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. In FOP mice, BLU-782 prevented the formation of injury-induced heterotopic ossification (HO) and tissue swelling (edema). BLU-782 also prevented the formation of HO following surgery in FOP mice. FDA has granted Fast Track Designation to BLU-782 for the treatment of FOP.

About the Clinical Trial

Eligibility Criteria*

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

Study Design*

• STUDY TYPE:  Interventional
• RANDOMIZED STUDY:  No
• PLACEBO CONTROLLED:  No
• LENGTH OF PARTICIPATION: n/a
• NUMBER OF STUDY VISITS: n/a

Status

Phase 1

Phase 1: Studies in humans that assess safety and toxicity of a treatment in a small group of healthy volunteers or patients with the disease of interest.  

, Active

Therapy Approach

Kinase Inhibitor

Study Sponsor

Blueprint Medicines