The Fibrodysplasia Ossificans Progressiva (FOP) Connection Registry

Fibrodysplasia Ossificans Progressiva (FOP) Clinical Trial

Official title:

FOP Connection: A Global Registry for the Fibrodysplasia Ossificans Progressiva Community

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT02745158
• Other study ID #: IFOPA-REG-001
• Status: Recruiting
• Phase: N/A
• First received: March 6, 2016
• Last updated: August 15, 2017
• Start date: July 2015
• Est. completion date: December 2025

Study information

• Verified date: March 2016
• Source: The International FOP Association
• Contact: Neal S Mantick, BS, MS
• Phone: 1-617-910-8508
• Email: registry[@]fopconnection.org
• Is FDA regulated: No
• Study type: Observational [Patient Registry]

Clinical Trial Summary

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.

Description:

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 800
• Est. completion date: December 2025
• Est. primary completion date: July 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

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.

Related Conditions & MeSH terms

• Fibrodysplasia Ossificans Progressiva (FOP)

Locations

Country	Name	City	State
United States	The International FOP Association	Casselberry	Florida

Sponsors (1)

Lead Sponsor	Collaborator
The International FOP Association

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Patient Reported Changes in Activities of Daily Living Using the FOP-Physical Functioning Questionnaire (PFQ)		Baseline, then every six months for up to 10 years
Other	Patient Reported Changes in Overall Health Using the PROMIS Global Health Scale		Baseline, then every six months for up to 10 years
Other	Patient Reported Changes in the Need for Assistive Devices, Aids, Attendants, and Adaptations Using a Survey Developed by the IFOPA	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.	Baseline, then every six months for up to 10 years
Primary	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.	Baseline, then every six months for up to 10 years
Primary	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.	Baseline, then every six months for up to 10 years
Primary	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.	Baseline, then every six months for up to 10 years
Secondary	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.	Baseline, then every six months for up to 10 years
Secondary	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.	Baseline, then every six months for up to 10 years
Secondary	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.	Baseline, then every six months for up to 10 years
Secondary	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.	Baseline, then every six months for up to 10 years
Secondary	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.	Baseline, then every six months for up to 10 years
Secondary	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.	Baseline, then every six months for up to 10 years
Secondary	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.	Baseline, then every six months for up to 10 years
Secondary	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.	Baseline, then every six months for up to 10 years
Secondary	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.	Baseline, then every six months for up to 10 years

External Links

•   Click here for more information about this study: The FOP Connection Registry
•   The International FOP Association is a 501(c)(3) non-profit organization supporting medical research, education and communication for those afflicted by the rare genetic condition Fibrodysplasia Ossificans Progressiva (FOP).