STOPFOP study update – November 2023

From Dr Marelise Eekhoff

The STOPFOP study investigates the safety and efficacy of oral saracatinib, an ACVR1 kinase inhibitor. Despite the significant challenges it encountered at inception from COVID and Brexit, the study is now well on track.

A total of 17 patients are actively participating through the Dutch site (Amsterdam UMC) and the German site (Klinikum Garmisch-Partenkirchen), and while the UK site in London (RNOH) still awaits approval, a number of UK patients have been included through the Amsterdam site for the time being. With the addition of these UK patients, the trial now involves participants from eight different European countries. In addition, with growing pre-clinical data on the potential of saracatinib, variant mutations could also be included after an amendment to the initial protocol was approved.

Both the investigators and the participants are excited about the trial’s progress. There have been no serious drug related adverse events to date and all participants who finished the trial chose to continue saracatinib through a prolonged extension phase of the trial. The STOPFOP team receives great support from the FOP associations and have also noticed increasing interest from people outside the community.

The team hopes to expand the scope of saracatinib to research in children, should their first data, that is expected in 2024, be promising.