Fibrodysplasia Ossificans Progressiva (FOP)
Conditions
Keywords
Genetic disease, Heterotopic ossification (HO), Skeletal dysplasia, Activin A receptor, type I (ACVR1)
Brief summary
Primary Objective(s): * To describe the demographic characteristics of people living with FOP and a matched cohort of non-FOP people living in the United States (U.S.) * To describe the prevalence of clinical characteristics of interest and the use of key medications in people living with FOP and a matched cohort of non-FOP people living in the U.S. * To compare the prevalence of key clinical characteristics and medication use in people living with FOP to a matched cohort of non-FOP people living in the U.S. * To estimate the crude mortality rate among people living with FOP
Interventions
OTHERNon-Interventional
No study treatment will be administered in this study.
Sponsors
Regeneron Pharmaceuticals
Study design
Observational model
COHORT
Time perspective
RETROSPECTIVE
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
Inclusion criteria
FOP Cohort(s): 1. Living with FOP in the United States who have been identified from the IFOPA membership database 2. Confirmed FOP diagnosis through IFOPA membership database will be tokenized and linked with HealthVerity's medical claims data, pharmacy claims data, laboratory data and obituary data, as defined in the protocol Comparator Cohort: 1. People without FOP living in the U.S. randomly sampled from HealthVerity's closed medical claims data and matched to the FOP population 2. Index date are defined as each person's cohort entry date. People may exit and re-enter the cohort, with the period during which they were not in the cohort excluded from the analysis 3. The comparator cohort will be matched to the cohort of people with FOP on the following variables: age, sex, insurance type and first index date
Exclusion criteria
1\. Failure to meet inclusion criteria
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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 hearing loss | Up to 5 years | — |
| Prevalence of dermatologic disorders | Up to 5 years | — |
| Prevalence of neoplasms | Up to 5 years | — |
| Average number of dispensations per FOP patient per year | Up to 5 years | — |
| Cumulative number of dispensations per FOP patient | Up to 5 years | — |
| Incidence of all-cause death | Up to 5 years | FOP Sub-Cohorts 1 and 2 Only |
Countries
United States
Outcome results
None listed