Clementia Natural History of FOP Study
This is a worldwide study sponsored by Clementia Pharmaceuticals, and includes a site at the Princess Alexandra Hospital (PAH) and Lady Cilento Children’s Hospital (LCCH) in Brisbane, with Professor Matthew Brown. Professor Brown is a doctor who was part of the research team involved in the discovery of the FOP gene and is now based at The Queensland University of Technology Diamantina Institute (at PAH).This is the first time there has been research on FOP conducted within Australia, marking a significant milestone for the management of FOP in Australia.
The aim of the study is to develop methods for measuring progression of FOP over time so that when in future clinical trials are performed these methods can be used to test whether trial medications slow down the disease. It is non-interventional and simply follows people with FOP for 3 years and does not involve taking any medications. Travel costs for the participants and caregivers are covered by Clementia.
The study is looking for FOP patient involvement so if you would like more information or have any questions, please contact the study coordinator, Linda Bradbury on 07 3443 7078 or via email.
Clementia Phase II Palovarotene trial
In 2014, Clementia Pharmaceuticals Inc announced clinical trials would begin in the US and France evaluating the effect of different doses of palovarotene on new bone formation during and after a flare-up in patients with FOP. In preclinical trials, palovarotene prevented heterotopic bone formation in mouse models of FOP. The Phase 2 clinical trial was designed to determine whether these effects can be replicated in patients with FOP. Patients were unable to take part in Part A within Australia and needed to travel to the established sites overseas. Part B of the Phase 2 trial was designed to assess the effects over 24 months. Sites included the Queensland University of Technology in Australia. For further information on inclusion see clinicaltrials.gov (trial NCT02279095).
Clementia have kindly agreed to share their presentation here from the Australasian FOP Patient Gathering in Brisbane in June 2016 regarding the Phase 2 palovarotene trial and their Patient Narrative Project. For more information about the Clementia studies please visit the Clementia Pharmaceuticals website.
Novartis Study of FOP Related Biomarkers
Novartis Pharmaceuticals has begun a study collecting urine samples from FOP patients for the assessment of biomarkers related to the disease, disease progression and for prediction of flare-ups of the disease. Disease related biomarkers in these patients are currently unknown. This study aims to support the development of novel therapies for this disease.