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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.

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

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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