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

Regeneron Pharmaceuticals131 enrolled

Overview

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

Study Type

OBSERVATIONAL

Enrollment

131

Conditions

Fibrodysplasia Ossificans Progressiva (FOP)

Interventions

Non-InterventionalOTHER

No study treatment will be administered in this study.

Eligibility

Sex: ALL

Medical Language ↔ Plain English

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

Outcomes

Primary Outcomes

Demographic characteristics

Time frame: Up to 5 years

Prevalence of cardiovascular disease

Time frame: Up to 5 years

Prevalence of metabolic disease

Time frame: Up to 5 years

Prevalence of hematology disease

Time frame: Up to 5 years

Prevalence of infection

Time frame: Up to 5 years

Prevalence of respiratory disease

Time frame: Up to 5 years

Prevalence of reproductive disease

Time frame: Up to 5 years

Prevalence of neurologic disorders

Time frame: Up to 5 years

Prevalence of skeletal disorders

Time frame: Up to 5 years

Prevalence of gastrointestinal disease

Time frame: Up to 5 years

Prevalence of hearing loss

Time frame: Up to 5 years

Prevalence of dermatologic disorders

Time frame: Up to 5 years

Prevalence of neoplasms

Time frame: Up to 5 years

Average number of dispensations per FOP patient per year

Time frame: Up to 5 years

Cumulative number of dispensations per FOP patient

Time frame: Up to 5 years

Incidence of all-cause death

FOP Sub-Cohorts 1 and 2 Only

Time frame: Up to 5 years

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

Overview

Conditions

Interventions

Eligibility

Locations (1)

Outcomes

Primary Outcomes