In-Home Evaluation of Episodic Administration of Palovarotene in Fibrodysplasia Ossificans Progressiva (FOP) Subjects
Status:
Terminated
Trial end date:
2016-08-04
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 (heterotopic ossification or HO) 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 gamma (RARĪ³) agonists
such as palovarotene to prevent HO following injury. This 36-month study will evaluate the
long-term safety and efficacy of episodic treatment with palovarotene for flare-ups in FOP
subjects who successfully complete two flare-up treatment periods (6 weeks duration) and two
follow-up periods (6 weeks duration) in Study PVO-1A-202.