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. IPN60130 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 IPN60130 for the treatment of FOP.

About the Clinical Trial

The FALKON Phase 2 trial is a global, multi-center, placebo-controlled trial. Approximately 90 patients 5 years of age or older with the R206H ACVR1 mutation or other FOP variants associated with progressive HO will be enrolled. The trial is designed to evaluate the safety of 2 dosing regimens of oral IPN60130 in inhibiting new HO volume compared with placebo (a dummy treatment) in adult and pediatric participants with FOP. 

Eligibility Criteria*

• AGE:  ≥ 15 years of age (The FALKON trial will be expanded to patients ≥ 5 years of age at a later date after safety data has been established)
• DISEASE ACTIVITY:  Participants must have disease progression in the preceding year of the screening visit
• MUTATIONS:  R206H ACVR1 mutation or other FOP variants

Study Design*

• STUDY TYPE:  Interventional
• RANDOMIZED STUDY:  Yes
• PLACEBO CONTROLLED:  Yes, participants will be randomized to placebo or high or low dose IPN60130 only for the first 12 months (part A); then all participants will receive active treatment in the following 12 months ( part B)
• LENGTH OF PARTICIPATION:  26 months
• NUMBER OF STUDY VISITS:  12 visits at the clinical site 

Status

Phase 2, Open, Enrolling

Therapy Approach

Kinase Inhibitor

Study Sponsor

Ipsen

*For the complete list of eligibility criteria and details on this study, visit ClinicalTrials.gov and enter Identifier NCT05039515.

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.