The Fibrodysplasia Ossificans Progressiva (FOP) Connection Registry is a global, non-interventional, voluntary database that captures demographic and disease data directly from FOP patients and their caregivers via a secure, web-based patient portal. A physician portal (in development) will allow physicians to enter clinical data about their patients. The objectives are to organize the international FOP community for participation in clinical trials; to enable FOP patients worldwide to report data in a shared forum; to improve the collective understanding of FOP natural history; and to advance the understanding of FOP treatment outcomes.
The FOP Connection Registry is a global, non-interventional, voluntary database that captures demographic and disease data directly from FOP patients and their caregivers via a secure, web-based patient portal. No experimental intervention is involved. The objectives are to organize the international FOP community for participation in clinical trials; to enable FOP patients worldwide to report data in a shared forum; to improve the collective understanding of FOP natural history; and to advance the understanding of FOP treatment outcomes. The Registry collects data through two sources. First, the Patient Portal (launched in July 2015) allows FOP patients and caregivers to enter information about their experiences living with FOP. Second, the Physician Portal (in development) will allow physicians to enter clinical data about patients under their care. The Registry will be capable of including data on specific marketed therapies under the direction and control of a sponsoring pharmaceutical company. Key identifiers will link the physician-reported data with the Patient Portal data. Participants must have a confirmed diagnosis of FOP and the participant (or a parent or legal guardian) must be willing and able to provide written informed consent. There are no exclusion criteria. Data collected in the Patient Portal include: patient demographics and diagnosis pathway; medical and dental care; clinical research participation and biospecimen donation; heterotopic ossification (bone growth and episodic flare-ups); other signs and symptoms by body system; patient-reported outcomes (physical functioning, pain, fatigue, and general health); and assistive devices, aids, attendants, and adaptations. Because the Registry is designed to accommodate participants along a broad spectrum of FOP disease severity, most of the data fields are optional, allowing participants a high degree of flexibility in how much information they contribute, which also minimizes participant burden. After completing the informed consent, participants enter their baseline (historical) data. Participants will be encouraged to update their information at least twice per year. Participants may withdraw their consent at any time without prejudice or providing an explanation. The Registry has no pre-specified end date and will continue for as long as it is sustainable and useful to the FOP community.
Study Type
OBSERVATIONAL
Enrollment
800
The International FOP Association
Casselberry, Florida, United States
RECRUITINGPatient Reported Changes in New Bone Growth Using a Patient-Directed Survey Developed by the IFOPA
The patient is able to report changes in new bone growth among 27 joints and body locations.
Time frame: Baseline, then every six months for up to 10 years
Patient Reported Changes in Episodic Flare-Ups Using a Patient-Directed Survey Developed by the IFOPA
The patient is able to report changes in episodic flare-ups among 27 joints and body locations.
Time frame: Baseline, then every six months for up to 10 years
Patient Reported Changes in Mobility Using a Patient-Directed Survey Developed by the IFOPA
The patient is able to report changes in mobility among 27 joints and body locations.
Time frame: Baseline, then every six months for up to 10 years
Patient Reported Changes in Health Resource Utilization Using a Patient-Directed Survey Developed by the IFOPA
The number of visits to a Physician and a Dentist, as well as the number of hospitalizations and reasons for the hospitalizations, are used to assess health resource utilization.
Time frame: Baseline, then every six months for up to 10 years
Patient Reported Changes in Signs Related to the Ear Using a Patient-Directed Survey Developed by the IFOPA
The patient is able to report new or continuing health issues related to the ears.
Time frame: Baseline, then every six months for up to 10 years
Patient Reported Changes in Signs Related to the Skin Using a Patient-Directed Survey Developed by the IFOPA
The patient is able to report new or continuing health issues related to the skin.
Time frame: Baseline, then every six months for up to 10 years
Patient Reported Changes in Signs Related to the Central Nervous System Using a Patient-Directed Survey Developed by the IFOPA
The patient is able to report new or continuing health issues related to the central nervous system.
Time frame: Baseline, then every six months for up to 10 years
Patient Reported Changes in Signs Related to the Pulmonary System Using a Patient-Directed Survey Developed by the IFOPA
The patient is able to report new or continuing health issues related to the pulmonary system.
Time frame: Baseline, then every six months for up to 10 years
Patient Reported Changes in Signs Related to the Gastrointestinal System Using a Patient-Directed Survey Developed by the IFOPA
The patient is able to report new or continuing health issues related to the gastrointestinal system.
Time frame: Baseline, then every six months for up to 10 years
Patient Reported Changes in Signs Related to the Cardiovascular System Using a Patient-Directed Survey Developed by the IFOPA
The patient is able to report new or continuing health issues related to the cardiovascular system.
Time frame: Baseline, then every six months for up to 10 years
Patient Reported Changes in Signs Related to the Renal System Using a Patient-Directed Survey Developed by the IFOPA
The patient is able to report new or continuing health issues related to the renal system.
Time frame: Baseline, then every six months for up to 10 years
Patient Reported Changes in Signs Related to the Endocrine System Using a Patient-Directed Survey Developed by the IFOPA
The patient is able to report new or continuing health issues related to the endocrine system.
Time frame: Baseline, then every six months for up to 10 years
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