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

Completed

• Conditions: Fibrodysplasia Ossificans Progressiva

• Registration Number: NCT04665323
• Lead Sponsor: Ipsen

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2020-11-25
• Study First Submitted QC: 2020-12-10
• Study First Posted: 2020-12-11
• Study Start: 2021-01-18
• Primary Completion: 2021-04-30
• Study Completion: 2021-04-30
• Status Verified: 2021-05
• Last Update Submitted: 2021-05-20
• Last Update Posted: 2021-05-21

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 411

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

Read More

Exclusion Criteria

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

Read More

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Responses to bespoke questionnaire describing the impact of FOP on modifications to the living environment (e.g. home modifications) and travels.	Baseline
Responses to bespoke questionnaire describing the expenses paid by the family for the care of the person living with FOP	Baseline
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)	Baseline
Quality of life of people living with FOP and their family members, measured using the Patient-Reported Outcomes Measurement Information System (PROMIS)	Baseline
Caregiver burden for the parent primary caregiver, measured using the Zarit Burden Interview (ZBI)	Baseline
Responses to bespoke questionnaire describing the emotional burden on family members	Baseline
Physical function of the person living with FOP, measured using the FOP Physical Function Questionnaire (FOP-PFQ).	Baseline
Joint function of the person living with FOP, measured using Patient-Reported Mobility Assessment (PRMA).	Baseline
Responses to bespoke questionnaire describing the types of healthcare services utilized by the person living with FOP over the last two years	Baseline
Responses to bespoke questionnaire describing the frequency of utilization of healthcare services by the person living with FOP over the last two years.	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	Baseline

Trial Locations

Locations (1)

Ipsen Central Contact; 🇺🇸 Cambridge, Massachusetts, United States