FOP is caused by a mutation in the ACVR1 gene that results in excessive bone morphogenetic protein (BMP) signaling, which regulates cartilage and bone development. The FOP mutation increases BMP signaling, resulting in the formation of heterotopic bone. BLU-782 is an oral investigational drug designed to selectively target the mutant FOP receptor (ACVR1/ALK2), the underlying cause of FOP. FDA has granted Fast Track Designation to BLU-782 for the treatment of FOP.

About the Clinical Trial

Ipsen conducted a

trial with BLU-782 in healthy volunteers (e.g. people without FOP) to establish the safety of the investigational drug.

Eligibility Criteria*

• AGE: ≥ 18
• DISEASE ACTIVITY: Healthy volunteers only
• MUTATIONS: n/a

Study Design*

• STUDY TYPE:  Interventional
• RANDOMIZED STUDY:  Yes
• PLACEBO CONTROLLED:  Yes
• LENGTH OF PARTICIPATION: n/a
• NUMBER OF STUDY VISITS: n/a

Status

Completed

Therapy Approach

Kinase Inhibitor

Study Sponsor

Ipsen

 

The IFOPA does not endorse nor recommend specific clinical trials. Please speak with your doctor if you are interested in participating in a clinical trial.