BLU-782

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

Blueprint Medicines is currently conducting a
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.  
trial with BLU-782 in healthy volunteers (e.g. people without FOP) to establish the safety of the investigational drug.

Eligibility Criteria Icon
Eligibility Criteria*
  • AGE: ≥ 18
  • DISEASE ACTIVITY: Healthy volunteers only
  • MUTATIONS: n/a
Study Design Icon
Study Design*
  • STUDY TYPE:  Interventional
  • RANDOMIZED STUDY:  Yes
  • PLACEBO CONTROLLED:  Yes
  • LENGTH OF PARTICIPATION: n/a
  • NUMBER OF STUDY VISITS: n/a
Status Icon
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 Icon
Therapy Approach

Kinase Inhibitor

Study Sponsor Icon
Study Sponsor

Blueprint Medicines

Make a Difference

Donate Connect Fundraise

Already a Member? Sign In

Sign Up for FOP Connection, Our Monthly eNewsletter