The aim of the STOPFOP trial is to see if the investigative drug AZD0530, also known as saracatinib, could be used to treat FOP. In people with FOP, a mutation occurs in the gene ACVR1 that encodes for the protein kinase ALK2. As a result, ALK2 becomes overactive causing muscles and connective tissues (e.g. tendons) to slowly turn into bone, which severely limits mobility and even breathing. There is currently no approved treatment for patients with FOP. Scientific research by the STOPFOP team has shown that AZD0530 blocks the activity of the pathogenic ALK2 kinase. The team has demonstrated in FOP-mice that the drug was effective in preventing ectopic bone formation which led to preserved mobility of joints.
AZD0530 is an investigative drug that has been developed by AstraZeneca initially as a potential treatment for patients with cancer. It has been tested for safety and efficacy in humans with cancer and healthy volunteers. Over 600 people have used the drug, which means researchers are familiar with the effects and safety profile of the drug. The drug is taken orally, as a once-daily dose.
This study is supported by a grant from the European Commission Innovative Medicine Initiative. The STOPFOP team will aim to recruit 20 adults with the classical FOP genetic mutation into a clinical trial to test the safety and efficacy of AZD0530. Each patient’s participation in the study will last 1.5 years, with a 6-month Randomized Placebo-controlled Trial (RCT) period followed by a 12-month open label extended treatment period. This ensures that all participants will receive the drug during the trial. Assessment measures will include CT scan, PET-scan and Patient Reported Outcome Measures (PROMs).
The STOPFOP team includes the academic researchers Dr Alex Bullock (University of Oxford) and Dr Paul Yu (Harvard University), the clinicians Dr Marelise Eekhoff (Coordinating Principal Investigator, Amsterdam UMC, The Netherlands), Dr Richard Keen (Royal National Orthopaedic Hospital, United Kingdom) and Dr Clemens Stockklausner (Klinikum Garmisch-Partenkirchen, Germany), as well as AstraZeneca. The researchers will work with patients, patient organizations and FOP experts throughout the trial. Dr Alex Bullock comments, “We would like to extend our sincere thanks to all the FOP foundations and families whose donations have supported this research to make this trial possible.”
“Our hope is that AZD0530, if effective, might make an important contribution to the treatment of FOP patients,” said Bernard Smilde, Associate Investigator of the STOPFOP trial.
Patients or caregivers who wish to learn more about this trial, or to contact the researchers, can find more information on the website www.stopfop.com.
This project has received funding from the Innovative Medicines Initiative 2 Joint Undertaking (JU) under grant agreement No 821600. The JU receives support from the European Union’s Horizon 2020 research and innovation programme and EFPIA.