Saracatinib Trial TO Prevent FOP

Summary

This is a phase 2 study, designed as a European multicentre 6-month double blind random-ized controlled trial (RCT) of AZD0530 versus matched placebo, followed by a 12 month trial comparing open-label extended AZD0530 treatment with historical control data. Study population: Male and female adult patients aged 18 years and older with a diagnosis of FOP who meet the inclusion (active disease) and exclusion criteria will be eligible for participation in this study. The total number of enrolled patients will be 20. Intervention: Patients will be randomized to receive either AZD0530 100mg once daily or matched placebo, taken orally for the first 6 months, immediately followed by an open-label extension in which all patients will receive AZD0530 100mg once daily oral dose for a further 12 months. Endpoints: Endpoints include objective change in heterotopic bone volume measured by low-dose whole-body computer tomography (CT) , \[18F\] NaF Positron Emission Tomography (PET) activity and patient reported outcome measures.

Timeline

Start

2020-08-05

Primary completion

2025-05-06

Completion

2025-05-06

Publications

• Smilde BJ, Stockklausner C, Keen R, Whittaker A, Bullock AN, von Delft A, van Schoor NM, Yu PB, Eekhoff EMW. Protocol paper: a multi-center, double-blinded, randomized, 6-month, placebo-controlled study followed by 12-month open label extension to evaluate the safety and efficacy of Saracatinib in Fibrodysplasia Ossificans Progressiva (STOPFOP). BMC Musculoskelet Disord. 2022 Jun 1;23(1):519. doi: 10.1186/s12891-022-05471-x.

Drugs

Indications