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.
Phase:
Phase 2
Details
Lead Sponsor:
VU University Medical Center
Collaborators:
AstraZeneca Brigham and Women's Hospital Innovative Medicines Initiative Klinikum Garmisch-Patenkirchen Royal National Orthopaedic Hospital NHS Trust University of Oxford