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

NCT06064656 — Completed, 131 participants, Regeneron Pharmaceuticals

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

Registry	NCT06064656
Status	Completed
Study type	Observational
Enrollment	131 (Actual)
Sponsor	Regeneron Pharmaceuticals (Industry)
Conditions	Fibrodysplasia Ossificans Progressiva (FOP)
Interventions	Non-Interventional (Other)
Dates	start 2023-06-29, primary completion 2023-09-22, completion 2023-09-29, first posted 2023-10-03, last updated 2025-07-03
Locations	1 site in United States

-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

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+Arms and interventions

Arm	Type	Interventions	Description
FOP Cohort		Other: Non-Interventional	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)
Comparator Cohort		Other: Non-Interventional	All individuals without FOP and with active enrollment (closed claims) during part or all of the study period (2018-2023)

Non-Interventional (Other): No study treatment will be administered in this study.

+Outcome measures

Primary outcomes

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

+Eligibility

Sex: All

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

+Locations (1)

Facility	City	Country	Status
Regeneron	Tarrytown, New York	United States

+Investigators

Clinical Trial Management, Study director, Regeneron Pharmaceuticals

My Notes

Public

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