Fibrodysplasia Ossificans Progressiva (FOP)NCT02745158
The Fibrodysplasia Ossificans Progressiva (FOP) Registry
An observational study of Fibrodysplasia Ossificans Progressiva (FOP), run by The International FOP Association at 1 site in United States.
Trial summary
The Fibrodysplasia Ossificans Progressiva (FOP) 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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Fibrodysplasia Ossificans Progressiva (FOP)
- How many people
- 800 participants
- Where
- 1 siteUnited States
- Study dates
- Jul 2015to 31 Dec 2040
- How long will I take part?
- 20 Years
- Phase
- Observational
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Individuals with Fibrodysplasia Ossificans Progressive (FOP) from the international FOP community will be invited to join the Registry.
Who may be able to join
- 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.
Who cannot join
- There are no exclusion criteria.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
The International Fibrodysplasia Ossificans Progressiva Association (IFOPA)
North Kansas City, Missouri, 064116
Mark S Hamilton, PhD, 1-203-605=2122, mark.hamilton@ifopa.org
Sammi Kile, MS, 1-720-341-6999, registry@ifopa.org
About this study
Official title: FOP Registry: A Global Registry for the Fibrodysplasia Ossificans Progressiva Community
The Fibrodysplasia Ossificans Progressiva (FOP) 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.
Read the detailed description
The FOP 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.
- Keywords
- 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
- Medical subject headings
- Myositis Ossificans, Ossification, Heterotopic, Osteochondroma, Hallux Valgus
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| FOP Patients | None. IFOPA-REG-001 is a FOP patient observational registry study. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Patient 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.
Measured: 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.
Measured: 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.
Measured: Baseline, then every six months for up to 10 years
Secondary outcomes (9)
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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: Baseline, then every six months for up to 10 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Apr 2016 (estimated)
- Study startJul 2015
- Primary completion31 Jul 2035 (estimated)
- Study completion31 Dec 2040 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Fibrodysplasia Ossificans Progressiva (FOP)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Other
- Time perspective
- Prospective
- Enrolment
- 800 participants (estimated)
- Target duration
- 20 Years
Sponsor and investigators
- Lead sponsor
- The International FOP Association
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Mark Hamilton, PhD, Study director, International Fibrodysplasia Ossificans Progressiva Association
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02745158
- Sponsor's protocol ID
- IFOPA-REG-001
- Status last verified
- Apr 2026
- First submitted
- 6 Mar 2016
- Last updated
- 8 Apr 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: 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.
Publications
Pignolo RJ, Kimel M, Whalen J, Kawata AK, Artyomenko A, Kaplan FS. The Fibrodysplasia Ossificans Progressiva Physical Function Questionnaire (FOP-PFQ): A patient-reported, disease-specific measure. Bone. 2023 Mar;168:116642. doi: 10.1016/j.bone.2022.116642. Epub 2022 Dec 13.
PubMed 36526263