYAL LIVERPOOL AND BROADGREEN UNIVERSITY HOSPITALS NHS TRUST
Organization address
address: PRESCOT STREET contact info |
UK (LIVERPOOL) | coordinator | 1˙540˙236.40 |
| 2 |
PSR Group BV
Organization address
address: Planetenweg 5 contact info |
NL (HOOFDDORP) | participant | 762˙028.00 |
| 3 |
THE UNIVERSITY OF LIVERPOOL
Organization address
address: Brownlow Hill, Foundation Building 765 contact info |
UK (LIVERPOOL) | participant | 653˙620.00 |
| 4 |
NARODNY USTAV REUMATICKYCH CHOROB-NURCH NIRD NATIONAL INSTITUTE OF RHEUMATIC DISEASES
Organization address
address: NABREZIE IVANA KRASKU 4 contact info |
SK (PIESTANY) | participant | 515˙307.50 |
| 5 |
SWEDISH ORPHAN BIOVITRUM AB (PUBL)
Organization address
address: TOMTEBODAVAGEN 23a contact info |
SE (Stockholm) | participant | 515˙000.00 |
| 6 |
NORDIC BIOSCIENCE COMPOUND DEVELOPMENT A/S
Organization address
address: Herlev Hovedgade 207 contact info |
DK (Helev) | participant | 499˙544.60 |
| 7 |
THE ALKAPTONURIA SOCIETY LTD
Organization address
address: PAGET ROAD 109 TRUMPINGTON contact info |
UK (CAMBRIDGE) | participant | 452˙358.00 |
| 8 |
ASSISTANCE PUBLIQUE - HOPITAUX DE PARIS
Organization address
address: 3 Avenue Victoria contact info |
FR (PARIS) | participant | 439˙855.70 |
| 9 |
ASSOCIATION INSTITUT NECKER
Organization address
address: RUE DE VAUGIRARD 156 contact info |
FR (PARIS) | participant | 219˙790.00 |
| 10 |
ASSOCIATION POUR LA LUTTE CONTRE L'ALCAPTONURIE
Organization address
address: CHEMIN DU CLOS-ST-MARTIN 18 contact info |
FR (L'ETANG-LA-VILLE) | participant | 162˙570.00 |
| 11 |
UNIVERSITA' DEGLI STUDI DI SIENA
Organization address
address: VIA BANCHI DI SOTTO 55 contact info |
IT (SIENA) | participant | 101˙339.00 |
| 12 |
CUDOS BV
Organization address
address: PLANETENWEG 5 contact info |
NL (HOOFDDORP) | participant | 79˙474.80 |
| 13 |
USTAV MOLEKULARNEJ FYZIOLOGIE A GENETIKY SLOVENSKEJ AKADEMIE VIED
Organization address
address: Vlarska 5 contact info |
SK (BRATISLAVA) | participant | 58˙875.00 |
Mappa
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Obiettivo del progetto (Objective)
'DevelopAKUre is a proposal to fund the clinical development of an orphan designated drug, nitisinone, for the treatment of a rare Mendelian disease, Alkaptonuria (AKU). AKU is a genetic deficiency of homogentisic acid dioxygenase, causing high levels of homogentisic acid (HGA). Oxidation of HGA to pigment polymer, termed ochronosis, alters connective tissues. This leads to multisystemic damage dominated by premature severe arthritis. Currently, multiple arthroplasty is inevitable since AKU is incurable and there is no effective palliative therapy. No data exists regarding the presence or absence of ochronosis before age 30 years. Hence, it is unknown whether treatment is necessary before then. A potential HGA-lowering therapy with nitisinone is available, but lacks outcome data. Thanks to our existing successful fundamental and clinical research (cell models, animal models, natural history studies), we are now ready for the final stage of clinical development of nitisinone for AKU in order to overcome these challenges. This will involve a dose finding study, a phase 3 clinical trial to prove efficacy, and a cross-sectional study in children and young adults to determine when to start treatment. The results of DevelopAKUre will allow us to make the case to the European Medicines Agency for marketing authorisation of nitisinone for AKU, thereby contributing to the goal of the International Rare Diseases Research Consortium of 200 new therapies by 2020. Our consortium has worked together for five years already. It includes Liverpool University’s AKU Research Team as the lead applicant, the AKU Society UK patient group for dissemination and patient recruitment, three SMEs (Denmark, Netherlands) for biomarker analysis and clinical trial coordination, an industry partner (Sweden) supplying the drug and regulatory support, three universities (UK, Italy, Slovakia) for the analysis of data, and three clinical trial centres (UK, France, Slovakia) to reach required numbers.'
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