An Efficacy and Safety Study of Palovarotene to Treat Preosseous Flare-ups in FOP Subjects
Status:
Completed
Trial end date:
2016-05-23
Target enrollment:
Participant gender:
Summary
Fibrodysplasia ossificans progressiva (FOP) is a rare, severely disabling disease
characterized by painful, recurrent episodes of soft tissue swelling (flare-ups) that result
in abnormal bone formation in muscles, tendons, and ligaments. Flare-ups begin early in life
and may occur spontaneously or after soft tissue trauma, vaccinations, or influenza
infections. Recurrent flare-ups progressively restrict movement by locking joints leading to
cumulative loss of function and disability. Mouse models of FOP have demonstrated the ability
of retinoic acid receptor (RAR) gamma agonists to prevent heterotopic ossification (HO)
following injury. The purpose of the study is to evaluate whether palovarotene, an RAR gamma
agonist, will prevent HO during and following a flare-up in subjects with FOP.