VISION DMD SIGNED
VISION-DMD - Phase 2 Clinical Trials of VBP15: An Innovative Steroid-like Intervention on Duchenne Muscular Dystrophy
Total Cost €
0EC-Contrib. €
0Partnership
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0VISION DMD project word cloud
Explore the words cloud of the VISION DMD project. It provides you a very rough idea of what is the project "VISION DMD" about.
Project "VISION DMD" data sheet
The following table provides information about the project.
| Coordinator |
UNIVERSITY OF NEWCASTLE UPON TYNE
Organization address contact info |
| Coordinator Country | United Kingdom [UK] |
| Project website | http://www.vision-dmd.info |
| Total cost | 16˙858˙748 € |
| EC max contribution | 6˙000˙000 € (36%) |
| Programme |
1. H2020-EU.3.1.3. (Treating and managing disease) |
| Code Call | H2020-PHC-2015-two-stage |
| Funding Scheme | RIA |
| Starting year | 2016 |
| Duration (year-month-day) | from 2016-01-01 to 2019-12-31 |
Partnership
Take a look of project's partnership.
| # | ||||
|---|---|---|---|---|
| 1 | UNIVERSITY OF NEWCASTLE UPON TYNE | UK (NEWCASTLE UPON TYNE) | coordinator | 2˙918˙331.00 |
| 2 | REVERAGEN BIOPHARMA INC. | US (ROCKVILLE MD) | participant | 1˙769˙834.00 |
| 3 | CERATIUM LIMITED | UK (WEST KIRBY) | participant | 467˙343.00 |
| 4 | STICHTING UNITED PARENT PROJECTS MUSCULAR DYSTROPHY | NL (VEENENDAAL) | participant | 339˙500.00 |
| 5 | ECRIN EUROPEAN CLINICAL RESEARCH INFRASTRUCTURE NETWORK | FR (PARIS) | participant | 336˙365.00 |
| 6 | FAKULTNI NEMOCNICE V MOTOLE | CZ (PRAHA 5) | participant | 168˙625.00 |
| 7 | Children's Research Institute (CRI) | US (Washington, DC) | participant | 0.00 |
| 8 | Reveragen Biopharma Limited | UK (West Kirby) | participant | 0.00 |
Map
Project 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
Deliverables
| Project Website | Websites, patent fillings, videos etc. | 2019-11-20 11:52:47 |
Take a look to the deliverables list in detail: detailed list of VISION DMD deliverables.
Publications
| year | authors and title | journal | last update |
|---|---|---|---|
| 2018 |
Laurie S. Conklin, Jesse M. Damsker, Eric P. Hoffman, William J. Jusko, Panteleimon D. Mavroudis, Benjamin D. Schwartz, Laurel J. Mengle-Gaw, Edward C. Smith, Jean K. Mah, Michela Guglieri, Yoram Nevo, Nancy Kuntz, Craig M. McDonald, Mar Tulinius, Monique M. Ryan, Richard Webster, Diana Castro, Richard S. Finkel, Andrea L. Smith, Lauren P. Morgenroth, Adrienne Arrieta, Maya Shimony, Mark Jaros, Ph Phase IIa trial in Duchenne muscular dystrophy shows vamorolone is a first-in-class dissociative steroidal anti-inflammatory drug published pages: 140-150, ISSN: 1043-6618, DOI: 10.1016/j.phrs.2018.09.007 |
Pharmacological Research 136 | 2019-12-16 |
| 2019 |
Eric P. Hoffman, Benjamin D. Schwartz, Laurel J. Mengle-Gaw, Edward C. Smith, Diana Castro, Jean K. Mah, Craig M. McDonald, Nancy L. Kuntz, Richard S. Finkel, Michela Guglieri, Katharine Bushby, Mar Tulinius, Yoram Nevo, Monique M. Ryan, Richard Webster, Andrea L. Smith, Lauren P. Morgenroth, Adrienne Arrieta, Maya Shimony, Catherine Siener, Mark Jaros, Phil Shale, John M. McCall, Kanneboyina Naga Vamorolone trial in Duchenne muscular dystrophy shows dose-related improvement of muscle function published pages: 10.1212/WNL.0000, ISSN: 0028-3878, DOI: 10.1212/wnl.0000000000008168 |
Neurology | 2019-11-20 |
| 2017 |
M. Guglieri, P. Clemens, A. Cnaan, J. Damsker, A. Arrieta, L. Morgenroth, R. Davis, C. Olsen, R. Head, K. Nagaraju, Y. Hathout, J. Haberlova, D. Athanasiou, E. Vroom, K. Bushby, E. Hoffman Vision DMD: Vamorolone (VBP15) drug development program for Duchenne muscular dystrophy published pages: e238, ISSN: 1090-3798, DOI: 10.1016/j.ejpn.2017.04.1270 |
European Journal of Paediatric Neurology 21 | 2019-11-20 |
| 2018 |
MUDr. Jana Haberlová, Ph.D. New therapies in neuromuscular disorders in childhoodNové možnosti léÄby vrozenýchneuromuskulárnÃch onemocnÄ›nà v dÄ›tském vÄ›ku published pages: 2018; 19(2): 108, ISSN: 1213-1814, DOI: |
Neurology for Practice bimonthly | 2019-11-20 |
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