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

NCT ID: NCT06064656

Last Updated: 2025-07-03

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Total Enrollment

131 participants

Study Classification

OBSERVATIONAL

Study Start Date

2023-06-29

Study Completion Date

2023-09-29

Brief Summary

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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

Detailed Description

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Conditions

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Fibrodysplasia Ossificans Progressiva (FOP)

Study Design

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Observational Model Type

COHORT

Study Time Perspective

RETROSPECTIVE

Study Groups

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FOP Cohort

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)

Non-Interventional

Intervention Type OTHER

No study treatment will be administered in this study.

Comparator Cohort

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

Non-Interventional

Intervention Type OTHER

No study treatment will be administered in this study.

Interventions

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Non-Interventional

No study treatment will be administered in this study.

Intervention Type OTHER

Eligibility Criteria

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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
Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Regeneron Pharmaceuticals

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Clinical Trial Management

Role: STUDY_DIRECTOR

Regeneron Pharmaceuticals

Locations

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Regeneron

Tarrytown, New York, United States

Site Status

Countries

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United States

Other Identifiers

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R2477-FOP-2143

Identifier Type: -

Identifier Source: org_study_id

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