CompletedNot Applicable

An International Cross-sectional Survey to Evaluate the Burden of Fibrodysplasia Ossificans Progressiva (FOP) on Patients and Their Families.

United States411 participantsStarted 2021-01-18

Plain-language summary

The FOP burden of illness (BoI) survey aims to assess the impact of the burden of FOP on patients and their families. The study is being conducted online and available for residents in Argentina, Brazil, Canada, France, Germany, Italy, Japan, Mexico, Poland, Russia, South Korea, Spain, Sweden, the US, and the UK.

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

. Individuals meeting one of the two categories below (A or B) are eligible to participate: A. Any individual with FOP, of any age; B. Any individual who is a family member of a person with FOP (i.e. either a parent / legal guardian or a sibling) and who is aged 18 years and older
. All adult participants and parents / legal guardians of minors should provide informed consent before starting filling out the survey

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

Quality of life of people living with FOP and their family members, measured using the EuroQol health-related quality of life (QoL) questionnaire (EQ-5D-5L)

Timeframe: Baseline

Quality of life of people living with FOP and their family members, measured using the Patient-Reported Outcomes Measurement Information System (PROMIS)

Timeframe: Baseline

Caregiver burden for the parent primary caregiver, measured using the Zarit Burden Interview (ZBI)

Timeframe: Baseline

Responses to bespoke questionnaire describing the emotional burden on family members

Timeframe: Baseline

Physical function of the person living with FOP, measured using the FOP Physical Function Questionnaire (FOP-PFQ).

Timeframe: Baseline

Joint function of the person living with FOP, measured using Patient-Reported Mobility Assessment (PRMA).

Timeframe: Baseline

Trial details

NCT IDNCT04665323

SponsorIpsen

Sponsor typeINDUSTRY

Study typeOBSERVATIONAL

Primary completion2021-04-30

View on ClinicalTrials.gov More Fibrodysplasia Ossificans Progressiva 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

. Individuals meeting one of the two categories below (A or B) are eligible to participate: A. Any individual with FOP, of any age; B. Any individual who is a family member of a person with FOP (i.e. either a parent / legal guardian or a sibling) and who is aged 18 years and older
. All adult participants and parents / legal guardians of minors should provide informed consent before starting filling out the survey

What they're measuring

Quality of life of people living with FOP and their family members, measured using the EuroQol health-related quality of life (QoL) questionnaire (EQ-5D-5L)

Timeframe: Baseline

Quality of life of people living with FOP and their family members, measured using the Patient-Reported Outcomes Measurement Information System (PROMIS)

Timeframe: Baseline

Caregiver burden for the parent primary caregiver, measured using the Zarit Burden Interview (ZBI)

Timeframe: Baseline

Responses to bespoke questionnaire describing the emotional burden on family members

Timeframe: Baseline

Physical function of the person living with FOP, measured using the FOP Physical Function Questionnaire (FOP-PFQ).

Timeframe: Baseline

Joint function of the person living with FOP, measured using Patient-Reported Mobility Assessment (PRMA).

Timeframe: Baseline