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CompletedNCT06064656Updated Jul 3, 2025

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

An observational study in Fibrodysplasia Ossificans Progressiva (FOP), sponsored by Regeneron Pharmaceuticals. Completed at 1 site in United States. Per ClinicalTrials.gov, last updated 2025-07-03.

Sponsored by Regeneron Pharmaceuticals · Observational

Study type
Observational
Model
Cohort
Time perspective
Retrospective
Enrollment
131
Sex
All
01

Study 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
02

Conditions studied

  • Fibrodysplasia Ossificans Progressiva (FOP)

Keywords

  • Genetic disease
  • Heterotopic ossification (HO)
  • Skeletal dysplasia
  • Activin A receptor, type I (ACVR1)
03

In context

Myositis Ossificans

25 studies on the registry are indexed under Myositis Ossificans; 5 are open to participants now.

Browse Myositis Ossificans studies →

Lead sponsor

Regeneron Pharmaceuticals is the lead sponsor of 400 studies on the registry; 91 are open to participants now.

Of its 120 completed or terminated interventional studies of FDA-regulated products, 77 (64%) have results posted.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
Child (0–17), Adult (18–64), Older adult (65+)
Sexes eligible
All
Accepts healthy volunteers
No
Sampling method
Non-probability sample

Study population

FOP Cohort(s):

The International Fibrodysplasia Ossificans Progressiva Association (IFOPA) membership has 235 confirmed people living with FOP in the US. From this membership database, there were 131 members or next of kin of members who opted-in to allow for the tokenization of their membership data to occur.

Comparator Cohort:

A random sample of 100,000 people from an insured population without FOP diagnosis living in the U.S. and matched to the FOP population.

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

Exclusion Criteria:

1. Failure to meet inclusion criteria

05

Study design

Observational model
Cohort
Time perspective
Retrospective
Enrollment
131 participants (actual)
Patient registry
No

Groups and cohorts

  • 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)

    Other: Non-Interventional

  • Comparator Cohort

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

    Other: Non-Interventional

Interventions

  • OtherNon-Interventional

    No study treatment will be administered in this study.

06

What researchers measure

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

    FOP Sub-Cohorts 1 and 2 Only

    Time frame: Up to 5 years

07

Study locations

1 site
  • Regeneron
    Tarrytown, New York 10591, United States
08

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Jul 3, 2025, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
09

Registry details

Key details

Study ID
NCT06064656
Lead sponsor
Regeneron Pharmaceuticals
Responsible party
Sponsor
First posted
Oct 3, 2023
Start date
Jun 29, 2023
Primary completion
Sep 22, 2023
Completion
Sep 29, 2023
Last update
Jul 3, 2025

Study contacts

Clinical Trial Management
study director · Regeneron Pharmaceuticals

Oversight

Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is completed, as verified in Jun 2025. You cannot join it, but the record below documents what was studied.

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