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

Fibrodysplasia Ossificans Progressiva Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04665323
• Other study ID #: CLIN-60120-450
• Status: Completed
• Start date: January 18, 2021
• Est. completion date: April 30, 2021

Study information

• Verified date: May 2021
• Source: Ipsen
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 411
• Est. completion date: April 30, 2021
• Est. primary completion date: April 30, 2021
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

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

Related Conditions & MeSH terms

• Fibrodysplasia Ossificans Progressiva

Locations

Country	Name	City	State
United States	Ipsen Central Contact	Cambridge	Massachusetts

Sponsors (1)

Lead Sponsor	Collaborator
Ipsen

Country where clinical trial is conducted

United States, 

Outcome

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