Clinical Trial: An Open-Label Extension Study of Palovarotene to Prevent Heterotopic Ossification in FOP Subjects in France

Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional




Official Title: A Phase 2, Open-Label, Efficacy and Safety Study of an RARγ Specific Agonist (Palovarotene) to Prevent Heterotopic Ossification in Subjects With Fibrodysplasia Ossificans Pr

Brief 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 (RARγ) agonists such as palovarotene (PVO) to prevent HO following injury. This 24-month study will (1) continue to follow FOP subjects from France who completed Clementia Study PVO-1A-201; (2) enroll up to eight additional new French subjects who have achieved at least 90% skeletal maturity; and (3) evaluate the ability of different palovarotene dosing regimens to prevent HO in these subjects.