Fibrodysplasia Ossificans Progressiva (FOP) is a rare, severely disabling disease characterized by heterotopic ossification (HO) often associated with painful, recurrent episodes of soft tissue swelling (flare-ups) that lead to ankyloses of major joints with cumulative and irreversible loss of movement and disability.

NCT03312634

Conditions

1. Fibrodysplasia Ossificans Progressiva

Interventions

1. Drug: Palovarotene

MeSH:Myositis Ossificans

- Previous participation in Clementia's natural history study (NCT02322255); clinically diagnosed with FOP, with the R206H ACVR1 mutation or other FOP variants reported to be associated with progressive HO (who have not participated in any Clementia-sponsored study); participants in Clementia's Phase 2 studies (NCT02279095 and NCT02979769) who cannot currently receive the chronic/flare-up regimen due to country of residence or those traveling long distances to participate in the Phase 2 studies. --- R206H ---

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. Study PVO-1A-201 as well as up to two new adult subjects were followed for up to 24 months. Subjects who participated under Amendment 1 will be followed for up to an additional 36 months. No new subjects will be enrolled.

NCT02979769

Conditions

1. Fibrodysplasia
2. Fibrodysplasia Ossificans Progressiva

Interventions

1. Drug: Palovarotene dose level 1
2. Drug: Palovarotene dose level 2

MeSH:Myositis Ossificans

Inclusion Criteria: - Completion of Study PVO-1A-201; or new Adult Cohort subjects with confirmed R206H mutation who (1) have had at least two symptomatic flare-ups in the past 2 years, but no flare-up symptoms within the past 4 weeks; (2) have a Cumulative Analogue Joint Involvement for FOP (CAJIS) score of 6-16 (inclusive); and (3) are able to receive 5 mg palovarotene daily. --- R206H ---

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. This 3-year, non-interventional, two-part, natural history study is designed to gain insight into total body HO, FOP disease progression, the impact of FOP on subjects' physical functioning, and clinical features and biomarkers that may be useful in the diagnosis and monitoring of disease progression. This natural history study will also provide important information to inform the design of subsequent interventional trials.

NCT02322255

Conditions

1. Fibrodysplasia Ossificans Progressiva

MeSH:Myositis Ossificans

Inclusion Criteria: - Subjects clinically diagnosed with classical FOP with documented R206H mutation or believed to carry the R206H mutation Exclusion Criteria: - Participation in an interventional clinical research study within the 4 weeks prior to enrollment Inclusion Criteria: - Subjects clinically diagnosed with classical FOP with documented R206H mutation or believed to carry the R206H mutation Exclusion Criteria: - Participation in an interventional clinical research study within the 4 weeks prior to enrollment Fibrodysplasia Ossificans Progressiva Myositis Ossificans This is a multi-center, natural history, non-interventional, longitudinal study in subjects with classic FOP. --- R206H ---

Inclusion Criteria: - Subjects clinically diagnosed with classical FOP with documented R206H mutation or believed to carry the R206H mutation Exclusion Criteria: - Participation in an interventional clinical research study within the 4 weeks prior to enrollment Inclusion Criteria: - Subjects clinically diagnosed with classical FOP with documented R206H mutation or believed to carry the R206H mutation Exclusion Criteria: - Participation in an interventional clinical research study within the 4 weeks prior to enrollment Fibrodysplasia Ossificans Progressiva Myositis Ossificans This is a multi-center, natural history, non-interventional, longitudinal study in subjects with classic FOP. --- R206H --- --- R206H ---

Inclusion Criteria: - Subjects clinically diagnosed with classical FOP with documented R206H mutation or believed to carry the R206H mutation Exclusion Criteria: - Participation in an interventional clinical research study within the 4 weeks prior to enrollment Inclusion Criteria: - Subjects clinically diagnosed with classical FOP with documented R206H mutation or believed to carry the R206H mutation Exclusion Criteria: - Participation in an interventional clinical research study within the 4 weeks prior to enrollment Fibrodysplasia Ossificans Progressiva Myositis Ossificans This is a multi-center, natural history, non-interventional, longitudinal study in subjects with classic FOP. --- R206H --- --- R206H --- --- R206H ---

Inclusion Criteria: - Subjects clinically diagnosed with classical FOP with documented R206H mutation or believed to carry the R206H mutation Exclusion Criteria: - Participation in an interventional clinical research study within the 4 weeks prior to enrollment Inclusion Criteria: - Subjects clinically diagnosed with classical FOP with documented R206H mutation or believed to carry the R206H mutation Exclusion Criteria: - Participation in an interventional clinical research study within the 4 weeks prior to enrollment Fibrodysplasia Ossificans Progressiva Myositis Ossificans This is a multi-center, natural history, non-interventional, longitudinal study in subjects with classic FOP. --- R206H --- --- R206H --- --- R206H --- --- R206H ---