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

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ClinicalTrials.gov Identifier: NCT04665323
Recruitment Status : Completed
First Posted : December 11, 2020
Last Update Posted : May 21, 2021
Sponsor:
Information provided by (Responsible Party):
Ipsen

Study Description
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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.

Condition or disease
Fibrodysplasia Ossificans Progressiva

Study Design
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Study Type : Observational
Actual Enrollment : 411 participants
Observational Model: Other
Time Perspective: Cross-Sectional
Official Title: An International Cross-sectional Survey to Evaluate the Burden of Fibrodysplasia Ossificans Progressiva (FOP) on Patients and Their Families.
Actual Study Start Date : January 18, 2021
Actual Primary Completion Date : April 30, 2021
Actual Study Completion Date : April 30, 2021

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Family Issues

Groups and Cohorts
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Group/Cohort
People living with FOP
Parent or legal guardian primary caregivers
Parent or legal guardian
Siblings


Outcome Measures
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Primary Outcome Measures :
  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 ]

Eligibility Criteria
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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Convenience Sample
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

Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04665323


Locations
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United States, Massachusetts
Ipsen Central Contact
Cambridge, Massachusetts, United States, 02142
Sponsors and Collaborators
Ipsen
Investigators
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Study Director: Ipsen Medical Director Ipsen
More Information
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Responsible Party: Ipsen
ClinicalTrials.gov Identifier: NCT04665323    
Other Study ID Numbers: CLIN-60120-450
First Posted: December 11, 2020    Key Record Dates
Last Update Posted: May 21, 2021
Last Verified: May 2021

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Studies a U.S. FDA-regulated Drug Product: No
Additional relevant MeSH terms:
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Myositis Ossificans
Myositis
Muscular Diseases
Musculoskeletal Diseases