CompletedNot Applicable

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

United States131 participantsStarted 2023-06-29

Plain-language 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

Who can participate

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

. 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

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Demographic characteristics

Timeframe: Up to 5 years

Prevalence of cardiovascular disease

Timeframe: Up to 5 years

Prevalence of metabolic disease

Timeframe: Up to 5 years

Prevalence of hematology disease

Timeframe: Up to 5 years

Prevalence of infection

Timeframe: Up to 5 years

Prevalence of respiratory disease

Timeframe: Up to 5 years

Prevalence of reproductive disease

Timeframe: Up to 5 years

Prevalence of neurologic disorders

Timeframe: Up to 5 years

Trial details

NCT IDNCT06064656

SponsorRegeneron Pharmaceuticals

Sponsor typeINDUSTRY

Study typeOBSERVATIONAL

Primary completion2023-09-22

View on ClinicalTrials.gov More Fibrodysplasia Ossificans Progressiva (FOP) trials

Plain-language summary

Who can participate

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

. 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

What they're measuring

Demographic characteristics

Timeframe: Up to 5 years

Prevalence of cardiovascular disease

Timeframe: Up to 5 years

Prevalence of metabolic disease

Timeframe: Up to 5 years

Prevalence of hematology disease

Timeframe: Up to 5 years

Prevalence of infection

Timeframe: Up to 5 years

Prevalence of respiratory disease

Timeframe: Up to 5 years

Prevalence of reproductive disease

Timeframe: Up to 5 years

Prevalence of neurologic disorders

Timeframe: Up to 5 years