SNPMiner Trials by Shray Alag


SNPMiner Trials: Clinical Trial Report


Report for Clinical Trial NCT02322255

Developed by Shray Alag, 2019.
SNP Clinical Trial Gene

A Natural History, Non-Interventional, Two-Part Study in Subjects With Fibrodysplasia Ossificans Progressiva (FOP)

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 Fibrodysplasia Ossificans Progressiva
MeSH: Myositis Ossificans


Primary Outcomes

Measure: Change from baseline in the total body burden of heterotopic ossification as assessed by the optimal imaging modality (low-dose whole body CT [excluding head]).

Time: Month 36

Secondary Outcomes

Measure: Change from baseline in physical function as assessed by range of motion.

Time: Month 12, Month 24, and Month 36

Measure: Change from baseline in patient-reported use of assistive devices and adaptations.

Time: Month 6, Month 12, Month 18, Month 24, Month 30, and Month 36

Measure: Change from baseline in a disease-specific patient-reported outcome measure (FOP-Physical Function Questionnaire [FOP-PFQ]).

Time: Month 6, Month 12, Month 18, Month 24, Month 30, and Month 36

Measure: Change from baseline in a patient-reported measure of physical and mental health (PROMIS Global Health Scale).

Time: Month 6, Month 12, Month 18, Month 24, Month 30, and Month 36

Measure: Change from baseline in biomarkers.

Time: Month 12, Month 24, and Month 36

Measure: Flare-up progression as assessed by the change from baseline in heterotopic ossification at the flare-up site.

Time: Flare-up initiation, Flare-up Days 42 and 84

Measure: Flare-up progression as assessed by the change from baseline in pain and swelling at the flare-up site.

Time: Flare-up initiation, Flare-up Days 42 and 84

Measure: Flare-up progression as assessed by the change from baseline biomarkers.

Time: Flare-up initiation, Flare-up Days 42 and 84

Measure: Flare-up progression as assessed by the change from baseline in physical function as assessed by range of motion.

Time: Flare-up initiation, Flare-up Days 42 and 84

Measure: Flare-up progression as assessed by the change from baseline in a disease-specific patient-reported outcome measure (FOP-Physical Function Questionnaire [FOP-PFQ]).

Time: Flare-up initiation, Flare-up Days 42 and 84

Measure: Flare-up progression as assessed by the change from baseline in a patient-reported outcome measure of physical and mental health (PROMIS Global Health Scale).

Time: Flare-up initiation, Flare-up Days 42 and 84

Time Perspective: Prospective

Case-Only


There is one SNP

SNPs


1 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 ---

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 ---



HPO Nodes