Fibrodysplasia Ossificans Progressiva (FOP)NCT06064656
A Non-Interventional Study of Clinical Characteristics and Mortality of US Patients With Fibrodysplasia Ossificans Progressiva (FOP)
An observational study of Fibrodysplasia Ossificans Progressiva (FOP), run by Regeneron Pharmaceuticals at 1 site in United States.
Trial 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… Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Fibrodysplasia Ossificans Progressiva (FOP)
- What is being tested?
- Non-Interventional
- How many people
- 131 participants
- Where
- 1 siteUnited States
- Study dates
- 29 Jun 2023to 29 Sep 2023
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
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.
Who may be able to join
- 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
Who cannot join
- 1. Failure to meet inclusion criteria
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Clinical Characteristics and Mortality in Patients With Fibrodysplasia Ossificans Progressiva (FOP): A Non-Interventional US National Retrospective Cohort
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
- Being tested
- Non-Interventional
- Keywords
- Genetic disease, Heterotopic ossification (HO), Skeletal dysplasia, Activin A receptor, type I (ACVR1)
- Medical subject headings
- Myositis Ossificans, Genetic Diseases, Inborn, Ossification, Heterotopic, Mucopolysaccharidosis IV
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Non-InterventionalOther
No study treatment will be administered in this study.
| Study group | Role | What they receive |
|---|---|---|
| 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) Receives: 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) Receives: Other: Non-Interventional |
What is being measured
The primary outcome is the main question the study is designed to answer.
Demographic characteristics
Measured: Up to 5 years
Prevalence of cardiovascular disease
Measured: Up to 5 years
Prevalence of metabolic disease
Measured: Up to 5 years
Prevalence of hematology disease
Measured: Up to 5 years
Prevalence of infection
Measured: Up to 5 years
Prevalence of respiratory disease
Measured: Up to 5 years
Prevalence of reproductive disease
Measured: Up to 5 years
Prevalence of neurologic disorders
Measured: Up to 5 years
Prevalence of skeletal disorders
Measured: Up to 5 years
Prevalence of gastrointestinal disease
Measured: Up to 5 years
Prevalence of hearing loss
Measured: Up to 5 years
Prevalence of dermatologic disorders
Measured: Up to 5 years
Prevalence of neoplasms
Measured: Up to 5 years
Average number of dispensations per FOP patient per year
Measured: Up to 5 years
Cumulative number of dispensations per FOP patient
Measured: Up to 5 years
Incidence of all-cause death
FOP Sub-Cohorts 1 and 2 Only
Measured: Up to 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Oct 2023
- Study start29 Jun 2023
- Primary completion22 Sep 2023
- Study completion29 Sep 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Fibrodysplasia Ossificans Progressiva (FOP)Studies of the same condition, open and under way.
- More trials run by Regeneron PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Retrospective
- Enrolment
- 131 participants (actual)
Sponsor and investigators
- Lead sponsor
- Regeneron Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Trial Management, Study director, Regeneron Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06064656
- Sponsor's protocol ID
- R2477-FOP-2143
- Status last verified
- Jun 2025
- First submitted
- 11 Sep 2023
- Last updated
- 3 Jul 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 3 Jul 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Clinical Characteristics and Mortality in Patients With Fibrodysplasia Ossificans Progressiva (FOP): A Non-Interventional US National Retrospective Cohort
- ClinicalTrials.gov ID
- NCT06064656
- Acronym / sponsor ID
- R2477-FOP-2143
- Keywords
- Genetic disease, Heterotopic ossification (HO), Skeletal dysplasia, Activin A receptor, type I (ACVR1)
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 131 participants (actual)
- Lead sponsor
- Regeneron PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 29 Jun 2023 (actual)
- Primary completion
- 22 Sep 2023 (actual)
- Study completion
- 29 Sep 2023 (actual)
- First posted
- 3 Oct 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| FOP Cohort | – | Other: Non-InterventionalSub-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-InterventionalAll individuals without FOP and with active enrollment (closed claims) during part or all of the study period (2018-2023) |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- 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.
Key inclusion criteria (7 listed)
- 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
- 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
Key exclusion criteria (1 listed)
- 1. Failure to meet inclusion criteria
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Non-InterventionalNo study treatment will be administered in this study. | Other | Comparator Cohort, FOP Cohort |
Primary outcome
Demographic characteristics
Time frame: Up to 5 years
Prevalence of cardiovascular disease
Time frame: Up to 5 years
Prevalence of metabolic disease
Time frame: Up to 5 years
Prevalence of hematology disease
Time frame: Up to 5 years
Prevalence of infection
Time frame: Up to 5 years
Prevalence of respiratory disease
Time frame: Up to 5 years
Prevalence of reproductive disease
Time frame: Up to 5 years
Prevalence of neurologic disorders
Time frame: Up to 5 years
Prevalence of skeletal disorders
Time frame: Up to 5 years
Prevalence of gastrointestinal disease
Time frame: Up to 5 years
Prevalence of hearing loss
Time frame: Up to 5 years
Prevalence of dermatologic disorders
Time frame: Up to 5 years
Prevalence of neoplasms
Time frame: Up to 5 years
Average number of dispensations per FOP patient per year
Time frame: Up to 5 years
Cumulative number of dispensations per FOP patient
Time frame: Up to 5 years
Incidence of all-cause death
Time frame: Up to 5 years
FOP Sub-Cohorts 1 and 2 Only
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Jul 2025.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Clinical Trial Management study director
Regeneron Pharmaceuticals
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Fibrodysplasia Ossificans Progressiva (FOP), run by Regeneron Pharmaceuticals at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 3 Jul 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Demographic characteristics (time frame: Up to 5 years). The study lists 16 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Jul 2025.