Obiettivo Hemophagocytic lymphohistiocytosis (HLH) is a rare, devastating disease characterized by uncontrolled immune response that primarily affects young infants and children. HLH is usually fatal if untreated. Even when treated, the overall survival rate is still as low as 60-70%. No drug has been formally developed for the treatment of this disease and current treatment methods require the use of drugs which have not formally been developed for this indication which have an unfavourable safety profile. The cytokine interferon-gamma (IFNγ) was shown to play a key pathological role in the disease. It has been demonstrated that neutralization of IFNγ with a monoclonal antibody (mAb) reverts the disease and rescues animals from death in murine models of primary HLH. In patients, evidence points towards a correlation between levels of IFNγ and disease activity. In order to target IFNγ in patients with HLH, NovImmune generated and characterized a fully human mAb, NI-0501, that neutralizes the biological activity of human IFNγ. This consortium proposes an adaptive clinical trial with a pilot and a pivotal phase to provide the necessary data for Market Access Authorization for NI-0501 in HLH. The pilot phase will enroll only patients well-known to have a primary form of HLH and who relapsed after having responded to an initial treatment while the pivotal phase will open the recruitment to newly diagnosed HLH patients. The transition between the pilot and the pivotal phase will be governed by strict pre-defined transition rules and will only occur if a favorable benefit-risk profile has been demonstrated. During the course of the pilot phase the consortium will attempt to generate the necessary information to justify the inclusion in the pivotal phase of a broad range of HLH patient (secondary forms of the disease), for which the pivotal role of IFNγ will have been demonstrated thanks to the research activities of members of the consortium. Campo scientifico medical and health sciencesbasic medicineimmunology Programma(i) FP7-HEALTH - Specific Programme "Cooperation": Health Argomento(i) HEALTH.2012.2.4.4-1 - Preclinical and/or clinical development of substances with a clear potential as orphan drugs Invito a presentare proposte FP7-HEALTH-2012-INNOVATION-1 Vedi altri progetti per questo bando Meccanismo di finanziamento CP-FP - Small or medium-scale focused research project Coordinatore NOVIMMUNE SA Contributo UE € 4 524 535,00 Indirizzo CHEMIN DES AULX 14 1228 Plan Les Ouates Geneve Svizzera Mostra sulla mappa Regione Schweiz/Suisse/Svizzera Région lémanique Genève Tipo di attività Private for-profit entities (excluding Higher or Secondary Education Establishments) Contatto amministrativo Nathalie Muller (Ms.) Collegamenti Contatta l’organizzazione Opens in new window Sito web Opens in new window Costo totale Nessun dato Partecipanti (5) Classifica in ordine alfabetico Classifica per Contributo UE Espandi tutto Riduci tutto LONZA BIOLOGICS PLC Regno Unito Contributo UE € 199 200,00 Indirizzo BATH ROAD 228 SL1 4DX LONDON Mostra sulla mappa Tipo di attività Private for-profit entities (excluding Higher or Secondary Education Establishments) Contatto amministrativo Gerry Kennedy (Mr.) Collegamenti Contatta l’organizzazione Opens in new window Sito web Opens in new window Costo totale Nessun dato SINTESI RESEARCH SRL Italia Contributo UE € 476 026,80 Indirizzo VIA RIPAMONTI GIUSEPPE 89 20141 MILANO Mostra sulla mappa Tipo di attività Private for-profit entities (excluding Higher or Secondary Education Establishments) Contatto amministrativo Paolo De Simoni (Dr.) Collegamenti Contatta l’organizzazione Opens in new window Sito web Opens in new window Costo totale Nessun dato OSPEDALE PEDIATRICO BAMBINO GESU Italia Contributo UE € 463 488,70 Indirizzo PIAZZA SANT ONOFRIO 4 00165 Roma Mostra sulla mappa Regione Centro (IT) Lazio Roma Tipo di attività Research Organisations Contatto amministrativo Sonya Jane Martin (Dr.) Collegamenti Contatta l’organizzazione Opens in new window Sito web Opens in new window Costo totale Nessun dato AZIENDA OSPEDALIERA UNIVERSITARIA MEYER IRCCS Italia Contributo UE € 84 201,30 Indirizzo VIALE PIERACCINI 24 50139 Firenze Mostra sulla mappa Regione Centro (IT) Toscana Firenze Tipo di attività Research Organisations Contatto amministrativo Matteo Sammartino (Dr.) Collegamenti Contatta l’organizzazione Opens in new window Sito web Opens in new window Costo totale Nessun dato PATERGRUS BVBA Belgio Contributo UE € 198 810,00 Indirizzo SPARHOEKDREEF 57 9880 Aalter Mostra sulla mappa Regione Vlaams Gewest Prov. Oost-Vlaanderen Arr. Gent Tipo di attività Private for-profit entities (excluding Higher or Secondary Education Establishments) Contatto amministrativo Marc Van De Craen (Dr.) Collegamenti Contatta l’organizzazione Opens in new window Sito web Opens in new window Costo totale Nessun dato
