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.

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

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.)

PanCareFollowUp (2019)

PanCareFollowUp: Novel, patient-centred survivorship care to improve care quality, effectiveness, cost-effectiveness and accessibility for survivors and caregivers

Read More  

INTE-AFRICA (2019)

Integrating and decentralising diabetes and hypertension services in Africa

Read More  

LEGACy (2019)

CeLac and European consortium for a personalized medicine approach to Gastric Cancer

Read More