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Saracatinib Trial tO Prevent FOP

Project description

New trial to treat rare disorder

First identified in the 18th century, fibrodysplasia ossificans progressiva (FOP) is a rare inherited connective tissue disorder. Caused by a mutation in the ACVR1 gene and characterised by the progressive heterotopic ossification of fibrous tissue, FOP results in paralysis. Sufferers have an average life expectancy of 40. While there is currently no approved treatment, the EU-funded STOPFOP project will study whether the investigative drug AZD0530, also known as saracatinib, could be used to treat FOP. The study will be performed in three FOP expert centres in Germany, the Netherlands and the United Kingdom. The project’s overall aim is to provide proof of concept that AZD0530 is an effective drug in the treatment of patients with FOP.


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.


Net EU contribution
€ 568 239,00
De boelelaan 1117
1081 HV Amsterdam

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West-Nederland Noord-Holland Groot-Amsterdam
Activity type
Research Organisations
Other funding
€ 0,75

Participants (5)