Objective The main objective of the project is to provide a treatment of the neurological symptoms of patients with Ataxia Telangiectasia (AT), a rare progressively disabling and life-shortening genetic disease for which no therapy is currently available. To achieve this, a pivotal Phase III study will be conducted, to allow regulatory filing to obtain market authorization in EU and USA by 2019. EryDex is an innovative product, developed by EryDel, used to administer dexamethasone sodium phosphate by ex-vivo encapsulation in autologous erythrocytes, which are infused into the patient. EryDex provides long-term delivery of low doses of dexamethasone without the typical steroid side effects and has reached a successful Phase II trial conducted in AT patients. The phase III trial will be an international, multi-center, 1 year, randomized, prospective, double-blind, placebo-controlled, designed to assess the effect of 2 dose ranges of EryDex, administered monthly by IV infusion, on neurological symptoms of AT patients. The protocol of the trial and the regulatory path to registration has already been agreed upon with EMA and FDA.An international patient registry will also be set with the aim of establishing and maintaining a comprehensive clinical database of patients with AT and closely related conditions, enabling the monitoring of AT epidemiology, the development of an evidence-based natural history of the condition, identification of biomarkers as well as development of clinical guidelines.The AT NEST, the first scale to assess symptoms specific to AT patients, coordinated by the AT centre at the John’s Hopkins University, will be tested in the study and if validated will represent the 1st scale assessing chief areas of impairment specific to AT. In parallel to the clinical trial, investigations into the molecular mechanisms of action of EryDex will be performed with the objective to provide the validation of a new biomarker predictive of treatment efficacy. Fields of science natural sciencescomputer and information sciencesdatabaseshumanitieshistory and archaeologyhistorynatural scienceschemical sciencesinorganic chemistryalkali metalsmedical and health scienceshealth sciencespublic healthepidemiology Programme(s) H2020-EU.3.1. - SOCIETAL CHALLENGES - Health, demographic change and well-being Main Programme H2020-EU.3.1.3. - Treating and managing disease Topic(s) PHC-14-2015 - New therapies for rare diseases Call for proposal H2020-PHC-2014-2015 See other projects for this call Sub call H2020-PHC-2015-two-stage Funding Scheme RIA - Research and Innovation action Coordinator ERYDEL SPA Net EU contribution € 4 331 575,00 Address Via meucci 3 20091 Bresso Italy See on map SME The organization defined itself as SME (small and medium-sized enterprise) at the time the Grant Agreement was signed. Yes Region Nord-Ovest Lombardia Milano Activity type Private for-profit entities (excluding Higher or Secondary Education Establishments) Links Contact the organisation Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Other funding € 0,00 Participants (6) Sort alphabetically Sort by Net EU contribution Expand all Collapse all JOHNS HOPKINS UNIVERSITY United States Net EU contribution € 217 500,00 Address North charles street 3400 21218 Baltimore See on map Activity type Higher or Secondary Education Establishments Links Contact the organisation Opens in new window Website Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Other funding € 0,00 MEDICAL RESEARCH INFRASTRUCTURE DEVELOPMENT AND HEALTH SERVICES FUND BY THE SHEBA MEDICAL CENTER Israel Net EU contribution € 270 000,00 Address Tel hashomer sheba medical center 52621 Ramat gan See on map Activity type Research Organisations Links Contact the organisation Opens in new window Website Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Other funding € 0,00 JOHANN WOLFGANG GOETHE-UNIVERSITAET FRANKFURT AM MAIN Germany Net EU contribution € 256 250,00 Address Theodor w adorno platz 1 60323 Frankfurt am main See on map Region Hessen Darmstadt Frankfurt am Main, Kreisfreie Stadt Activity type Higher or Secondary Education Establishments Links Contact the organisation Opens in new window Website Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Other funding € 0,00 Universita' degli Studi di Urbino Carlo Bo Italy Net EU contribution € 400 000,00 Address Via aurelio saffi 2 61029 Urbino See on map Region Centro (IT) Marche Pesaro e Urbino Activity type Higher or Secondary Education Establishments Links Contact the organisation Opens in new window Website Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Other funding € 0,00 THE ATAXIA-TELANGIECTASIA SOCIETY United Kingdom Net EU contribution € 254 000,00 Address Iacr rothamsted AL5 2JQ Harpenden See on map SME The organization defined itself as SME (small and medium-sized enterprise) at the time the Grant Agreement was signed. Yes Region East of England Bedfordshire and Hertfordshire Hertfordshire Activity type Other Links Contact the organisation Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Other funding € 0,00 ABSISKEY France Net EU contribution € 142 500,00 Address 41b rue dupetit thouars 49000 Angers See on map SME The organization defined itself as SME (small and medium-sized enterprise) at the time the Grant Agreement was signed. Yes Region Pays de la Loire Pays de la Loire Maine-et-Loire Activity type Private for-profit entities (excluding Higher or Secondary Education Establishments) Links Contact the organisation Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Other funding € 0,00
