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Clinical Development of Nitisinone for Alkaptonuria

Objectif

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.

Champ scientifique

Programme(s)

Thème(s)

Appel à propositions

FP7-HEALTH-2012-INNOVATION-1
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Régime de financement

CP-FP - Small or medium-scale focused research project

Coordinateur

ROYAL LIVERPOOL AND BROADGREEN UNIVERSITY HOSPITALS NHS TRUST
Contribution de l’UE
€ 1 540 236,42
Adresse
PRESCOT STREET
L7 8XP LIVERPOOL
Royaume-Uni

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Région
North West (England) Merseyside Liverpool
Type d’activité
Research Organisations
Contact administratif
Lakshminarayan Ranganath (Dr.)
Liens
Coût total
Aucune donnée

Participants (14)

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Dernière mise à jour: 26 Avril 2022

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Permalink: https://cordis.europa.eu/project/id/304985/fr

European Union, 2024