The Fibrodysplasia Ossificans Progressiva (FOP) Connection Registry
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| ClinicalTrials.gov Identifier: NCT02745158 |
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Recruitment Status :
Recruiting
First Posted : April 20, 2016
Last Update Posted : August 17, 2017
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| Condition or disease |
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| Fibrodysplasia Ossificans Progressiva (FOP) |
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 [Patient Registry] |
| Estimated Enrollment : | 800 participants |
| Observational Model: | Cohort |
| Time Perspective: | Prospective |
| Target Follow-Up Duration: | 10 Years |
| Official Title: | FOP Connection: A Global Registry for the Fibrodysplasia Ossificans Progressiva Community |
| Actual Study Start Date : | July 2015 |
| Estimated Primary Completion Date : | July 2025 |
| Estimated Study Completion Date : | December 2025 |
| Group/Cohort |
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| FOP Patients |
- Patient Reported Changes in New Bone Growth Using a Patient-Directed Survey Developed by the IFOPA [ Time Frame: Baseline, then every six months for up to 10 years ]The patient is able to report changes in new bone growth among 27 joints and body locations.
- Patient Reported Changes in Episodic Flare-Ups Using a Patient-Directed Survey Developed by the IFOPA [ Time Frame: Baseline, then every six months for up to 10 years ]The patient is able to report changes in episodic flare-ups among 27 joints and body locations.
- Patient Reported Changes in Mobility Using a Patient-Directed Survey Developed by the IFOPA [ Time Frame: Baseline, then every six months for up to 10 years ]The patient is able to report changes in mobility among 27 joints and body locations.
- Patient Reported Changes in Health Resource Utilization Using a Patient-Directed Survey Developed by the IFOPA [ Time Frame: Baseline, then every six months for up to 10 years ]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.
- Patient Reported Changes in Signs Related to the Ear Using a Patient-Directed Survey Developed by the IFOPA [ Time Frame: Baseline, then every six months for up to 10 years ]The patient is able to report new or continuing health issues related to the ears.
- Patient Reported Changes in Signs Related to the Skin Using a Patient-Directed Survey Developed by the IFOPA [ Time Frame: Baseline, then every six months for up to 10 years ]The patient is able to report new or continuing health issues related to the skin.
- Patient Reported Changes in Signs Related to the Central Nervous System Using a Patient-Directed Survey Developed by the IFOPA [ Time Frame: Baseline, then every six months for up to 10 years ]The patient is able to report new or continuing health issues related to the central nervous system.
- Patient Reported Changes in Signs Related to the Pulmonary System Using a Patient-Directed Survey Developed by the IFOPA [ Time Frame: Baseline, then every six months for up to 10 years ]The patient is able to report new or continuing health issues related to the pulmonary system.
- Patient Reported Changes in Signs Related to the Gastrointestinal System Using a Patient-Directed Survey Developed by the IFOPA [ Time Frame: Baseline, then every six months for up to 10 years ]The patient is able to report new or continuing health issues related to the gastrointestinal system.
- Patient Reported Changes in Signs Related to the Cardiovascular System Using a Patient-Directed Survey Developed by the IFOPA [ Time Frame: Baseline, then every six months for up to 10 years ]The patient is able to report new or continuing health issues related to the cardiovascular system.
- Patient Reported Changes in Signs Related to the Renal System Using a Patient-Directed Survey Developed by the IFOPA [ Time Frame: Baseline, then every six months for up to 10 years ]The patient is able to report new or continuing health issues related to the renal system.
- Patient Reported Changes in Signs Related to the Endocrine System Using a Patient-Directed Survey Developed by the IFOPA [ Time Frame: Baseline, then every six months for up to 10 years ]The patient is able to report new or continuing health issues related to the endocrine system.
- Patient Reported Changes in Activities of Daily Living Using the FOP-Physical Functioning Questionnaire (PFQ) [ Time Frame: Baseline, then every six months for up to 10 years ]
- Patient Reported Changes in Overall Health Using the PROMIS Global Health Scale [ Time Frame: Baseline, then every six months for up to 10 years ]
- Patient Reported Changes in the Need for Assistive Devices, Aids, Attendants, and Adaptations Using a Survey Developed by the IFOPA [ Time Frame: Baseline, then every six months for up to 10 years ]The patient is able to respond Yes or No to questions related to his/her need for Assistive Devices, Aids, Attendants, and Adaptations as a result of his/her FOP disease.
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
| Ages Eligible for Study: | Child, Adult, Older Adult |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
| Sampling Method: | Probability Sample |
Inclusion Criteria:
- Participants must have a confirmed diagnosis of FOP.
- Participants (or a parent or legal guardian) must be willing and able to provide written informed consent.
Exclusion Criteria:
- There are no exclusion criteria.
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): NCT02745158
| Contact: Neal S Mantick, BS, MS | 1-617-910-8508 | registry@fopconnection.org | |
| Contact: Betsy Bogard, BS, MS | 1-407-365-4194 | registry@fopconnection.org |
| United States, Florida | |
| The International FOP Association | Recruiting |
| Casselberry, Florida, United States, 32707 | |
| Contact: Neal S Mantick, BS, MS 617-910-8508 registry@fopconnection.org | |
| Study Director: | Betsy Bogard, BS, MS | The International FOP Association |
Additional Information:
| Responsible Party: | The International FOP Association |
| ClinicalTrials.gov Identifier: | NCT02745158 History of Changes |
| Other Study ID Numbers: |
IFOPA-REG-001 |
| First Posted: | April 20, 2016 Key Record Dates |
| Last Update Posted: | August 17, 2017 |
| Last Verified: | March 2016 |
| Individual Participant Data (IPD) Sharing Statement: | |
| Plan to Share IPD: | Yes |
| Plan Description: | Formal data sharing agreements are planned between the International FOP Association and biopharmaceutical companies doing research in Fibrodysplasia Ossificans Progressiva (FOP). Publications are also planned with biopharmaceutical companies and physician researchers. |
Keywords provided by The International FOP Association:
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Fibrodysplasia ossificans progressiva FOP International FOP Association Patient Advocacy Group Patient Registry Natural History Patient-Reported Outcomes Observational Database Bone Morphogenetic Protein BMP Bone Growth Joint |
Mobility Flare-Up Heterotopic Ossification Ectopic Bone Osteochondroma Hallux Valgus ALK2 ACVR1 Activin A IFOPA Activin A Receptor Type I Activin Receptor-like Kinase-2 |
Additional relevant MeSH terms:
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Myositis Ossificans Myositis Muscular Diseases Musculoskeletal Diseases |