STOPFOP

Saracatinib trial to prevent FOP
STOPFOP logo

FACTS & FIGURES

Start Date
End Date
Call
IMI2 - Call 13
Grant agreement number
821600

Type of Action: 
RIA (Research and Innovation Action)

Contributions
IMI Funding
999 710
EFPIA in kind
1 000 000
Other
2
Total Cost
1 999 712

Summary

Fibrodysplasia ossificans progressive (FOP) is a rare disease in which the muscles and connective tissues (e.g. tendons and ligaments) slowly turn into bone. There is no treatment; as the disease progresses, the build-up of bone material around the joints gradually limits patients’ mobility, and can also result in difficulties eating, speaking and even breathing.

The aim of STOPFOP is to see if a drug called AZD0530 could be used to treat FOP. FOP is caused by a mutation in a gene that codes for a protein called ALK2 kinase. Studies in the lab have shown that AZD0530 blocks the action of ALK2 kinase, and in mice, the drug successfully stopped the formation of bone material in the soft tissues and kept the mice’s limbs moving.

AZD0530, also known as saracatinib, has already been tested for safety in humans in healthy volunteers and as a treatment for certain cancers. Now, the STOPFOP team plans to run a clinical trial of AZD0530 in 16 adults with FOP to see if it reduces the formation of new bone. They will work with patients, FOP experts and regulatory authorities throughout the project.

Participants Show participants on map

EFPIA companies
  • Astrazeneca AB, Södertälje, Sweden
Universities, research organisations, public bodies, non-profit groups
  • Klinikum Garmisch-Partenkirchen GMBH, Garmisch-Partenkirchen, Germany
  • Stichting Vumc, Amsterdam, Netherlands
  • The Brigham And Women'S Hospital Inc, Boston, United States
  • The Royal National Orthopaedic Hospital National Health Service Trust, Stanmore, United Kingdom
  • University of Oxford, Oxford, United Kingdom

CONTACT

Project coordinator
Marelise EEKHOFF
STICHTING VUMC
emw.eekhoff[at]vumc.nl