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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 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.	NL (AMSTERDAM)	coordinator	525˙756.00
2	THE ROYAL NATIONAL ORTHOPAEDIC HOSPITAL NATIONAL HEALTH SERVICE TRUST THE ROYAL NATIONAL ORTHOPAEDIC HOSPITAL NATIONAL HEALTH SERVICE TRUST Organization address address: BROCKLEY HILL city: STANMORE postcode: HA7 4LP 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 (STANMORE)	participant	253˙559.00
3	KLINIKUM GARMISCH-PARTENKIRCHEN GMBH KLINIKUM GARMISCH-PARTENKIRCHEN GMBH Organization address address: AUENSTRASSE 6 city: GARMISCH PARTENKIRCHEN postcode: 82467 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.	DE (GARMISCH PARTENKIRCHEN)	participant	216˙645.00
4	THE BRIGHAM AND WOMEN'S HOSPITAL INC THE BRIGHAM AND WOMEN'S HOSPITAL INC Organization address address: FRANCIS STREET 75 city: BOSTON MA postcode: 2241 website: www.brighamandwomens.org contact info title: n.a. name: n.a. surname: n.a. function: n.a. email: n.a. telephone: n.a. fax: n.a.	US (BOSTON MA)	participant	1˙875.00
5	THE CHANCELLOR, MASTERS AND SCHOLARS OF THE UNIVERSITY OF OXFORD THE CHANCELLOR, MASTERS AND SCHOLARS OF THE UNIVERSITY OF OXFORD Organization address address: WELLINGTON SQUARE UNIVERSITY OFFICES city: OXFORD postcode: OX1 2JD website: www.ox.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 (OXFORD)	participant	1˙875.00
6	ASTRAZENECA AB ASTRAZENECA AB Organization address address: Vastra Maelarhamnen city: SODERTAELJE postcode: 151 85 website: www.astrazeneca.com contact info title: n.a. name: n.a. surname: n.a. function: n.a. email: n.a. telephone: n.a. fax: n.a.	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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