International Study on Long-term Safety and Effectiveness of Palovarotene in Children and Adults With Fibrodysplasia Ossificans Progressiva FOP

What this Trial Is About

This research aims to collect long-term safety and effectiveness data on palovarotene in children and adults with fibrodysplasia ossificans progressiva FOP, a very rare and disabling disease characterized by abnormal bone growth where bone should not form. The study will include participants who are either already receiving palovarotene as prescribed or are not treated with palovarotene. It seeks to assess real-world outcomes, including flare-up episodes, growth, and bone fractures, comparing those treated and untreated with palovarotene. Participants will be observed in two groups those exposed to palovarotene and those unexposed. The study will document safety events related to palovarotene from baseline until 30 days after the last dose. Effectiveness will also be described by monitoring physical function, use of assistive devices, lung function, flare-up characteristics, and pregnancy outcomes. Data collection will occur regularly over an extended period, up to eleven years. During the study, participants will undergo assessments every six months or annually for measures such as joint involvement, physical function questionnaires, lung capacity tests, and flare-up tracking. Pregnancy outcomes and developmental follow-up of babies will be monitored monthly during pregnancy and up to one year after birth. The main safety outcomes include the frequency and seriousness of adverse events related to palovarotene. Overall, participation can last up to eleven years with ongoing monitoring to understand long-term impacts.

A Study to Document and to Further Describe Long-term Safety and Effectiveness of Palovarotene in Participants With Fibrodysplasia Ossificans Progressiva (FOP)