Fibrodysplasia Ossificans ProgressivaNCT04665323
An International Cross-sectional Survey to Evaluate the Burden of Fibrodysplasia Ossificans Progressiva (FOP) on Patients and Their Families.
An observational study of Fibrodysplasia Ossificans Progressiva, run by Ipsen at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Fibrodysplasia Ossificans Progressiva
- How many people
- 411 participants
- Where
- 1 siteUnited States
- Study dates
- 18 Jan 2021to 30 Apr 2021
- Phase
- Observational
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: Convenience Sample
Who may be able to join
- 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
Who cannot join
- 1. Any individuals who are unable to complete the online survey independently or to get assistance to physically enter the answers
Trial locations and contacts
1 site in 1 country.
United States1 site
Ipsen Central Contact
Cambridge, Massachusetts, 02142
About this study
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.
- Conditions
- Fibrodysplasia Ossificans Progressiva
- Medical subject headings
- Myositis Ossificans
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| People living with FOP | Not described | |
| Parent or legal guardian primary caregivers | Not described | |
| Parent or legal guardian | Not described | |
| Siblings | Not described |
What is being measured
The primary outcome is the main question the study is designed to answer.
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)
Measured: Baseline
Quality of life of people living with FOP and their family members, measured using the Patient-Reported Outcomes Measurement Information System (PROMIS)
Measured: Baseline
Caregiver burden for the parent primary caregiver, measured using the Zarit Burden Interview (ZBI)
Measured: Baseline
Responses to bespoke questionnaire describing the emotional burden on family members
Measured: Baseline
Physical function of the person living with FOP, measured using the FOP Physical Function Questionnaire (FOP-PFQ).
Measured: Baseline
Joint function of the person living with FOP, measured using Patient-Reported Mobility Assessment (PRMA).
Measured: Baseline
Responses to bespoke questionnaire describing the types of healthcare services utilized by the person living with FOP over the last two years
Measured: Baseline
Responses to bespoke questionnaire describing the frequency of utilization of healthcare services by the person living with FOP over the last two years.
Measured: Baseline
Responses to bespoke questionnaire describing the impact of FOP on modifications to the living environment (e.g. home modifications) and travels.
Measured: Baseline
Responses to bespoke questionnaire describing the expenses paid by the family for the care of the person living with FOP
Measured: Baseline
Responses to bespoke questionnaire describing the Impact of FOP on education, employment, and career choices for people living with FOP and their family members
Measured: Baseline
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Dec 2020
- Study start18 Jan 2021
- Primary completion30 Apr 2021
- Study completion30 Apr 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Fibrodysplasia Ossificans ProgressivaStudies of the same condition, open and under way.
- More trials run by IpsenThe 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
- Other
- Time perspective
- Cross-sectional
- Enrolment
- 411 participants (actual)
Sponsor and investigators
- Lead sponsor
- Ipsen
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Ipsen Medical Director, Study director, Ipsen
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04665323
- Sponsor's protocol ID
- CLIN-60120-450
- Status last verified
- May 2021
- First submitted
- 25 Nov 2020
- Last updated
- 21 May 2021
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No