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Completed

NCT Number: NCT04665323

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

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.

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Key information

Conditions

Fibrodysplasia Ossificans Progressiva Muscular Diseases Musculoskeletal Diseases Myositis Myositis Ossificans

Sex eligibility

All sexes

Study type

Observational

Primary location

Ipsen Central Contact

Cambridge, Massachusetts, 02142, United States

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 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

Exclusion criteria

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

Treatment and study plan

Primary outcomes

  1. 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)

    Time frame: Baseline

  2. Quality of life of people living with FOP and their family members, measured using the Patient-Reported Outcomes Measurement Information System (PROMIS)

    Time frame: Baseline

  3. Caregiver burden for the parent primary caregiver, measured using the Zarit Burden Interview (ZBI)

    Time frame: Baseline

  4. Responses to bespoke questionnaire describing the emotional burden on family members

    Time frame: Baseline

  5. Physical function of the person living with FOP, measured using the FOP Physical Function Questionnaire (FOP-PFQ).

    Time frame: Baseline

  6. Joint function of the person living with FOP, measured using Patient-Reported Mobility Assessment (PRMA).

    Time frame: Baseline

  7. Responses to bespoke questionnaire describing the types of healthcare services utilized by the person living with FOP over the last two years

    Time frame: Baseline

  8. Responses to bespoke questionnaire describing the frequency of utilization of healthcare services by the person living with FOP over the last two years.

    Time frame: Baseline

  9. Responses to bespoke questionnaire describing the impact of FOP on modifications to the living environment (e.g. home modifications) and travels.

    Time frame: Baseline

  10. Responses to bespoke questionnaire describing the expenses paid by the family for the care of the person living with FOP

    Time frame: Baseline

  11. Responses to bespoke questionnaire describing the Impact of FOP on education, employment, and career choices for people living with FOP and their family members

    Time frame: Baseline

Sponsors and collaborators

Lead sponsor

Ipsen

Industry

Registry information

Important dates

Study start
2021
Primary completion
2021
Study completion
2021
First posted
Dec 11, 2020
Registry last updated
May 21, 2021

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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