NCT04307953

Saracatinib Trial TO Prevent FOP

• Status: Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 20 (estimated)
• Sponsor: Amsterdam UMC, location VUmc · Academic / Other
• Sex: All
• Age: 18 Years – 65 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Fibrodysplasia Ossificans Progressiva

Interventions

Type	Name	Description
DRUG	AZD0530 Difumarate	AZD0530 for the duration of the trial
DRUG	Matching placebo	Matching placebo during 6 month RCT, AZD0530 thereafter

Timeline

• Start date: 2020-08-05
• Primary completion: 2025-05-06
• Completion: 2025-05-06
• First posted: 2020-03-13
• Last updated: 2024-05-03

Locations

3 sites across 3 countries: Germany, Netherlands, United Kingdom