Clinical trial NCT02745158: The Fibrodysplasia Ossificans Progressiva (FOP) Connection Registry
Official title: FOP Connection: A Global Registry for the Fibrodysplasia Ossificans Progressiva Community
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Fibrodysplasia Ossificans Progressiva (FOP)
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
Activin A
IFOPA
Activin A Receptor Type I
Activin Receptor-like Kinase-2
Lead sponsor: The International FOP Association
Sponsor class: OTHER
First posted: 2016-04-20
Enrollment: 800
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: Individuals with Fibrodysplasia Ossificans Progressive (FOP) from the international FOP community will be invited to join the Registry.

Brief 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.

Detailed 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.

Eligibility criteria: 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.

Primary outcomes:
measure: Patient Reported Changes in New Bone Growth Using a Patient-Directed Survey Developed by the IFOPA
timeFrame: Baseline, then every six months for up to 10 years
measure: Patient Reported Changes in Episodic Flare-Ups Using a Patient-Directed Survey Developed by the IFOPA
timeFrame: Baseline, then every six months for up to 10 years
measure: Patient Reported Changes in Mobility Using a Patient-Directed Survey Developed by the IFOPA
timeFrame: Baseline, then every six months for up to 10 years
Secondary outcomes:
measure: Patient Reported Changes in Health Resource Utilization Using a Patient-Directed Survey Developed by the IFOPA
timeFrame: Baseline, then every six months for up to 10 years
measure: Patient Reported Changes in Signs Related to the Ear Using a Patient-Directed Survey Developed by the IFOPA
timeFrame: Baseline, then every six months for up to 10 years
measure: Patient Reported Changes in Signs Related to the Skin Using a Patient-Directed Survey Developed by the IFOPA
timeFrame: Baseline, then every six months for up to 10 years
measure: Patient Reported Changes in Signs Related to the Central Nervous System Using a Patient-Directed Survey Developed by the IFOPA
timeFrame: Baseline, then every six months for up to 10 years
measure: Patient Reported Changes in Signs Related to the Pulmonary System Using a Patient-Directed Survey Developed by the IFOPA
timeFrame: Baseline, then every six months for up to 10 years
measure: Patient Reported Changes in Signs Related to the Gastrointestinal System Using a Patient-Directed Survey Developed by the IFOPA
timeFrame: Baseline, then every six months for up to 10 years
measure: Patient Reported Changes in Signs Related to the Cardiovascular System Using a Patient-Directed Survey Developed by the IFOPA
timeFrame: Baseline, then every six months for up to 10 years
measure: Patient Reported Changes in Signs Related to the Renal System Using a Patient-Directed Survey Developed by the IFOPA
timeFrame: Baseline, then every six months for up to 10 years
measure: Patient Reported Changes in Signs Related to the Endocrine System Using a Patient-Directed Survey Developed by the IFOPA
timeFrame: Baseline, then every six months for up to 10 years
Other outcomes:
measure: Patient Reported Changes in Activities of Daily Living Using the FOP-Physical Functioning Questionnaire (PFQ)
timeFrame: Baseline, then every six months for up to 10 years
measure: Patient Reported Changes in Overall Health Using the PROMIS Global Health Scale
timeFrame: Baseline, then every six months for up to 10 years
measure: Patient Reported Changes in the Need for Assistive Devices, Aids, Attendants, and Adaptations Using a Survey Developed by the IFOPA
timeFrame: Baseline, then every six months for up to 10 years
Locations:
city: Casselberry
country: United States
facility: The International FOP Association
state: Florida
Study officials:
affiliation: The International FOP Association
name: Betsy Bogard, BS, MS
role: STUDY_DIRECTOR
References: