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Completed

NCT Number: NCT06064656

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

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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Key information

Conditions

Fibrodysplasia Ossificans Progressiva (FOP) Carbohydrate Metabolism, Inborn Errors Congenital, Hereditary, and Neonatal Diseases and Abnormalities Connective Tissue Diseases Genetic Diseases, Inborn Lysosomal Storage Diseases Metabolic Diseases Metabolism, Inborn Errors Mucinoses Mucopolysaccharidoses Mucopolysaccharidosis IV Muscular Diseases Musculoskeletal Diseases Myositis Myositis Ossificans Nutritional and Metabolic Diseases Ossification, Heterotopic Pathologic Processes Pathological Conditions, Signs and Symptoms Skin and Connective Tissue Diseases

Sex eligibility

All sexes

Study type

Observational

Primary location

Regeneron

Tarrytown, New York, 10591, United States

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

FOP Cohort(s):

  • Living with FOP in the United States who have been identified from the IFOPA membership database
  • 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:

  • People without FOP living in the U.S. randomly sampled from HealthVerity's closed medical claims data and matched to the FOP population
  • 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
  • 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

  • Failure to meet inclusion criteria

Treatment and study plan

Non-Interventional

Other

No study treatment will be administered in this study.

Primary outcomes

  1. Demographic characteristics

    Time frame: Up to 5 years

  2. Prevalence of cardiovascular disease

    Time frame: Up to 5 years

  3. Prevalence of metabolic disease

    Time frame: Up to 5 years

  4. Prevalence of hematology disease

    Time frame: Up to 5 years

  5. Prevalence of infection

    Time frame: Up to 5 years

  6. Prevalence of respiratory disease

    Time frame: Up to 5 years

  7. Prevalence of reproductive disease

    Time frame: Up to 5 years

  8. Prevalence of neurologic disorders

    Time frame: Up to 5 years

  9. Prevalence of skeletal disorders

    Time frame: Up to 5 years

  10. Prevalence of gastrointestinal disease

    Time frame: Up to 5 years

  11. Prevalence of hearing loss

    Time frame: Up to 5 years

  12. Prevalence of dermatologic disorders

    Time frame: Up to 5 years

  13. Prevalence of neoplasms

    Time frame: Up to 5 years

  14. Average number of dispensations per FOP patient per year

    Time frame: Up to 5 years

  15. Cumulative number of dispensations per FOP patient

    Time frame: Up to 5 years

  16. Incidence of all-cause death

    Time frame: Up to 5 years

    FOP Sub-Cohorts 1 and 2 Only

Sponsors and collaborators

Lead sponsor

Regeneron Pharmaceuticals

Industry

Registry information

Official study title

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

Important dates

Study start
2023
Primary completion
2023
Study completion
2023
First posted
Oct 3, 2023
Registry last updated
Jul 3, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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