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STOPFOP SIGNED

Saracatinib Trial tO Prevent FOP

Total Cost €

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EC-Contrib. €

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Partnership

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 STOPFOP project word cloud

Explore the words cloud of the STOPFOP project. It provides you a very rough idea of what is the project "STOPFOP" about.

previously    adults    activin    therapies    exist    patient    tissues    clinical    receptor    astrazeneca    protein    involvement    repurposing    inhibitor    2a    regulation    extension    saracatinib    rarity    ideal    kinase    formed    treatment    assets    proof    fibrodysplasia    preserving    teams    disabling    universities    data    centers    once    12    reduce    movement    approved    death    life    neofunction    amsterdam    pricing    soft    rare    boards    oxford    garmen    syndrome    harvard    activating    ensured    stimulation    expert    50    morphogenetic    azd0530    disease    patients    limb    solution    investments    uk    risking    active    drug    historical    showed    alk2    preclinical    placebo    performed    safety    de    efficacy    stakeholder    congenital    bone    roadmap    germany    regulatory    unexplored    16    ectopic    mutations    r206h    partenkirchen    progressiva    affordable    5nm    engagement    disability    hypothesis    ossificans    dsm    mice    advisory    people    expectations    shortening    blocked    acvr1    contractures    fop    label    potent    indication    progressive    mediated    randomized    netherlands    establishing    agencies    option    london   

Project "STOPFOP" data sheet

The following table provides information about the project.

Coordinator
STICHTING VUMC 

Organization address
address: DE BOELELAAN 1117
city: AMSTERDAM
postcode: 1081 HV
website: www.vumc.nl

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 Netherlands [NL]
 Total cost 1˙999˙712 €
 EC max contribution 999˙710 € (50%)
 Programme 1. H2020-EU.3.1.7. (Innovative Medicines Initiative 2 (IMI2))
 Code Call H2020-JTI-IMI2-2017-13-two-stage
 Funding Scheme IMI2-RIA
 Starting year 2019
 Duration (year-month-day) from 2019-05-01   to  2022-04-30

 Partnership

Take a look of project's partnership.

# participants  country  role  EC contrib. [€] 
1    STICHTING VUMC NL (AMSTERDAM) coordinator 525˙756.00
2    THE ROYAL NATIONAL ORTHOPAEDIC HOSPITAL NATIONAL HEALTH SERVICE TRUST UK (STANMORE) participant 253˙559.00
3    KLINIKUM GARMISCH-PARTENKIRCHEN GMBH DE (GARMISCH PARTENKIRCHEN) participant 216˙645.00
4    THE BRIGHAM AND WOMEN'S HOSPITAL INC US (BOSTON MA) participant 1˙875.00
5    THE CHANCELLOR, MASTERS AND SCHOLARS OF THE UNIVERSITY OF OXFORD UK (OXFORD) participant 1˙875.00
6    ASTRAZENECA AB SE (SODERTAELJE) participant 0.00

Map

 Project objective

Fibrodysplasia ossificans progressiva (FOP) is a rare, disabling and life-shortening congenital syndrome for which no effective therapies exist. Repurposing of AZD0530 (saracatinib, AstraZeneca) would be an ideal solution for de-risking early clinical studies. Using existing assets and investments, this may allow more affordable pricing once an indication is approved. Ectopic bone is formed in soft tissues due to activating mutations in the bone morphogenetic protein receptor kinase ALK2/ACVR1, leading to progressive contractures and early death. Preclinical studies showed AZD0530, previously unexplored in FOP, to be a potent (5nM) inhibitor of ALK2 kinase and ALK2-R206H-mediated neofunction after activin stimulation. In mice, AZD0530 blocked ectopic bone formation preserving limb movement.

Hypothesis: AZD0530 will reduce ectopic bone formation and progressive disability in people with FOP.

AIM: to provide proof of concept that AZD0530 is an effective drug in the treatment of patients with FOP.

Methods: Based on the rarity of the disease and expected drug efficacy (50% reduction in new bone), a phase 2A proof of concept study including a 6 month randomized placebo controlled study and 12 month open label extension study using historical data, is proposed including 16 adults with active FOP disease. The study will be performed in three European FOP expert Centers (Amsterdam The Netherlands – Lead, London UK, and Garmen Partenkirchen Germany). The study will be performed in collaboration with the expert preclinical teams at the Universities of Oxford and Harvard. FOP expert and patient engagement as well as safety will be ensured by establishing advisory, DSM and stakeholder boards. Early involvement of the regulatory agencies are planned.

Expectations: we will develop a roadmap for further studies and regulation of this new treatment option in FOP based on the results.

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

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