A Non-Interventional Study of Clinical Characteristics and Mortality of US Patients With Fibrodysplasia Ossificans Progressiva (FOP)

Study Overview
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Brief Summary
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Primary Objective(s): To describe the demographic characteristics of people living with FOP To describe the prevalence of clinical characteristics of interest in people living with FOP To describe the use of key medications in people living with FOP To estimate the crude mortality rate among people with FOP

Official Title
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Clinical Characteristics and Mortality in Patients With Fibrodysplasia Ossificans Progressiva (FOP): A Non-Interventional US National Retrospective Cohort

Conditions
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Fibrodysplasia Ossificans Progressiva (FOP)

Intervention / Treatment
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* Other: Non-Interventional


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Confirmed FOP diagnosis Members from the International Fibrodysplasia Ossificans Progressiva Association (iFOPA) cohort who have opted-in to be tokenized by Health Verity Marketplace Must be enrolled in the closed medical claims data source for any duration of time between January 1, 2018 and December 31, 2022 Exclusion Criteria: 1. Failure to meet inclusion criteria
Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Sub-Cohort 1<br>All patients with active enrollment (closed claims) during part or all the study period. | Other: Non-Interventional<br>* No study treatment will be administered on this study.<br>|
| Sub-Cohort 2<br>All patients with active enrollment (closed claims) augmented with open claims | Other: Non-Interventional<br>* No study treatment will be administered on this study.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Demographic characteristics |  | Up to 5 years | 
| Prevalence of cardiovascular disease |  | Up to 5 years | 
| Prevalence of metabolic disease |  | Up to 5 years | 
| Prevalence of hematology disease |  | Up to 5 years | 
| Prevalence of infection |  | Up to 5 years | 
| Prevalence of respiratory disease |  | Up to 5 years | 
| Prevalence of reproductive disease |  | Up to 5 years | 
| Prevalence of neurologic disorders |  | Up to 5 years | 
| Prevalence of skeletal disorders |  | Up to 5 years | 
| Prevalence of gastrointestinal disease |  | Up to 5 years | 
| Prevalence of deafness |  | Up to 5 years | 
| Prevalence of dermatologic disorders |  | Up to 5 years | 
| Prevalence of neoplasms |  | Up to 5 years | 
| Average number of prescriptions per FOP patient per year |  | Up to 5 years | 
| Cumulative number of prescriptions per FOP patient |  | Up to 5 years | 
| Incidence of all-cause death |  | Up to 5 years | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Genetic disease, Heterotopic ossification (HO), Skeletal dysplasia, Activin A receptor, type I (ACVR1)