Objective VISION-DMD aims to advance clinical development of the orphan drug VBP15 as a new therapy to revolutionise care for all patients with Duchenne muscular dystrophy (DMD) by 2020, in line with IRDiRC goals. DMD is an incurable, rare muscle wasting disease; boys progressively weaken, lose ambulation and death occurs by early adulthood. Corticosteroids (CS) are widely recognised to increase muscle strength and delay disease progression but global acceptance as standard of care is very variable due to severe side effects. VBP15 is an innovative steroid-like drug designed to retain or better CS efficacy and improve membrane stabilization with reduced or no side effects. VBP15 will increase the therapeutic window to slow disease progression and improve quality of life and lifespan for all DMD patients. Building on positive preclinical and Phase 1 results funded by government grants and international patient groups and based on FDA and EMA advice, VISION-DMD proposes a Phase 2 registration directed clinical programme aimed at an affordable therapy: Phase 2a will study the safety and tolerability of ascending doses of VBP15 in ambulant DMD boys; Phase 2b will demonstrate the efficacy and safety of two doses of VBP15 in young ambulant DMD boys. Both studies will be followed by extension studies for long term safety and efficacy data collection leading to cumulative exposure of up to 2100 drug months. The project proposes the Time to Stand Test as a highly relevant and reliable primary endpoint. Innovative exploratory serum biomarkers and novel wide scale MRI techniques will be used to investigate the VBP15 pharmacodynamics and the effect on muscle cellular pathology. VBP15 will meet the unmet need for better treatment for DMD with widespread acceptance and potentially be used in combination with stratified therapies as they are developed. The Consortium links the leading networks TREAT-NMD and CINRG with ECRIN-ERIC, for trial delivery and regulatory undertakings in Europe/US Fields of science medical and health sciencesbasic medicineneurologymuscular dystrophiesduchenne muscular dystrophy 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 UNIVERSITY OF NEWCASTLE UPON TYNE Net EU contribution € 2 918 331,29 Address Kings gate NE1 7RU Newcastle upon tyne United Kingdom See on map Region North East (England) Northumberland and Tyne and Wear Tyneside 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 Participants (9) Sort alphabetically Sort by Net EU contribution Expand all Collapse all Reveragen Biopharma Limited United Kingdom Net EU contribution € 0,00 Address The oaks, 3 village road CH48 3JN West kirby 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 North West (England) Merseyside Wirral 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 STICHTING UNITED PARENT PROJECTS MUSCULAR DYSTROPHY Netherlands Net EU contribution € 339 500,00 Address Koninginnelaan 69 3905 GG Veenendaal See on map Region West-Nederland Utrecht Utrecht 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 ECRIN EUROPEAN CLINICAL RESEARCH INFRASTRUCTURE NETWORK Participation ended France Net EU contribution € 336 365,87 Address 30 bd saint-jacques batiment b 75014 Paris See on map Region Ile-de-France Ile-de-France Paris 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 Third-party Legal entity other than a subcontractor which is affiliated or legally linked to a participant. The entity carries out work under the conditions laid down in the Grant Agreement, supplies goods or provides services for the action, but did not sign the Grant Agreement. A third party abides by the rules applicable to its related participant under the Grant Agreement with regard to eligibility of costs and control of expenditure. LUXEMBOURG INSTITUTE OF HEALTH Participation ended Luxembourg Net EU contribution € 0,00 Address 1a rue rue thomas edison 1445 Strassen See on map Region Luxembourg Luxembourg Luxembourg 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 Third-party Legal entity other than a subcontractor which is affiliated or legally linked to a participant. The entity carries out work under the conditions laid down in the Grant Agreement, supplies goods or provides services for the action, but did not sign the Grant Agreement. A third party abides by the rules applicable to its related participant under the Grant Agreement with regard to eligibility of costs and control of expenditure. Masarykova univerzita Participation ended Czechia Net EU contribution € 0,00 Address Zerotinovo namesti 9 601 77 Brno See on map Region Česko Jihovýchod Jihomoravský kraj 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 CERATIUM LIMITED United Kingdom Net EU contribution € 467 343,75 Address The haven 20 burlingham avenue CH48 8AP West kirby 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 North West (England) Merseyside Wirral Activity type Private for-profit entities (excluding 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 FAKULTNI NEMOCNICE V MOTOLE Czechia Net EU contribution € 168 625,00 Address V uvalu 84 150 06 Praha 5 See on map Region Česko Praha Hlavní město Praha 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 Children's Research Institute (CRI) Participation ended United States Net EU contribution € 0,00 Address Michigan avenue 111 20010 Washington, dc See on map Activity type Research Organisations 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 REVERAGEN BIOPHARMA INC. United States Net EU contribution € 1 769 834,09 Address 155 gibbs street suite 433 20850 Rockville md See on map SME The organization defined itself as SME (small and medium-sized enterprise) at the time the Grant Agreement was signed. Yes 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 € 10 858 748,72
