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

Regeneron Pharmaceuticals

Status: Completed

Conditions: Fibrodysplasia Ossificans Progressiva (FOP)

Treatments: Other: Non-Interventional

Study type: Observational

Funder types: Industry

Identifiers

NCT06064656

R2477-FOP-2143

Details and patient eligibility

About

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

Enrollment: 131 patients

Sex: All

Volunteers: No Healthy Volunteers

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

Trial design

131 participants in 2 patient groups

FOP Cohort

Description:

Sub-Cohort 1: All patients with active enrollment (closed claims) during part or all the study period (2018-2023) Sub-Cohort 2: All patients with both active enrollment (closed claims) and open claims encounters during study period (2018-2022)

Treatment:

Other: Non-Interventional

Comparator Cohort

Description:

All individuals without FOP and with active enrollment (closed claims) during part or all of the study period (2018-2023)

Treatment:

Other: Non-Interventional