Ipsen (Shanghai) innovation pharmaceuticals Co., Ltd.

Fibrodysplasia ossificans progressiva (FOP) is an extremely rare and severe, disabling genetic disease characterized by the formation of heterotopic ossification (HO) in soft tissues such as muscles, tendons, and ligaments where bone should not be present, often accompanied by painful, recurrent soft tissue swelling (acute attacks). Heterotopic bone formation is progressive and irreversible, and can lead to stiffness of major joints, severely impairing mobility, and causing severe morbidity and progressive disability. The primary objective of this study is to compare the efficacy of two oral doses of IPN60130 monotherapy versus placebo in suppressing new HO volume in adult and pediatric participants with FOP. In addition, an imaging substudy will investigate the utility of [18F]NaF PET-CT for the assessment of HO lesions. Adults and older pediatric participants ≥15 years of age can participate in this substudy.