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

NCT04665323 | Completed

• 1 other identifier: CLIN-60120-450
• Study Type: observational
• Target: 411
• Locations: 1 country
• Sites: 1

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.

Conditions

Fibrodysplasia Ossificans Progressiva

Outcome Measures

Primary Outcomes (11)

• 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 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

• 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

Study Arms (4)

People living with FOP

Parent or legal guardian primary caregivers

Parent or legal guardian

Siblings

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Convenience Sample

You may qualify if:

• 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

You may not qualify if:

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

Sponsors & Collaborators

• Ipsen: lead

Study Sites (1)

Ipsen Central Contact

Cambridge, Massachusetts, 02142, United States

Location

MeSH Terms

Conditions

Myositis Ossificans

Condition Hierarchy (Ancestors)

Myositis; Muscular Diseases; Musculoskeletal Diseases

Study Officials

• Ipsen Medical Director

  Ipsen

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: OTHER
• Time Perspective: CROSS SECTIONAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 25, 2020
• First Posted: December 11, 2020
• Study Start: January 18, 2021
• Primary Completion: April 30, 2021
• Study Completion: April 30, 2021
• Last Updated: May 21, 2021

Record last verified: 2021-05

Locations

US (1)