Clinical trial NCT04665323: An International Cross-sectional Survey to Evaluate the Burden of Fibrodysplasia Ossificans Progressiva (FOP) on Patients and Their Families.
Official title: An International Cross-sectional Survey to Evaluate the Burden of Fibrodysplasia Ossificans Progressiva (FOP) on Patients and Their Families.
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Fibrodysplasia Ossificans Progressiva
Lead sponsor: Ipsen
Sponsor class: INDUSTRY
First posted: 2020-12-11
Enrollment: 411
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: OTHER
Time perspective: CROSS_SECTIONAL
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Convenience Sample

Brief 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.


Eligibility criteria: Inclusion Criteria:

1. 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
2. All adult participants and parents / legal guardians of minors should provide informed consent before starting filling out the survey

Exclusion Criteria:

1. Any individuals who are unable to complete the online survey independently or to get assistance to physically enter the answers

Primary outcomes:
measure: 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
measure: Quality of life of people living with FOP and their family members, measured using the Patient-Reported Outcomes Measurement Information System (PROMIS)
timeFrame: Baseline
measure: Caregiver burden for the parent primary caregiver, measured using the Zarit Burden Interview (ZBI)
timeFrame: Baseline
measure: Responses to bespoke questionnaire describing the emotional burden on family members
timeFrame: Baseline
measure: Physical function of the person living with FOP, measured using the FOP Physical Function Questionnaire (FOP-PFQ).
timeFrame: Baseline
measure: Joint function of the person living with FOP, measured using Patient-Reported Mobility Assessment (PRMA).
timeFrame: Baseline
measure: Responses to bespoke questionnaire describing the types of healthcare services utilized by the person living with FOP over the last two years
timeFrame: Baseline
measure: Responses to bespoke questionnaire describing the frequency of utilization of healthcare services by the person living with FOP over the last two years.
timeFrame: Baseline
measure: Responses to bespoke questionnaire describing the impact of FOP on modifications to the living environment (e.g. home modifications) and travels.
timeFrame: Baseline
measure: Responses to bespoke questionnaire describing the expenses paid by the family for the care of the person living with FOP
timeFrame: Baseline
measure: Responses to bespoke questionnaire describing the Impact of FOP on education, employment, and career choices for people living with FOP and their family members
timeFrame: Baseline
Secondary outcomes:
Other outcomes:
Locations:
city: Cambridge
country: United States
facility: Ipsen Central Contact
state: Massachusetts
Study officials:
affiliation: Ipsen
name: Ipsen Medical Director
role: STUDY_DIRECTOR
References: