STOPFOP
Research type
Research Study
Full title
Saracatinib trial TO Prevent FOP
IRAS ID
1006232
Contact name
EMW Eekhoff
Contact email
Sponsor organisation
VU University Medical Center
Eudract number
2019-003324-20
Clinicaltrials.gov Identifier
Research summary
Fibrodysplasia Ossificans Progressiva (FOP) is a genetic, chronic and devastating disease characterised by the progressive growth of bone (Heterotopic Ossification, HO) of skeletal muscle, ligaments, tendons and fascia which leads to joint immobility, pain and premature death. The disease has a prevalence of approximately 1 in 1.5 million people. The formation of HO begins in early childhood and progresses during periods of disease ‘flare-up’. There are currently no licensed treatments available that have been shown to successfully prevent the formation of HO in FOP patients.
The drug AZD0530 (Saracatinib) has been found to prevent heterotopic bone formation in pre-clinical studies and as a re-purposed drug, Saracatinib has the advantage of extensive safety data from 28 registered clinical trials involving over 600 participants.
This study, sponsored by VUmc, aims to assess the safety and efficacy of daily oral Saracatinib in the management of FOP. The study is designed as a multi-centre 6-month double blind randomised controlled trial (RCT) of Saracatinib versus placebo, followed by a 12 month period comparing open-label Saracatinib with control data from the Clementia Natural History Study in addition to placebo data from the double blind phase. 20 adult participants will be recruited across the sites. The primary objective of the study is to measure the efficacy of Saracatinib on HO formation measured by low-dose whole body computed tomography (CT). The secondary objectives of the study are to measure safety and tolerability of Saracatinib (Adverse event recording, Electrocardiogram, blood tests) and further assess efficacy (imaging, functional assessment, patient reported outcomes).
REC name
North West - Haydock Research Ethics Committee
REC reference
22/NW/0305
Date of REC Opinion
8 Sep 2023
REC opinion
Further Information Favourable Opinion