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VISION DMD SIGNED

VISION-DMD - Phase 2 Clinical Trials of VBP15: An Innovative Steroid-like Intervention on Duchenne Muscular Dystrophy

Total Cost €

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EC-Contrib. €

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Partnership

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Outcomes and
results

VISION 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.

line grants designed steroid progressively death fda therapy preclinical severe adulthood progression combination exposure ambulation acceptance care recognised window global serum standard cumulative doses networks young stratified nmd tolerability affordable 2100 effect cinrg strength endpoint techniques eric data orphan regulatory potentially ambulant revolutionise government muscle pharmacodynamics biomarkers incurable vbp15 life delay occurs trial patient duchenne disease undertakings pathology ema 2020 advice unmet safety proposes directed boys efficacy treatment weaken time corticosteroids retain stand ascending links quality lose followed therapies irdirc stabilization rare extension muscular vision exploratory dystrophy drug building mri lifespan cellular positive cs 2b goals membrane collection dmd treat months primary ecrin groups clinical us innovative 2a registration slow therapeutic patients international wasting meet

Project "VISION DMD" data sheet

The following table provides information about the project.

Coordinator	UNIVERSITY OF NEWCASTLE UPON TYNE Organization address address: KINGS GATE city: NEWCASTLE UPON TYNE postcode: NE1 7RU website: http://www.ncl.ac.uk/ contact info title: n.a. name: n.a. surname: n.a. function: n.a. email: n.a. telephone: n.a. fax: n.a.
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.

#	participants	country	role	EC contrib. [€]
1	UNIVERSITY OF NEWCASTLE UPON TYNE UNIVERSITY OF NEWCASTLE UPON TYNE Organization address address: KINGS GATE city: NEWCASTLE UPON TYNE postcode: NE1 7RU website: http://www.ncl.ac.uk/ contact info title: n.a. name: n.a. surname: n.a. function: n.a. email: n.a. telephone: n.a. fax: n.a.	UK (NEWCASTLE UPON TYNE)	coordinator	2˙918˙331.00
2	REVERAGEN BIOPHARMA INC. REVERAGEN BIOPHARMA INC. Organization address address: 155 GIBBS STREET SUITE 433 city: ROCKVILLE MD postcode: 20850 website: n.a. contact info title: n.a. name: n.a. surname: n.a. function: n.a. email: n.a. telephone: n.a. fax: n.a.	US (ROCKVILLE MD)	participant	1˙769˙834.00
3	CERATIUM LIMITED CERATIUM LIMITED Organization address address: THE HAVEN 20 BURLINGHAM AVENUE city: WEST KIRBY postcode: CH48 8AP website: www.ceratium.eu contact info title: n.a. name: n.a. surname: n.a. function: n.a. email: n.a. telephone: n.a. fax: n.a.	UK (WEST KIRBY)	participant	467˙343.00
4	STICHTING UNITED PARENT PROJECTS MUSCULAR DYSTROPHY STICHTING UNITED PARENT PROJECTS MUSCULAR DYSTROPHY Organization address address: KONINGINNELAAN 69 city: VEENENDAAL postcode: 3905 GG website: n.a. contact info title: n.a. name: n.a. surname: n.a. function: n.a. email: n.a. telephone: n.a. fax: n.a.	NL (VEENENDAAL)	participant	339˙500.00
5	ECRIN EUROPEAN CLINICAL RESEARCH INFRASTRUCTURE NETWORK ECRIN EUROPEAN CLINICAL RESEARCH INFRASTRUCTURE NETWORK Organization address address: 5 RUE WATT city: PARIS postcode: 75013 website: www.ecrin.org contact info title: n.a. name: n.a. surname: n.a. function: n.a. email: n.a. telephone: n.a. fax: n.a.	FR (PARIS)	participant	336˙365.00
6	FAKULTNI NEMOCNICE V MOTOLE FAKULTNI NEMOCNICE V MOTOLE Organization address address: V UVALU 84 city: PRAHA 5 postcode: 150 06 website: www.fnmotol.cz contact info title: n.a. name: n.a. surname: n.a. function: n.a. email: n.a. telephone: n.a. fax: n.a.	CZ (PRAHA 5)	participant	168˙625.00
7	Children's Research Institute (CRI) Children's Research Institute (CRI) Organization address address: Michigan Avenue 111 city: Washington, DC postcode: 20010 website: n.a. contact info title: n.a. name: n.a. surname: n.a. function: n.a. email: n.a. telephone: n.a. fax: n.a.	US (Washington, DC)	participant	0.00
8	Reveragen Biopharma Limited Reveragen Biopharma Limited Organization address address: The Oaks, 3 Village Road city: West Kirby postcode: CH48 3JN website: n.a. contact info title: n.a. name: n.a. surname: n.a. function: n.a. email: n.a. telephone: n.a. fax: n.a.	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

List of 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

List of 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 HaberlovÃ¡, Ph.D. New therapies in neuromuscular disorders in childhoodNovÃ© moÅ¾nosti lÃ©Äby vrozenÃ½chneuromuskulÃ¡rnÃch onemocnÄ›nÃ v dÄ›tskÃ©m vÄ›ku published pages: 2018; 19(2): 108, ISSN: 1213-1814, DOI:	Neurology for Practice bimonthly	2019-11-20

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