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 aPhase 1
trial with BLU-782 in healthy volunteers (e.g. people without FOP) to establish the safety of the investigational drug.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.
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
Phase 1
, ActivePhase 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.
Therapy Approach
Kinase Inhibitor
Study Sponsor
Blueprint MedicinesThe IFOPA does not endorse nor recommend specific clinical trials. Please speak with your doctor if you are interested in participating in a clinical trial.
