MedPath

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

Completed
Conditions
Fibrodysplasia Ossificans Progressiva (FOP)
Interventions
Other: Non-Interventional
Registration Number
NCT06064656
Lead Sponsor
Regeneron Pharmaceuticals
Brief Summary

Primary Objective(s):

* To describe the demographic characteristics of people living with FOP

* To describe the prevalence of clinical characteristics of interest in people living with FOP

* To describe the use of key medications in people living with FOP

* To estimate the crude mortality rate among people with FOP

Detailed Description

Not available

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
131
Inclusion Criteria
  1. Confirmed FOP diagnosis
  2. Members from the International Fibrodysplasia Ossificans Progressiva Association (iFOPA) cohort who have opted-in to be tokenized by Health Verity Marketplace
  3. Must be enrolled in the closed medical claims data source for any duration of time between January 1, 2018 and December 31, 2022
Exclusion Criteria
  1. Failure to meet inclusion criteria

Study & Design

Study Type
OBSERVATIONAL
Study Design
Not specified
Arm && Interventions
GroupInterventionDescription
Sub-Cohort 1Non-InterventionalAll patients with active enrollment (closed claims) during part or all the study period.
Sub-Cohort 2Non-InterventionalAll patients with active enrollment (closed claims) augmented with open claims
Primary Outcome Measures
NameTimeMethod
Prevalence of dermatologic disordersUp to 5 years
Prevalence of cardiovascular diseaseUp to 5 years
Prevalence of metabolic diseaseUp to 5 years
Prevalence of hematology diseaseUp to 5 years
Prevalence of deafnessUp to 5 years
Demographic characteristicsUp to 5 years
Prevalence of infectionUp to 5 years
Prevalence of respiratory diseaseUp to 5 years
Prevalence of reproductive diseaseUp to 5 years
Prevalence of neurologic disordersUp to 5 years
Prevalence of skeletal disordersUp to 5 years
Prevalence of gastrointestinal diseaseUp to 5 years
Prevalence of neoplasmsUp to 5 years
Average number of prescriptions per FOP patient per yearUp to 5 years
Cumulative number of prescriptions per FOP patientUp to 5 years
Incidence of all-cause deathUp to 5 years
Secondary Outcome Measures
NameTimeMethod

Trial Locations

Locations (1)

Regeneron

🇺🇸

Tarrytown, New York, United States

© Copyright 2025. All Rights Reserved by MedPath