An Open-Label Extension Study of Palovarotene to Prevent Heterotopic Ossification in People With Fibrodysplasia Ossificans Progressiva (FOP) in France

Fibrodysplasia Ossificans Progressiva Clinical Trial

Official title: A Phase 2, Open-Label Extension, Efficacy and Safety Study of a Retinoic Acid Receptor Gamma (RARγ)-Specific Agonist (Palovarotene) in the Treatment of Preosseous Flare-ups in Subjects With Fibrodysplasia Ossificans Progressiva (FOP)

Status Completed

Clinical Trial Summary

Fibrodysplasia Ossificans Progressiva (FOP) is a rare, severely disabling disease characterized by heterotopic ossification (HO), i.e., abnormal bone formation, often associated with painful, recurrent episodes of soft tissue swelling (flare-ups). Lesions begin in early childhood and lead to progressive ankyloses of major joints with resultant loss of movement. In this study, the ability of different palovarotene dosing regimens to prevent the formation of new HO will be evaluated in adult and pediatric participants with FOP in France.

Clinical Trial Description

The main objective of this Phase 2, open-label study is to evaluate the safety and efficacy of different palovarotene dosing regimens in participants with FOP in France. Efficacy will be assessed based on the ability of palovarotene to prevent the formation of new heterotopic ossification (HO) as assessed by low-dose whole body computed tomography (WBCT) scan, excluding head. Participants who successfully completed study PVO-1A-201 as well as up to two new adult participants were followed for up to 24 months. Participants under Amendment 1 will be followed for up to an additional 48 months. No new participants will be enrolled. The Adult Cohort will include all participants with at least 90% skeletal maturity, regardless of age. The Pediatric Cohort will include all participants with less than 90% skeletal maturity. Any Pediatric Cohort participants who achieves ≥90% skeletal maturity during the study can be considered for enrollment into the Adult Cohort at the discretion of then Investigator. Participants that were skeletally immature at their last assessment will be invited back to participate in a safety follow-up (Part D) that includes yearly visits for up to a 2-year follow-up period following last dose. No dosing will occur in Part D. ;

Related Conditions & MeSH terms

• Fibrodysplasia Ossificans Progressiva

• NCT number: NCT02979769
• Study type: Interventional
• Source: Ipsen
• Status: Completed
• Phase: Phase 2
• Start date: November 28, 2016
• Completion date: June 28, 2022