Opendata, web and dolomites

VISION DMD SIGNED

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

Total Cost €

0

EC-Contrib. €

0

Partnership

0

Views

0

 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.

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

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 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

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

year authors and title journal last update
List of publications.
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 léčby vrozenýchneuromuskulárních onemocnění v dětském věku
published pages: 2018; 19(2): 108, ISSN: 1213-1814, DOI:
Neurology for Practice bimonthly 2019-11-20

Are you the coordinator (or a participant) of this project? Plaese send me more information about the "VISION DMD" project.

For instance: the website url (it has not provided by EU-opendata yet), the logo, a more detailed description of the project (in plain text as a rtf file or a word file), some pictures (as picture files, not embedded into any word file), twitter account, linkedin page, etc.

Send me an  email (fabio@fabiodisconzi.com) and I put them in your project's page as son as possible.

Thanks. And then put a link of this page into your project's website.

The information about "VISION DMD" are provided by the European Opendata Portal: CORDIS opendata.

More projects from the same programme (H2020-EU.3.1.3.)

GACD (2019)

Global Alliance for Chronic Diseases Secretariat

Read More  

ISLET (2020)

Advancing Innovative Stem Cell-based Therapy for Diabetes in Europe

Read More  

SBR (2020)

SMART BONE REGENERATION

Read More